Biliary Tract Cancer (BTC)
Conditions
Brief summary
A Phase 2, multicenter, open-label, 2-stage study to assess the safety, tolerability, and efficacy of XERMELO in combination with first-line (1L) therapy (cisplatin \[cis\] plus gemcitabine \[gem\])
Detailed description
A Phase 2, multicenter, open-label, 2-stage study to assess the safety, tolerability, and efficacy of XERMELO in combination with first-line (1L) therapy cis plus gem in patients with unresectable, locally advanced, recurrent or metastatic biliary tract cancer (intrahepatic or extrahepatic cholangiocarcinoma, gallbladder cancer), who are naïve to tumor-directed therapy in the locally advanced or metastatic setting, and for which treatment with 1L therapy (defined as a combination of cis plus gem) is planned.
Interventions
XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) three times a day plus first line therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) three times a day plus first line therapy for the duration of the study
Sponsors
TerSera Therapeutics LLC
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Male or female adults, ≥18 years of age. Patients of childbearing potential must agree to use an adequate method of contraception during the study and for 30 days after the last dose of XERMELO * Histopathologically or cytologically-confirmed, unresectable, locally advanced, recurrent, or metastatic biliary tract cancer (BTC) * Naïve to tumor-directed therapy in locally advanced, unresectable, or metastatic setting * Measurable disease * Eastern Cooperative Oncology Group (ECOG) performance status 0-1 * Plans to initiate treatment with 1L therapy (cisplatin plus gemcitabine) * Ability to provide written informed consent prior to participation in any study-related procedure
Exclusion criteria
* Prior exposure to XERMELO, telotristat ethyl, telotristat etiprate, LX1032, or LX1606 * Primary tumor site in the ampulla of Vater * Treatment with photodynamic therapy for localized disease or to relieve biliary obstruction in the presence of metastatic disease within the past 30 days * Hematology laboratory values of: a. Absolute neutrophil count (ANC) ≤1,500 cells/mm\^3; or b. Platelets ≤100,000 cells/mm\^3; or c. Hemoglobin (Hgb) ≤9 g/dL; or d. White blood count (WBC) ≤3,000 cells/mm\^3 * Hepatic laboratory values of aspartate aminotransferase (AST) or alanine aminotransferase (ALT): a. \>5 x upper limit of normal (ULN) if patient has documented history of hepatic metastases; or b. \>2.5 x ULN if no liver metastases are present * Serum albumin \<2.8 g/dL * Total bilirubin \>1.5 x ULN or \>1.5 mg/dL * Prothrombin time (PT) or international normalized ratio (INR) \>1.5 x ULN * Serum creatinine or serum urea \>1.5 x ULN * Estimated glomerular filtration rate (eGFR) \<50 mL/min * Positive pregnancy test, pregnant, or breastfeeding * Any other clinically significant laboratory abnormality that would compromise patient safety or the outcome of the study * Any clinically significant and/or uncontrolled cardiac-related abnormality that would compromise patient safety or the outcome of the study * Myocardial infarction within the past 6 months * Active bleeding diathesis * Life expectancy ≤3 months * Current complaints of persistent constipation or history of chronic constipation, bowel obstruction or fecaloma within the past 6 months * Receiving chronic treatment with corticosteroids ≥5 mg of prednisone per day (or equivalent) or other immunosuppressive agent(s) * History and/or uncontrolled hepatitis B surface antigen (HBsAg), hepatitis C antibody (HCV Ab), or human immunodeficiency virus (HIV)-1 or HIV-2 * History of substance or alcohol abuse within the past 2 years * History of galactose intolerance, deficiency of Lapp lactase, or glucose-galactose malabsorption * History of malignancy or active treatment for malignancy within 5 years * Receipt of live, attenuated vaccine or close contact with someone who has received a live, attenuated vaccine within the past 1 month * Receipt of any investigational agent or study treatment (ie, any treatment or therapy not approved by the FDA for the treatment of BTC) within the past 30 days * Receipt of any protein or antibody-based therapeutic agents within the past 3 months * Treatment with any tumor-directed therapy within the past 6 months with curative intent * Existence of any surgical or medical condition that, in the judgment of the Investigator, might compromise patient safety or the outcome of the study * Presence of any clinically significant findings (relative to the patient population) during review of medical history or upon physical exam that, in the Investigator's or Medical Monitor's opinion, would compromise patient safety or the outcome of the study * Evidence of brain metastases * Unable or unwilling to communicate or cooperate with the Investigator for any reason * Employee of Sponsor or clinical site, or relative of any member of a clinical site's staff
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | Month 6 | Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions, or similar definition as accurate and appropriate. |
| Project Overall Survival Rate at Month 6 | 6 Months | Overall Survival (OS) was defined as the time from the frist dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring- date of First dose +1). Use Kaplan Meier method to project survival rate at month 6. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Median Progression Free Survival | Month 12 | Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12. |
| Disease Control Rate (DCR), Central Radiologist's Assessment | Month 6 | Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR.. |
| Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment | Month 6 | Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation. (CR) + partial response (PR) at Months 6 |
| Overall (Objective) Response Rate, Central Radiologist's Assessment | End of Study as defined up to 24 months | Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation. |
| Summary of Duration of Progression Free Survival, Local Radiologist's Assessment | up to 7 months | Summary of Duration of Median Progression Free Survival, Local Radiologist's Assessment. Patient progression was defined from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 7 months. |
| Progression Free Survival, Local Radiologist's Assessment | Month 12 | Summary of Median Progression Free Survival, Local Radiologist's Assessment. Defined as the time from first dose of study treatment until the first date of either disease progression or death due to any cause. Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12. |
| Overall (Objective) Response Rate, Local Read | 6 Months | Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 6. |
| Overall (Objective) Response Rate, Local Reader's Assessment | 12 Months | Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 12. |
| Overall Survival (OS) | First dose of study treatment until the date of death due to any cause, whichever came first, a median of approximately 17.67 months | Overall Survival (OS) was defined as the time from first dose of study treatment until the date of death due to any cause. |
| Disease Control Rate (DCR), Local Reviewer | Month 12 as defined by 1 year | Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR. |
| Disease Control Rate End of Study, Local Reviewer | End of Study as defined up to 24 months | Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR. |
| Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA) | Month 6 | Mean change from Baseline to Month 6 plasma level 5-hydroxyindoleacetic acid (5-HIAA) |
| Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9) | Month 6 | Mean change from Baseline to month 6 in plasma carbohydrate antigen 19-9 (CA 19-9) |
| Change From Baseline in Plasma Carbohydrate Antigen 19-9 (CA 19-9) | End of Study as defined up to 24 months | Mean change from Baseline to End of Study in plasma carbohydrate antigen 19-9 (CA 19-9) |
| Weight Change From Baseline | Month 6 | Mean change in weight at Month 6 from baseline measurement |
| Change From Baseline in Serum Albumin | Month 6 | Mean change from Baseline to Month 6 serum albumin levels |
| Overall (Objective) Response Rate (ORR), Local Reader's Assessment | End of Study as defined up to 24 months | Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation. |
| Project Overall Survival Rate at Month 12 | 12 Months | Overall Survival (OS) was defined as the time from the first dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring - date of first dose +1). Use Kaplan Meier method to project survival rate at month 12. |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg Xermelo 250mg plus 1L therapy for a week, then Xermelo 500mg plus 1L therapy for the duration of the study
telotristat ethyl: XERMELO (telotristat ethyl) tablets administered as 250 mg (1 x 250-mg tablet) tid plus 1L therapy for 7 days, then XERMELO (telotristat ethyl) tablets administered as 500 mg (2 x 250-mg tablets) tid plus 1L therapy for the duration of the study | 53 |
| Total | 53 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Study did not meet its primary endpoint. | 53 |
Baseline characteristics
| Characteristic | Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 31 Participants |
| Age, Categorical Between 18 and 65 years | 22 Participants |
| Age, Continuous | 63.4 Years STANDARD_DEVIATION 10.96 |
| Baseline Plasma 5-Hydroxyindoleacetic Acid (5-HIAA) Levels </= to ULN </= ULN | 31 Participants |
| Baseline Plasma 5-Hydroxyindoleacetic Acid (5-HIAA) Levels </= to ULN >ULN | 22 Participants |
| Body Mass Index (BMI) | 28.85 kg/m2 STANDARD_DEVIATION 6.343 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 2 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 50 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Height | 66.4 inches STANDARD_DEVIATION 3.37 |
| Race (NIH/OMB) American Indian or Alaska Native | 0 Participants |
| Race (NIH/OMB) Asian | 2 Participants |
| Race (NIH/OMB) Black or African American | 4 Participants |
| Race (NIH/OMB) More than one race | 0 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 1 Participants |
| Race (NIH/OMB) White | 46 Participants |
| Region of Enrollment United States | 53 participants |
| Serum Albumin Abnormal | 4 Participants |
| Serum Albumin Normal | 48 Participants |
| Serum Albumin Not Available | 1 Participants |
| Serum CA19-9 Abnormal | 32 Participants |
| Serum CA19-9 Normal | 19 Participants |
| Serum CA19-9 Not Available | 2 Participants |
| Serum CEA Abnormal | 26 Participants |
| Serum CEA Normal | 25 Participants |
| Serum CEA Not Available | 2 Participants |
| Sex: Female, Male Female | 33 Participants |
| Sex: Female, Male Male | 20 Participants |
| Weight | 81.9 kilogram STANDARD_DEVIATION 17.4 |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | 3 / 53 |
| other Total, other adverse events | 53 / 53 |
| serious Total, serious adverse events | 24 / 53 |
Outcome results
Primary
Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment
Progression is defined using Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0), as a 20% increase in the sum of the longest diameter of target lesions, or a measurable increase in a non-target lesion, or the appearance of new lesions, or similar definition as accurate and appropriate.
