Multiple Myeloma
Conditions
Brief summary
The investigators hypothesize that prophylactic E-selectin inhibition via administration of uproleselan during melphalan conditioning will reduce the gastrointestinal (GI) toxicity in multiple myeloma (MM) patients undergoing auto-transplant, as assessed via diarrhea severity scoring per CTCAE v5.0, while potentially increasing chemosensitivity of malignant MM cells to high-dose melphalan.
Interventions
Sponsors
GlycoMimetics Incorporated
The Foundation for Barnes-Jewish Hospital
Washington University School of Medicine
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
SUPPORTIVE_CARE
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Biopsy-confirmed multiple myeloma (MM) (per IMWG criteria). * Undergoing first auto-HCT for MM in first partial response (PR) or better * Conditioning regimen to be single agent melphalan (200 mg/m\^2) * Adults 18 to 75 years of age, inclusive * ECOG performance status ≤ 2 * Mobilized ≥ 5.0 x 10\^6 CD34+ cells/kg (i.e. sufficient CD34+ HSCs for one auto-HCT, with at least one back-up graft in reserve) * Adequate bone marrow and organ function prior to stem cell mobilization as defined below: * Leukocytes, absolute neutrophil count, and platelets within institutional standard limits for high-dose melphalan autologous stem cell transplant * Total bilirubin ≤ 1.5 x ULN (unless the patient has a history of Gilbert's Syndrome, in which case, total bilirubin must be ≤ 2.5 times the ULN) * AST(SGOT)/ALT(SGPT) ≤ 3.0 x ULN * Creatinine clearance ≥ 30 mL/min by Cockcroft-Gault * Baseline pulmonary function test (PFT) with carbon monoxide diffusion capacity in the lung (DLCO) ≥ 50% and forced expiratory volume in 1 second (FEV1) both within institutional standard limits for high-dose melphalan autologous stem cell transplant * The effects of uproleselan (GMI-1271) on the developing human fetus are unknown. For this reason, women of childbearing potential and men must agree to use adequate contraception (hormonal or barrier method of birth control, prior sterilization procedure, abstinence, etc.) prior to study entry, for the duration of study participation and for 12 weeks after the completion of the study. Should a woman become pregnant or suspect she is pregnant while participating in this study, she must inform her treating physician immediately. Should a man who is participating in the study become aware that he has impregnated a partner, he must inform his treating physician immediately. * Ability to understand and willingness to sign an IRB approved written informed consent document (or that of legally authorized representative, if applicable).
Exclusion criteria
* A history of other malignancy with the exception of malignancies for which all treatment was completed at least 2 years before registration and the patient has no evidence of disease. * Active signs or symptoms of CNS involvement by malignancy (lumbar puncture not required). Prior history of CNS involvement is acceptable, if patient has completed treatment for CNS involvement with documented treatment response. * Prior exposure to uproleselan (GMI-1271) * Currently receiving any other investigational agents * A history of allergic reactions attributed to compounds of similar chemical or biologic composition to uproleselan or melphalan * Known active infection with hepatitis A, B (e.g., HBsAg positive), or C (e.g., anti-HCV positive), or human immunodeficiency virus * Uncontrolled acute life-threatening bacterial, viral, or fungal infection-Myocardial infarction within 6 months of uproleselan/placebo dosing, or subject has current significant cardiovascular disease, such as uncontrolled or symptomatic arrhythmias, congestive heart failure, hemodynamic instability, or any Class 3 or 4 cardiac disease as defined by the New York Heart Association Functional Classification * Any medical, psychiatric, or other condition which, in the opinion of the investigator, unfavorably alters the risk-benefit of subject participation, is likely to interfere with trial completion, assessments, or interpretation of trial results, or otherwise would make the subject an inappropriate subject for this trial. * Pregnant and/or breastfeeding. * Women of childbearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly effective contraception during the trial and for 12 weeks following the last dose of uproleselan/placebo. Women who are postmenopausal with amenorrhea for at least 1 year prior to trial entry and follicle stimulating hormone (FSH) serum levels consistent with postmenopausal status (\>28U/L) will be considered NOT of childbearing potential. Highly effective contraception includes: * Total abstinence with a male partner. * Female sterilization (has had surgical bilateral oophorectomy with or without hysterectomy) or tubal ligation at least 6 weeks before uproleselan/placebo. In case of oophorectomy alone, the subject would be eligible only when the reproductive status of the woman has been confirmed by follow up hormone level assessment. * Male sterilization (at least 6 months prior to Screening). For female subjects on the trial, the vasectomized male partner should be the sole partner for that subject. * BOTH of the following forms of contraception consistently used together: * Injected, transdermal, or implanted hormonal methods of contraception or other forms of hormonal contraception that have comparable efficacy (failure rate \<1%) with the exception of intrauterine devices, which are excluded due to the risk of infection and bleeding. * Barrier methods of contraception: condom or occlusive cap (diaphragm or cervical/vault caps) with or without spermicidal foam/gel/film/cream/vaginal suppository. * Men who are sexually active and not willing to use condoms during the trial and for 12 weeks following the last dose of uproleselan/placebo, unless they have undergone vasectomy for sterilization (at least 6 months prior to Screening), are excluded from trial participation. * Men who are sexually active and not willing to use condoms during the trial and for 12 weeks following the last dose of uproleselan/placebo, unless they have undergone vasectomy for sterilization (at least 6 months prior to Screening), are excluded from trial participation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Change in Diarrhea as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 is defined as no diarrhea, or no change from baseline. Grade 1 is defined as an increase of \<4 stools per day over baseline; mild increase in ostomy output compared to baseline. Grade 2 is defined as an increase of 4-6 stools per day over baseline; moderate increase in ostomy output compared to baseline; limiting instrumental ADL. Grade 3 is defined as an increase of \>=7 stools per day over baseline; hospitalization indicated; severe increase in ostomy output compared to baseline; limited self care ADL. Grade 4 is defined as life-threatening consequences; urgent intervention indicated. Grade 5 is defined as death. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change in Esophagitis as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 esophagitis is defined as no presence of esophagitis. Grade 1 esophagitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 esophagitis is defined as symptomatic; altered GI function; limiting instrumental ADL. Grade 3 esophagitis is defined as severely altered GI function; TPN indicated; elective invasive intervention indicated; limiting self care ADL. Grade 4 esophagitis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 esophagitis is defined as death. |
