Chronic Gastrointestinal Graft vs Host Disease
Conditions
Keywords
BDP, orBec, GVHD, beclomethasone dipropionate, bone marrow transplant, hematopoietic cell transplant, HCT, stem cell transplant, marrow transplant, SCT, beclomethasone 17,21-dipropionate
Brief summary
Use of an oral topically-active glucocorticoid with limited side effects may control the gastrointestinal inflammatory process of GVHD and minimize glucocorticoid exposure.
Interventions
DRUGorBec
DRUGPlacebo
Sponsors
Soligenix
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Receipt of allogeneic hematopoietic cell transplant \>100 days prior to consent * Documented cGVHD as defined by the NIH consensus criteria in at least one organ system other than the GI tract (for example, cGVHD of the oral cavity would qualify as a criterion) * Endoscopic findings consistent with GI GVHD * Must be able to swallow tablets * Must be able to read and understand informed consent * Adequate birth control methods for the duration of the study
Exclusion criteria
* \>500 mL/day of diarrhea on any 1 day within 3 days prior to the first dose of study drug * GI infection * Multi-organ failure or other condition that, in the opinion of the investigator, would compromise the patient's ability to complete the study. * HIV seropositivity * Pregnant or nursing female * Use of any investigational drug to treat chronic GVHD within 28 days of the first dose of study drug * Evidence of recurrent or progressing malignant disorder that was the indication for HCT
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms | 16 weeks | Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Placebo Control
Placebo | 1 |
| orBec Investigational drug
orBec | 1 |
| Total | 2 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Part 2 | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | Placebo | orBec | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 1 Participants | 1 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants | 0 Participants | 1 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 | 0 Participants | 0 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 | 1 Participants | 1 Participants | 2 Participants |
| Region of Enrollment United States | 1 participants | 1 participants | 2 participants |
| Sex: Female, Male Female | 1 Participants | 0 Participants | 1 Participants |
| Sex: Female, Male Male | 0 Participants | 1 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | — / — | — / — |
| other Total, other adverse events | 0 / 1 | 0 / 1 |
| serious Total, serious adverse events | 1 / 1 | 0 / 1 |
Outcome results
Primary
Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms
Patients who achieved a Complete Response (CR) of their GI GVHD Symptoms during the Part 1, 16 week treatment period and who remained a CR at the end of the 16 week treatment period were to be eligible to continue into Part 2 of the study. All others were to discontinue. GI GVHD was assessed using a composite score based on the symptoms of satiety, nausea/vomiting, and anorexia. Each symptom was scored on a scale of 0-3, such that the minimum score = 0 and the maximum score = 9. At entry, all subjects must have a score of ≥ 3. A CR will be defined as a composite score of 0.
Time frame: 16 weeks
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Placebo | Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms | 1 Participants |
| orBec | Gastrointestinal (GI) Graft-vs-Host Disease (GVHD) Symptoms | 0 Participants |