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Group | Category | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | Safety Population | Responders, Central Reviewer | 12 Participants |
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | Safety Population | Non-Responders, Central Reviewer | 41 Participants |
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | Per Protocol Population | Responders, Central Reviewer | 12 Participants |
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | Per Protocol Population | Non-Responders, Central Reviewer | 30 Participants |
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | By Treatment Cycle Population | Responders, Central Reviewer | 12 Participants |
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Number of Participants With Progression-free Survival (PFS) as Evaluated by Central Radiologist's Assessment | By Treatment Cycle Population | Non-Responders, Central Reviewer | 21 Participants |
Primary
Project Overall Survival Rate at Month 6
Overall Survival (OS) was defined as the time from the frist dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring- date of First dose +1). Use Kaplan Meier method to project survival rate at month 6.
Time frame: 6 Months
Population: Safety Population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Project Overall Survival Rate at Month 6 | 0.87 Proportion of participants |
Secondary
Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9)
Mean change from Baseline to month 6 in plasma carbohydrate antigen 19-9 (CA 19-9)
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9) | -229.82 U/mL | Standard Deviation 2484.902 |
Secondary
Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9)
Mean change from Baseline to Month 12 in plasma carbohydrate antigen 19-9 (CA 19-9)
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Carbohydrate Antigen 19-9 (CA 19-9) | -18.04 U/mL | Standard Deviation 38.665 |
Secondary
Change From Baseline in Plasma Carbohydrate Antigen 19-9 (CA 19-9)
Mean change from Baseline to End of Study in plasma carbohydrate antigen 19-9 (CA 19-9)
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Plasma Carbohydrate Antigen 19-9 (CA 19-9) | -153.98 Units per milliliter | Standard Deviation 456.21 |
Secondary
Change From Baseline in Serum Albumin
Mean change from Baseline to End of Study serum albumin levels
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Serum Albumin | -1.2 g/L | Standard Deviation 4.99 |
Secondary
Change From Baseline in Serum Albumin
Mean change from Baseline to Month 6 serum albumin levels
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Serum Albumin | -0.2 g/L | Standard Deviation 4.59 |
Secondary
Change From Baseline in Serum Albumin
Mean change from Baseline to Month 12 serum albumin levels
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Change From Baseline in Serum Albumin | -2.2 g/L | Standard Deviation 2.86 |
Secondary
Disease Control Rate (DCR), Central Radiologist's Assessment
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Time frame: End of Study up to 24 months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate (DCR), Central Radiologist's Assessment | 35 Participants |
Secondary
Disease Control Rate (DCR), Central Radiologist's Assessment
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate (DCR), Central Radiologist's Assessment | 33 Participants |
Secondary
Disease Control Rate (DCR), Central Radiologist's Assessment
Per Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.0) for target lesions and assessed by MRI: Complete Response (CR), Disappearance of all target lesions; Partial Response (PR), \>=30% decrease in the sum of the longest diameter of target lesions; Overall Response (OR) = CR + PR..
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate (DCR), Central Radiologist's Assessment | 33 Participants |
Secondary
Disease Control Rate (DCR), Local Reviewer
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Time frame: Month 12 as defined by 1 year
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate (DCR), Local Reviewer | 40 Participants |
Secondary
Disease Control Rate (DCR), Local Reviewer
Disease control rate (DCR), Local Reviewer, 6 Months
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate (DCR), Local Reviewer | 40 Participants |
Secondary
Disease Control Rate End of Study, Local Reviewer
Disease control rate (DCR) was defined as the proportion of patients with best overall response of SD longer than 6 weeks from first dosing, confirmed PR, or confirmed CR.