| Change in Gastritis as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 gastritis is defined as no presence of esophagitis. Grade 1 gastritis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 gastritis is defined as symptomatic; altered GI function; medical intervention indicated. Grade 3 gastritis is defined as severely altered eating or gastric function; TPN or hospitalization indicated. Grade 4 gastritis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 gastritis is defined as death. |
| Change in Esophageal Pain as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 esophageal pain is defined as no esophageal pain. Grade 1 esophageal pain is defined as mild pain. Grade 2 esophageal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 esophageal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 esophageal pain defined in the CTCAE v5.0. |
| Change in Abdominal Pain as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 abdominal pain is defined as no abdominal pain. Grade 1 abdominal pain is defined as mild pain. Grade 2 abdominal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 abdominal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 abdominal pain defined in the CTCAE v5.0. |
| Change in Nausea as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 nausea is defined as no nausea. Grade 1 nausea is defined as loss of appetite without alteration in eating habits. Grade 2 nausea is defined as oral intake decreased without significant weight loss, dehydration or malnutrition. Grade 3 nausea is defined as inadequate oral caloric or fluid intake; tube feeding, TPN, or hospitalization indicated. There is no grade 4 or 5 nausea defined in the CTCAE v5.0. |
| Change in Vomiting as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 vomiting is defined as no vomiting. Grade 1 vomiting is defined as intervention not indicated. Grade 2 vomiting is defined as outpatient IV hydration; medical intervention indicated. Grade 3 vomiting is defined as tube feeding, TPN, or hospitalization indicated. Grade 4 vomiting is defined as life-threatening consequences. Grade 5 vomiting is defined as death. |
| Change in Enterocolitis as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 enterocolitis is defined as no enterocolitis. Grade 1 enterocolitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 enterocolitis is defined as abdominal pain; mucus or blood in stool. Grade 3 enterocolitis is defined as severe or persistent abdominal pain; fever; ileus; peritoneal signs. Grade 4 enterocolitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 enterocolitis is defined as death. |
| Change in Proctitis as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 proctitis is defined as no proctitis. Grade 1 proctitis is defined as rectal discomfort, intervention not indicated. Grade 2 proctitis is defined as symptomatic (e.g., rectal discomfort, passing blood or mucus); medical intervention indicated; limiting instrumental ADL. Grade 3 proctitis is defined as severe symptoms; fecal urgency or stool incontinence; limiting self-care ADL. Grade 4 proctitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 proctitis is defined as death. |
| Change in Hemorrhoids as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 hemorrhoids is defined as no hemorrhoids. Grade 1 hemorrhoids is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 hemorrhoids is defined as symptomatic; banding or medical intervention indicated. Grade 3 hemorrhoids is defined as severe symptoms; invasive intervention indicated. There is no grade 4 or 5 hemorrhoids defined in the CTCAE v5.0. |
| Time to Neutrophil Engraftment | Through date of discharge (up to be 18 days) | -Defined as ANC ≥0.5 x 10\^9/L for 3 consecutive days or ≥1.0 x 10\^9/L for 1 day |
| Change in Oral Mucositis as Assessed Per CTCAE v5.0 | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Grade 0 oral mucositis is defined as no presence of mucositis. Grade 1 oral mucositis is defined as asymptomatic or mild symptoms; intervention not indicated. Grade 2 oral mucositis is defined as moderate pain or ulcer that does not interfere with oral intake; modified diet indicated. Grade 3 oral mucositis is defined as severe pain; interfering with oral intake. Grade 4 oral mucositis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 oral mucositis is defined as death. |
| Duration of Hospital Length of Stay | From date of admission for auto-HCT to date of discharge (up to be 18 days) | — |
| Change in Bristol Stool Scale | From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days) | Change in Bristol Stool Scale as measured by incidence of Type 7 Bristol Stool Scale: liquid consistency with no solid pieces. |
| Change in Nutritional Status as Assessed by Change in Standing Weight | Day -3, Day 8, and date of discharge or Day 14 (whichever is sooner) (up to be 18 days) | Patient standing weight in kilograms was taken at specific time points to assess any changes in nutritional status. |
| Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available) | Through date of discharge (upto be 18 days) | — |
| Time to First Antibiotics | Through date of discharge (up to be day 18) | Measured by the time in days to the first antibiotic dose for bacteremia. |
| Incidence of Clostridium Difficile Infections | Through date of discharge (up to be 18 days) | — |
| Median Daily Dose of Anti-diarrheal Medications | Through date of discharge (up to be 18 days) | This is defined as the number of doses of anti-diarrheal medications, such as loperamide or lomotil, that participants took daily. |
| Median Daily Dose of Pain Medications | Through date of discharge (up to be 18 days) | The median daily dose of pain medications is provided as morphine equivalents. |
| Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days) | * Questions regarding gastrointestinal toxicities * Responses are scored from 1-5 with 1=no symptoms to 5=severe symptoms |
| Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days) | * Questions regarding gastrointestinal, pain, and psychological symptoms interfering with daily activities * Responses are scored from 1-5 with 1=not at all to 5=very much |
| Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days | Before conditioning and at day +14 or date of discharge (whichever is sooner) (up to be 18 days) | — |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Uproleselan + Standard of Care Melphalan Patients will receive six doses of Uproleselan intravenously. The doses will begin the evening of Day -3 and continue every 12 hours through the morning of Day 0, prior to hematopoietic stem cell transplant (auto-HCT). On the evening of Day -2, patients also receive one dose of standard of care Melphalan intravenously. | 26 |
| Placebo + Standard of Care Melphalan Patients will receive six doses of placebo intravenously. The doses will begin the evening of Day -3 and continue every 12 hours through the morning of Day 0, prior to hematopoietic stem cell transplant (auto-HCT). On the evening of Day -2, patients also receive one dose of standard of care Melphalan intravenously. | 25 |
| Total | 51 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Adverse Event | 0 | 1 |
Baseline characteristics
| Characteristic | Uproleselan + Standard of Care Melphalan | Total | Placebo + Standard of Care Melphalan |
|---|---|---|---|
| Age, Continuous | 65.5 years | 64 years | 64 years |
| Ethnicity (NIH/OMB) Hispanic or Latino | 0 Participants | 0 Participants | 0 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 26 Participants | 51 Participants | 25 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Asian | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Black or African American | 0 Participants | 2 Participants | 2 Participants |
| Race (NIH/OMB) More than one race | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants |
| Race (NIH/OMB) White | 26 Participants | 49 Participants | 23 Participants |
| Region of Enrollment United States | 26 Participants | 51 Participants | 25 Participants |
| Sex: Female, Male Female | 9 Participants | 20 Participants | 11 Participants |
| Sex: Female, Male Male | 17 Participants | 31 Participants | 14 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 2 / 26 | 3 / 25 |
| other Total, other adverse events | 26 / 26 | 25 / 25 |
| serious Total, serious adverse events | 5 / 26 | 5 / 25 |
Outcome results
Primary
Change in Diarrhea as Assessed Per CTCAE v5.0
Grade 0 is defined as no diarrhea, or no change from baseline. Grade 1 is defined as an increase of \<4 stools per day over baseline; mild increase in ostomy output compared to baseline. Grade 2 is defined as an increase of 4-6 stools per day over baseline; moderate increase in ostomy output compared to baseline; limiting instrumental ADL. Grade 3 is defined as an increase of \>=7 stools per day over baseline; hospitalization indicated; severe increase in ostomy output compared to baseline; limited self care ADL. Grade 4 is defined as life-threatening consequences; urgent intervention indicated. Grade 5 is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 1 | 0.23 CTCAE grade | Standard Deviation 0.51 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 6 | 1.19 CTCAE grade | Standard Deviation 0.85 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -1 | 0.31 CTCAE grade | Standard Deviation 0.55 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 7 | 1.42 CTCAE grade | Standard Deviation 0.95 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 2 | 0.42 CTCAE grade | Standard Deviation 0.58 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 8 | 1.38 CTCAE grade | Standard Deviation 0.85 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 9 | 1.54 CTCAE grade | Standard Deviation 0.86 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 3 | 0.65 CTCAE grade | Standard Deviation 0.75 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 10 | 1.54 CTCAE grade | Standard Deviation 0.86 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 0 | 0.19 CTCAE grade | Standard Deviation 0.4 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 4 | 0.88 CTCAE grade | Standard Deviation 0.71 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 12 | 1.23 CTCAE grade | Standard Deviation 0.95 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 13 | 1.00 CTCAE grade | Standard Deviation 0.98 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 5 | 1.00 CTCAE grade | Standard Deviation 0.75 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 14 | 0.92 CTCAE grade | Standard Deviation 0.76 |
| Uproleselan + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 11 | 1.46 CTCAE grade | Standard Deviation 1.07 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 14 | 0.95 CTCAE grade | Standard Deviation 0.72 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -3 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -2 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day -1 | 0.38 CTCAE grade | Standard Deviation 0.58 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 0 | 0.21 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 1 | 0.25 CTCAE grade | Standard Deviation 0.53 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 2 | 0.46 CTCAE grade | Standard Deviation 0.66 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 3 | 0.88 CTCAE grade | Standard Deviation 0.85 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 4 | 1.08 CTCAE grade | Standard Deviation 1.02 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 5 | 1.29 CTCAE grade | Standard Deviation 0.55 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 6 | 1.42 CTCAE grade | Standard Deviation 0.97 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 7 | 1.38 CTCAE grade | Standard Deviation 0.77 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 8 | 1.63 CTCAE grade | Standard Deviation 0.92 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 9 | 1.54 CTCAE grade | Standard Deviation 0.93 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 10 | 1.92 CTCAE grade | Standard Deviation 0.97 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 11 | 1.63 CTCAE grade | Standard Deviation 0.92 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 12 | 1.17 CTCAE grade | Standard Deviation 0.92 |
| Placebo + Standard of Care Melphalan | Change in Diarrhea as Assessed Per CTCAE v5.0 | Day 13 | 1.13 CTCAE grade | Standard Deviation 0.76 |
Secondary
Change in Abdominal Pain as Assessed Per CTCAE v5.0
Grade 0 abdominal pain is defined as no abdominal pain. Grade 1 abdominal pain is defined as mild pain. Grade 2 abdominal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 abdominal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 abdominal pain defined in the CTCAE v5.0.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 2 | 0.35 CTCAE grade | Standard Deviation 0.56 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 0 | 0.16 CTCAE grade | Standard Deviation 0.37 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 1 | 0.19 CTCAE grade | Standard Deviation 0.4 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 3 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 4 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 5 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 6 | 0.31 CTCAE grade | Standard Deviation 0.68 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 7 | 0.35 CTCAE grade | Standard Deviation 0.63 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 8 | 0.58 CTCAE grade | Standard Deviation 0.7 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 9 | 0.54 CTCAE grade | Standard Deviation 0.81 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 10 | 0.38 CTCAE grade | Standard Deviation 0.64 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 11 | 0.15 CTCAE grade | Standard Deviation 0.37 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 12 | 0.23 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 13 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 14 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 10 | 0.46 CTCAE grade | Standard Deviation 0.78 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 1 | 0.17 CTCAE grade | Standard Deviation 0.38 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 6 | 0.21 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 14 | 0.05 CTCAE grade | Standard Deviation 0.21 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 7 | 0.29 CTCAE grade | Standard Deviation 0.46 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day -1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 11 | 0.38 CTCAE grade | Standard Deviation 0.65 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 0 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 8 | 0.54 CTCAE grade | Standard Deviation 0.88 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 2 | 0.25 CTCAE grade | Standard Deviation 0.44 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 13 | 0.22 CTCAE grade | Standard Deviation 0.42 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 3 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 9 | 0.50 CTCAE grade | Standard Deviation 0.98 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 4 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 12 | 0.29 CTCAE grade | Standard Deviation 0.62 |
| Placebo + Standard of Care Melphalan | Change in Abdominal Pain as Assessed Per CTCAE v5.0 | Day 5 | 0.13 CTCAE grade | Standard Deviation 0.34 |
Secondary
Change in Bristol Stool Scale