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Disease Control Rate End of Study, Local Reviewer | 40 Participants |
Secondary
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
Mean change from Baseline to Month 6 plasma level 5-hydroxyindoleacetic acid (5-HIAA)
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA) | -1.735 micrograms/Liter | Standard Deviation 8.6933 |
Secondary
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
Mean change from Baseline to Month 12 in plasma level 5-hydroxyindoleacetic Acid (5-HIAA)
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA) | -5.608 micrograms/Liter | Standard Deviation 6.5192 |
Secondary
Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA)
Mean change from Baseline to End of Study in plasma 5-hydroxyindoleacetic acid (5-HIAA)
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Mean Change From Baseline in Plasma 5-hydroxyindoleacetic Acid (5-HIAA) | 4.154 micrograms/Liter | Standard Deviation 5.7003 |
Secondary
Median Progression Free Survival
Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12.
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Median Progression Free Survival | 6.233 Months |
Secondary
Overall (Objective) Response Rate, Central Radiologist's Assessment
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation.
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate, Central Radiologist's Assessment | 7 Participants |
Secondary
Overall (Objective) Response Rate, Local Read
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 6.
Time frame: 6 Months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate, Local Read | 6 Participants |
Secondary
Overall (Objective) Response Rate, Local Reader's Assessment
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment at Month 12.
Time frame: 12 Months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate, Local Reader's Assessment | 8 Participants |
Secondary
Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation. (CR) + partial response (PR) at Months 6
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment | 7 Participants |
Secondary
Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment
Overall response rate (ORR) was defined as the proportion of patients (Number of Responders) with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until Month 12.
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate (ORR), Central Radiologist's Assessment | 7 Participants |
Secondary
Overall (Objective) Response Rate (ORR), Local Reader's Assessment
Overall response rate (ORR) was defined as the proportion of patients with best overall response of confirmed PR or confirmed CR. The best overall response was the best overall response recorded from the start of study treatment until the EOS considering any requirement for confirmation.
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall (Objective) Response Rate (ORR), Local Reader's Assessment | 8 Participants |
Secondary
Overall Survival (OS)
Overall Survival (OS) was defined as the time from first dose of study treatment until the date of death due to any cause.
Time frame: First dose of study treatment until the date of death due to any cause, whichever came first, a median of approximately 17.67 months
Population: Safety Population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Overall Survival (OS) | 17.667 Months |
Secondary
Progression Free Survival, Local Radiologist's Assessment
Summary of Median Progression Free Survival, Local Radiologist's Assessment, End of Study
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Progression Free Survival, Local Radiologist's Assessment | 7.467 Months |
Secondary
Progression Free Survival, Local Radiologist's Assessment
Summary of Median Progression Free Survival, Local Radiologist's Assessment. Defined as the time from first dose of study treatment until the first date of either disease progression or death due to any cause. Scheduled disease assessment at Cycle 19 Day 1 was used to determine PFS response rate at Month 12.
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Progression Free Survival, Local Radiologist's Assessment | 7.467 Months |
Secondary
Project Overall Survival Rate at Month 12
Overall Survival (OS) was defined as the time from the first dose of study treatment until the date of death due to any cause. Duration of Overall Survival (OS) in days is defined as (Date of event/censoring - date of first dose +1). Use Kaplan Meier method to project survival rate at month 12.
Time frame: 12 Months
Population: Safety population
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Project Overall Survival Rate at Month 12 | 0.60 Proportion of participants |
Secondary
Summary of Duration of Progression Free Survival, Local Radiologist's Assessment
Summary of Duration of Median Progression Free Survival, Local Radiologist's Assessment. Patient progression was defined from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 7 months.
Time frame: up to 7 months
Population: Safety Population
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Summary of Duration of Progression Free Survival, Local Radiologist's Assessment | 7.000 Months |
Secondary
Weight Change From Baseline
Mean change in weight at Month 12 from baseline measurement
Time frame: Month 12
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Weight Change From Baseline | 1.69 kg | Standard Deviation 7.473 |
Secondary
Weight Change From Baseline
Mean change in weight from baseline to End of Study
Time frame: End of Study as defined up to 24 months
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Weight Change From Baseline | -3.28 kg | Standard Deviation 8.92 |
Secondary
Weight Change From Baseline
Mean change in weight at Month 6 from baseline measurement
Time frame: Month 6
Population: Safety Population
| Arm | Measure | Value (MEAN) | Dispersion |
|---|---|---|---|
| Xermelo 250mg Plus 1L Therapy for a Week, Then Xermelo 500mg | Weight Change From Baseline | -0.69 kg | Standard Deviation 9.759 |