Change in Bristol Stool Scale as measured by incidence of Type 7 Bristol Stool Scale: liquid consistency with no solid pieces.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 6, AM | 12 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -1, PM | 0 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 6, PM | 12 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 2, AM | 3 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 7, AM | 14 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -2, PM | 0 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 7, PM | 15 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 2, PM | 2 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 8, AM | 14 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 0, AM | 1 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 8, PM | 19 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 3, AM | 11 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 9, AM | 18 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -2, AM | 0 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 9, PM | 13 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 3, PM | 5 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 10, AM | 13 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 0, PM | 0 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 10, PM | 10 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 4, AM | 8 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 11, AM | 9 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -1, AM | 1 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 11, PM | 8 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 4, PM | 4 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 12, AM | 11 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 1, AM | 5 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 12, PM | 8 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 5, AM | 7 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 13, AM | 6 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -3, PM | 0 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 13, PM | 3 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 5, PM | 6 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 14, AM | 3 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 1, PM | 1 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 14, PM | 2 Participants |
| Uproleselan + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -3, AM | 0 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 14, PM | 4 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -3, AM | 1 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -3, PM | 1 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -2, AM | 2 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -2, PM | 1 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -1, AM | 0 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day -1, PM | 1 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 0, AM | 3 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 0, PM | 1 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 1, AM | 4 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 1, PM | 3 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 2, AM | 5 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 2, PM | 6 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 3, AM | 8 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 3, PM | 10 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 4, AM | 12 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 4, PM | 11 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 5, AM | 12 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 5, PM | 10 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 6, AM | 11 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 6, PM | 11 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 7, AM | 10 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 7, PM | 12 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 8, AM | 15 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 8, PM | 14 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 9, AM | 16 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 9, PM | 17 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 10, AM | 16 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 10, PM | 19 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 11, AM | 19 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 11, PM | 11 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 12, AM | 9 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 12, PM | 7 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 13, AM | 7 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 13, PM | 8 Participants |
| Placebo + Standard of Care Melphalan | Change in Bristol Stool Scale | Day 14, AM | 7 Participants |
Secondary
Change in Enterocolitis as Assessed Per CTCAE v5.0
Grade 0 enterocolitis is defined as no enterocolitis. Grade 1 enterocolitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 enterocolitis is defined as abdominal pain; mucus or blood in stool. Grade 3 enterocolitis is defined as severe or persistent abdominal pain; fever; ileus; peritoneal signs. Grade 4 enterocolitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 enterocolitis is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 2 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 5 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 6 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 7 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 8 | 0.27 CTCAE grade | Standard Deviation 0.67 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 9 | 0.27 CTCAE grade | Standard Deviation 0.6 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 10 | 0.23 CTCAE grade | Standard Deviation 0.59 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 11 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 12 | 0.08 CTCAE grade | Standard Deviation 0.39 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 13 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 14 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 1 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 2 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 11 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 12 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 6 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 13 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 7 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 8 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 14 | 0.05 CTCAE grade | Standard Deviation 0.21 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 9 | 0.38 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Enterocolitis as Assessed Per CTCAE v5.0 | Day 10 | 0.38 CTCAE grade | Standard Deviation 0.82 |
Secondary
Change in Esophageal Pain as Assessed Per CTCAE v5.0
Grade 0 esophageal pain is defined as no esophageal pain. Grade 1 esophageal pain is defined as mild pain. Grade 2 esophageal pain is defined as moderate pain; limiting instrumental ADL. Grade 3 esophageal pain is defined as severe pain; limiting self care ADL. There is no grade 4 or 5 esophageal pain defined in the CTCAE v5.0.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 2 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 4 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 6 | 0.19 CTCAE grade | Standard Deviation 0.49 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 7 | 0.35 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 8 | 0.46 CTCAE grade | Standard Deviation 0.81 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 9 | 0.46 CTCAE grade | Standard Deviation 0.71 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 10 | 0.38 CTCAE grade | Standard Deviation 0.64 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 11 | 0.31 CTCAE grade | Standard Deviation 0.55 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 12 | 0.19 CTCAE grade | Standard Deviation 0.49 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 13 | 0.23 CTCAE grade | Standard Deviation 0.59 |
| Uproleselan + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 14 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 10 | 0.33 CTCAE grade | Standard Deviation 0.76 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 6 | 0.29 CTCAE grade | Standard Deviation 0.75 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 14 | 0.14 CTCAE grade | Standard Deviation 0.47 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 7 | 0.46 CTCAE grade | Standard Deviation 0.88 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 0 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 11 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 8 | 0.33 CTCAE grade | Standard Deviation 0.76 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 2 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 13 | 0.13 CTCAE grade | Standard Deviation 0.46 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 9 | 0.29 CTCAE grade | Standard Deviation 0.69 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 4 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 12 | 0.17 CTCAE grade | Standard Deviation 0.56 |
| Placebo + Standard of Care Melphalan | Change in Esophageal Pain as Assessed Per CTCAE v5.0 | Day 5 | 0.13 CTCAE grade | Standard Deviation 0.34 |
Secondary
Change in Esophagitis as Assessed Per CTCAE v5.0
Grade 0 esophagitis is defined as no presence of esophagitis. Grade 1 esophagitis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 esophagitis is defined as symptomatic; altered GI function; limiting instrumental ADL. Grade 3 esophagitis is defined as severely altered GI function; TPN indicated; elective invasive intervention indicated; limiting self care ADL. Grade 4 esophagitis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 esophagitis is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 6 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 7 | 0.23 CTCAE grade | Standard Deviation 0.71 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 2 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 8 | 0.58 CTCAE grade | Standard Deviation 1.03 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 9 | 0.54 CTCAE grade | Standard Deviation 0.9 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 10 | 0.46 CTCAE grade | Standard Deviation 0.86 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 11 | 0.38 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 12 | 0.19 CTCAE grade | Standard Deviation 0.57 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 13 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 14 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 14 | 0.14 CTCAE grade | Standard Deviation 0.35 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 6 | 0.17 CTCAE grade | Standard Deviation 0.48 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 7 | 0.25 CTCAE grade | Standard Deviation 0.68 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 8 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 9 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 10 | 0.25 CTCAE grade | Standard Deviation 0.61 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 11 | 0.29 CTCAE grade | Standard Deviation 0.62 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 12 | 0.21 CTCAE grade | Standard Deviation 0.51 |
| Placebo + Standard of Care Melphalan | Change in Esophagitis as Assessed Per CTCAE v5.0 | Day 13 | 0.13 CTCAE grade | Standard Deviation 0.34 |
Secondary
Change in Gastritis as Assessed Per CTCAE v5.0
Grade 0 gastritis is defined as no presence of esophagitis. Grade 1 gastritis is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 gastritis is defined as symptomatic; altered GI function; medical intervention indicated. Grade 3 gastritis is defined as severely altered eating or gastric function; TPN or hospitalization indicated. Grade 4 gastritis is defined as life-threatening consequences; urgent operative intervention indicated. Grade 5 gastritis is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 8 | 0.19 CTCAE grade | Standard Deviation 0.63 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 2 | 0.15 CTCAE grade | Standard Deviation 0.46 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 4 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 5 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 6 | 0.08 CTCAE grade | Standard Deviation 0.39 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 7 | 0.12 CTCAE grade | Standard Deviation 0.59 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 9 | 0.23 CTCAE grade | Standard Deviation 0.65 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 10 | 0.19 CTCAE grade | Standard Deviation 0.63 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 11 | 0.12 CTCAE grade | Standard Deviation 0.59 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 12 | 0.08 CTCAE grade | Standard Deviation 0.39 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 13 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 14 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 10 | 0.13 CTCAE grade | Standard Deviation 0.45 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 7 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 14 | 0.05 CTCAE grade | Standard Deviation 0.21 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 6 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 11 | 0.17 CTCAE grade | Standard Deviation 0.48 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 8 | 0.04 CTCAE grade | Standard Deviation 0.21 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 13 | 0.09 CTCAE grade | Standard Deviation 0.29 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 2 | 0.13 CTCAE grade | Standard Deviation 0.45 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 9 | 0.09 CTCAE grade | Standard Deviation 0.29 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 3 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 12 | 0.13 CTCAE grade | Standard Deviation 0.34 |
| Placebo + Standard of Care Melphalan | Change in Gastritis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
Secondary
Change in Hemorrhoids as Assessed Per CTCAE v5.0
Grade 0 hemorrhoids is defined as no hemorrhoids. Grade 1 hemorrhoids is defined as asymptomatic; clinical or diagnostic observations only; intervention not indicated. Grade 2 hemorrhoids is defined as symptomatic; banding or medical intervention indicated. Grade 3 hemorrhoids is defined as severe symptoms; invasive intervention indicated. There is no grade 4 or 5 hemorrhoids defined in the CTCAE v5.0.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 0 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 2 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 3 | 0.08 CTCAE grade | Standard Deviation 0.39 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 4 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 5 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 6 | 0.15 CTCAE grade | Standard Deviation 0.46 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 7 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 8 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 9 | 0.15 CTCAE grade | Standard Deviation 0.37 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 10 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 11 | 0.15 CTCAE grade | Standard Deviation 0.37 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 12 | 0.16 CTCAE grade | Standard Deviation 0.37 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 13 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 14 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 10 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 6 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 14 | 0.09 CTCAE grade | Standard Deviation 0.29 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 7 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 11 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 8 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 13 | 0.09 CTCAE grade | Standard Deviation 0.29 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 9 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 12 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Hemorrhoids as Assessed Per CTCAE v5.0 | Day 5 | 0.08 CTCAE grade | Standard Deviation 0.28 |
Secondary
Change in Nausea as Assessed Per CTCAE v5.0
Grade 0 nausea is defined as no nausea. Grade 1 nausea is defined as loss of appetite without alteration in eating habits. Grade 2 nausea is defined as oral intake decreased without significant weight loss, dehydration or malnutrition. Grade 3 nausea is defined as inadequate oral caloric or fluid intake; tube feeding, TPN, or hospitalization indicated. There is no grade 4 or 5 nausea defined in the CTCAE v5.0.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -1 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 0 | 0.35 CTCAE grade | Standard Deviation 0.63 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 1 | 0.62 CTCAE grade | Standard Deviation 0.75 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 2 | 1.04 CTCAE grade | Standard Deviation 0.82 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 3 | 1.00 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 4 | 1.00 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 5 | 0.92 CTCAE grade | Standard Deviation 0.63 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 6 | 1.23 CTCAE grade | Standard Deviation 0.82 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 7 | 1.35 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 8 | 1.23 CTCAE grade | Standard Deviation 0.91 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 9 | 1.19 CTCAE grade | Standard Deviation 0.85 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 10 | 1.08 CTCAE grade | Standard Deviation 0.84 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 11 | 0.65 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 12 | 0.62 CTCAE grade | Standard Deviation 0.8 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 13 | 0.65 CTCAE grade | Standard Deviation 0.85 |
| Uproleselan + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 14 | 0.20 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 10 | 1.42 CTCAE grade | Standard Deviation 1.02 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 6 | 1.33 CTCAE grade | Standard Deviation 0.76 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -3 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 14 | 0.59 CTCAE grade | Standard Deviation 0.96 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -2 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day -1 | 0.21 CTCAE grade | Standard Deviation 0.51 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 7 | 1.42 CTCAE grade | Standard Deviation 0.88 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 0 | 0.42 CTCAE grade | Standard Deviation 0.72 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 11 | 1.29 CTCAE grade | Standard Deviation 0.95 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 1 | 0.63 CTCAE grade | Standard Deviation 0.82 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 8 | 1.21 CTCAE grade | Standard Deviation 0.93 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 2 | 1.13 CTCAE grade | Standard Deviation 0.95 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 13 | 0.83 CTCAE grade | Standard Deviation 0.98 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 3 | 0.92 CTCAE grade | Standard Deviation 0.88 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 9 | 1.38 CTCAE grade | Standard Deviation 1.01 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 4 | 1.17 CTCAE grade | Standard Deviation 0.82 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 12 | 1.00 CTCAE grade | Standard Deviation 0.98 |
| Placebo + Standard of Care Melphalan | Change in Nausea as Assessed Per CTCAE v5.0 | Day 5 | 1.29 CTCAE grade | Standard Deviation 0.86 |
Secondary
Change in Nutritional Status as Assessed by Change in Standing Weight
Patient standing weight in kilograms was taken at specific time points to assess any changes in nutritional status.
Time frame: Day -3, Day 8, and date of discharge or Day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day -3 | 93.26 kilograms | Standard Deviation 23.22 |
| Uproleselan + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day 8 | 92.81 kilograms | Standard Deviation 22.43 |
| Uproleselan + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day 14 or day of discharge, whichever is sooner | 93.34 kilograms | Standard Deviation 24.11 |
| Placebo + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day -3 | 89.48 kilograms | Standard Deviation 22.37 |
| Placebo + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day 8 | 87.24 kilograms | Standard Deviation 20.67 |
| Placebo + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Change in Standing Weight | Day 14 or day of discharge, whichever is sooner | 89.44 kilograms | Standard Deviation 20.58 |
Secondary
Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days
Time frame: Before conditioning and at day +14 or date of discharge (whichever is sooner) (up to be 18 days)
Population: One subject dropped out of Placebo + Standard of Care Melphalan arm due to adverse events and was not included in the analysis for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days | 0.19 Days | Standard Deviation 0.98 |
| Placebo + Standard of Care Melphalan | Change in Nutritional Status as Assessed by Total Parenteral Nutrition (TPN) Days | 0.00 Days | Standard Deviation 0 |
Secondary
Change in Oral Mucositis as Assessed Per CTCAE v5.0
Grade 0 oral mucositis is defined as no presence of mucositis. Grade 1 oral mucositis is defined as asymptomatic or mild symptoms; intervention not indicated. Grade 2 oral mucositis is defined as moderate pain or ulcer that does not interfere with oral intake; modified diet indicated. Grade 3 oral mucositis is defined as severe pain; interfering with oral intake. Grade 4 oral mucositis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 oral mucositis is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 3 | 0.16 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 2 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 4 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 5 | 0.12 CTCAE grade | Standard Deviation 0.33 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 6 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 7 | 0.31 CTCAE grade | Standard Deviation 0.62 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 8 | 0.23 CTCAE grade | Standard Deviation 0.51 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 9 | 0.38 CTCAE grade | Standard Deviation 0.64 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 10 | 0.35 CTCAE grade | Standard Deviation 0.56 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 11 | 0.27 CTCAE grade | Standard Deviation 0.53 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 12 | 0.15 CTCAE grade | Standard Deviation 0.46 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 13 | 0.15 CTCAE grade | Standard Deviation 0.54 |
| Uproleselan + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 14 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 10 | 0.33 CTCAE grade | Standard Deviation 0.7 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 6 | 0.17 CTCAE grade | Standard Deviation 0.48 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -3 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 14 | 0.23 CTCAE grade | Standard Deviation 0.53 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -2 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 7 | 0.42 CTCAE grade | Standard Deviation 0.88 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 11 | 0.38 CTCAE grade | Standard Deviation 0.65 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 0 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 8 | 0.33 CTCAE grade | Standard Deviation 0.7 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 1 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 13 | 0.22 CTCAE grade | Standard Deviation 0.52 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 2 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 9 | 0.38 CTCAE grade | Standard Deviation 0.71 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 4 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 12 | 0.21 CTCAE grade | Standard Deviation 0.51 |
| Placebo + Standard of Care Melphalan | Change in Oral Mucositis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
Secondary
Change in Proctitis as Assessed Per CTCAE v5.0
Grade 0 proctitis is defined as no proctitis. Grade 1 proctitis is defined as rectal discomfort, intervention not indicated. Grade 2 proctitis is defined as symptomatic (e.g., rectal discomfort, passing blood or mucus); medical intervention indicated; limiting instrumental ADL. Grade 3 proctitis is defined as severe symptoms; fecal urgency or stool incontinence; limiting self-care ADL. Grade 4 proctitis is defined as life-threatening consequences; urgent intervention indicated. Grade 5 proctitis is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 2 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 4 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 5 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 6 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 7 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 8 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 9 | 0.08 CTCAE grade | Standard Deviation 0.27 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 10 | 0.08 CTCAE grade | Standard Deviation 0.39 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 11 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 12 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 13 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 14 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 10 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 6 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 14 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day -1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 7 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 0 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 11 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 1 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 8 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 13 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 9 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 4 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 12 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Proctitis as Assessed Per CTCAE v5.0 | Day 5 | 0.04 CTCAE grade | Standard Deviation 0.2 |
Secondary
Change in Vomiting as Assessed Per CTCAE v5.0
Grade 0 vomiting is defined as no vomiting. Grade 1 vomiting is defined as intervention not indicated. Grade 2 vomiting is defined as outpatient IV hydration; medical intervention indicated. Grade 3 vomiting is defined as tube feeding, TPN, or hospitalization indicated. Grade 4 vomiting is defined as life-threatening consequences. Grade 5 vomiting is defined as death.
Time frame: From day -3 to date of discharge or day 14 (whichever is sooner) (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEAN) | Dispersion |
|---|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 6 | 0.19 CTCAE grade | Standard Deviation 0.49 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 7 | 0.46 CTCAE grade | Standard Deviation 0.86 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 2 | 0.35 CTCAE grade | Standard Deviation 0.69 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 8 | 0.62 CTCAE grade | Standard Deviation 0.94 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -1 | 0.04 CTCAE grade | Standard Deviation 0.2 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 9 | 0.42 CTCAE grade | Standard Deviation 0.7 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 3 | 0.15 CTCAE grade | Standard Deviation 0.46 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 10 | 0.19 CTCAE grade | Standard Deviation 0.49 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 11 | 0.15 CTCAE grade | Standard Deviation 0.54 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 4 | 0.15 CTCAE grade | Standard Deviation 0.46 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 12 | 0.08 CTCAE grade | Standard Deviation 0.28 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 0 | 0.12 CTCAE grade | Standard Deviation 0.43 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 13 | 0.00 CTCAE grade | Standard Deviation 0 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 5 | 0.19 CTCAE grade | Standard Deviation 0.57 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 14 | 0.12 CTCAE grade | Standard Deviation 0.44 |
| Uproleselan + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 1 | 0.27 CTCAE grade | Standard Deviation 0.6 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 14 | 0.09 CTCAE grade | Standard Deviation 0.43 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -3 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -2 | 0.00 CTCAE grade | Standard Deviation 0 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day -1 | 0.08 CTCAE grade | Standard Deviation 0.41 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 1 | 0.29 CTCAE grade | Standard Deviation 0.62 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 2 | 0.63 CTCAE grade | Standard Deviation 0.82 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 3 | 0.50 CTCAE grade | Standard Deviation 0.83 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 4 | 0.67 CTCAE grade | Standard Deviation 0.92 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 5 | 0.54 CTCAE grade | Standard Deviation 0.78 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 6 | 0.46 CTCAE grade | Standard Deviation 0.83 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 7 | 0.67 CTCAE grade | Standard Deviation 0.92 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 8 | 0.42 CTCAE grade | Standard Deviation 0.78 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 9 | 0.42 CTCAE grade | Standard Deviation 0.78 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 10 | 0.38 CTCAE grade | Standard Deviation 0.77 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 11 | 0.38 CTCAE grade | Standard Deviation 0.77 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 12 | 0.29 CTCAE grade | Standard Deviation 0.62 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 13 | 0.13 CTCAE grade | Standard Deviation 0.46 |
| Placebo + Standard of Care Melphalan | Change in Vomiting as Assessed Per CTCAE v5.0 | Day 0 | 0.29 CTCAE grade | Standard Deviation 0.62 |
Secondary
Duration of Hospital Length of Stay
Time frame: From date of admission for auto-HCT to date of discharge (up to be 18 days)
Population: One subject dropped out of Placebo + Standard of Care Melphalan arm due to adverse events and was not included in the analysis for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Duration of Hospital Length of Stay | 18.42 Days | Standard Deviation 1.98 |
| Placebo + Standard of Care Melphalan | Duration of Hospital Length of Stay | 18.46 Days | Standard Deviation 1.84 |
Secondary
Incidence of Clostridium Difficile Infections
Time frame: Through date of discharge (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Uproleselan + Standard of Care Melphalan | Incidence of Clostridium Difficile Infections | 3 Participants |
| Placebo + Standard of Care Melphalan | Incidence of Clostridium Difficile Infections | 1 Participants |
Secondary
Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available)
Time frame: Through date of discharge (upto be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Uproleselan + Standard of Care Melphalan | Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available) | 8 Participants |
| Placebo + Standard of Care Melphalan | Incidence of Infection Assessed by Rates of Bacteremia (With Organism Reported When Available) | 5 Participants |
Secondary
Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0
* Questions regarding gastrointestinal toxicities * Responses are scored from 1-5 with 1=never and 5=almost constantly
Time frame: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - date of discharge or Day 14 (whichever is sooner) | 4.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - Day 8 | 4.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - date of discharge or Day 14 (whichever is sooner) | 3.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - Day 8 | 4.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - Day 8 | 4.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - date of discharge or Day 14 (whichever is sooner) | 1.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - date of discharge or Day 14 (whichever is sooner) | 4.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - date of discharge or Day 14 (whichever is sooner) | 1.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - Day 8 | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - date of discharge or Day 14 (whichever is sooner) | 3.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - Day 8 | 2.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of nausea - Day 8 | 4.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bloating - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of bowel incontinence - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - Day 8 | 2.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of diarrhea - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of vomiting - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of abdominal pain - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Frequency of heartburn - Day -3 | 1.00 score on a scale |
Secondary
Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0
* Questions regarding gastrointestinal toxicities * Responses are scored from 1-5 with 1=no symptoms to 5=severe symptoms
Time frame: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - Day 8 | 3.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - date of discharge or Day 14 (whichever is sooner) | 3.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - Day 8 | 3.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - Day 8 | 2.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of difficulty swallowing - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - Day 8 | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of vomiting - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of mouth or throat sores - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - Day 8 | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - Day 8 | 4.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of abdominal pain - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of decreased appetite - date of discharge or Day 14 (whichever is sooner) | 3.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of bloating - date of discharge or Day 14 (whichever is sooner) | 1.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - Day 8 | 3.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of heartburn - date of discharge or Day 14 (whichever is sooner) | 1.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Patient Reported Outcomes as Measured by the CTCAE Pro Form v1.0 | Severity of nausea - date of discharge or Day 14 (whichever is sooner) | 2.50 score on a scale |
Secondary
Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0
* Questions regarding gastrointestinal, pain, and psychological symptoms interfering with daily activities * Responses are scored from 1-5 with 1=not at all to 5=very much
Time frame: Day -3, Day +8, date of discharge or Day +14 (whichever is sooner) (up to 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Group | Value (MEDIAN) |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - Day 8 | 3.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - Day 8 | 2.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - Day -3 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - Day 8 | 1.00 score on a scale |
| Uproleselan + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - date of discharge or Day 14 (whichever is sooner) | 2.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - Day 8 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Mouth or throat sore interference - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - Day 8 | 1.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - Day -3 | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Bowel incontinence interference - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - Day 8 | 3.50 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Abdominal pain interference - date of discharge or Day 14 (whichever is sooner) | 1.00 score on a scale |
| Placebo + Standard of Care Melphalan | Median Change in Scores of Quality of Life as Measured by the CTCAE Pro Form v 1.0 | Decreased appetite interference - date of discharge or Day 14 (whichever is sooner) | 3.00 score on a scale |
Secondary
Median Daily Dose of Anti-diarrheal Medications
This is defined as the number of doses of anti-diarrheal medications, such as loperamide or lomotil, that participants took daily.
Time frame: Through date of discharge (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Uproleselan + Standard of Care Melphalan | Median Daily Dose of Anti-diarrheal Medications | 2.20 number of doses in one day |
| Placebo + Standard of Care Melphalan | Median Daily Dose of Anti-diarrheal Medications | 2.00 number of doses in one day |
Secondary
Median Daily Dose of Pain Medications
The median daily dose of pain medications is provided as morphine equivalents.
Time frame: Through date of discharge (up to be 18 days)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Uproleselan + Standard of Care Melphalan | Median Daily Dose of Pain Medications | 13.02 mg morphine equivalents |
| Placebo + Standard of Care Melphalan | Median Daily Dose of Pain Medications | 15.65 mg morphine equivalents |
Secondary
Time to First Antibiotics
Measured by the time in days to the first antibiotic dose for bacteremia.
Time frame: Through date of discharge (up to be day 18)
Population: The number of participants analyzed at each time point may differ from the overall number of participants analyzed because the data was not collected. One patient in the placebo + standard of care melphalan arm was not evaluable for the outcome measure.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Uproleselan + Standard of Care Melphalan | Time to First Antibiotics | 10.00 days |
| Placebo + Standard of Care Melphalan | Time to First Antibiotics | 9.00 days |
Secondary
Time to Neutrophil Engraftment
-Defined as ANC ≥0.5 x 10\^9/L for 3 consecutive days or ≥1.0 x 10\^9/L for 1 day
Time frame: Through date of discharge (up to be 18 days)
Population: One subject dropped out of Placebo + Standard of Care Melphalan arm due to adverse events and was not included in the analysis for this outcome measure.
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Uproleselan + Standard of Care Melphalan | Time to Neutrophil Engraftment | 12.77 Days | Standard Deviation 0.76 |
| Placebo + Standard of Care Melphalan | Time to Neutrophil Engraftment | 12.50 Days | Standard Deviation 0.98 |