Advanced or Metastatic Gastric or Gastroesophageal Cancer, Advanced or Metastatic Solid Tumor, Myeloid or Lymphoid Neoplasms (MLN)
Conditions
Keywords
Futibatinib, Gastric cancer, Gastro-esophageal junction cancer, Solid tumor, Myeloid neoplasm, Lymphoid neoplasm, FGFR, Amplification, Rearrangement, TAS-120
Brief summary
The purpose of this study is to evaluate the efficacy and safety of futibatinib in patients with FGFR aberrations in 3 distinct cohorts. Patients will be enrolled into one of 3 cohorts: patients with advanced, metastatic or locally-advanced solid tumors harboring FGFR1-4 rearrangements (excluding primary brain tumors and intrahepatic cholangiocarcinoma \[iCCA\]); patients with gastric or gastro-esophageal junction (GEJ) cancer harboring FGFR2 amplification; and patients with myeloid or lymphoid neoplasms with FGFR1 rearrangements.
Detailed description
Study TAS-120-202 is an open-label, multinational, 3-arm Phase 2 study evaluating the efficacy, safety, tolerability, PK, and pharmacodynamics of futibatinib in patients with FGFR aberrations. Eligible patients will be assigned to 1 of 3 treatment cohorts based on diagnosis and FGFR gene aberration status. Patients will receive futibatinib at an oral dose of 20 mg once a day on a continuous 28-day cycle. The study will enroll approximately: * Cohort A: 60 patients with locally advanced, advanced, or metastatic solid tumor harboring FGFR rearrangements other than primary brain tumor or iCCA; * Cohort B: 35 patients with locally-advanced, advanced, or metastatic gastric cancer or gastro-esophageal junction (GEJ) with FGFR2 amplification; * Cohort C: 20 patients with myeloid or lymphoid neoplasms (MLN) with FGFR1 rearrangements Treatment in all cohorts will continue until disease progression, unacceptable toxicity, or any other of the criteria for treatment discontinuation is met. For patients who discontinue treatment for reasons other than disease progression, tumor assessments should be continued until radiologic disease progression is documented or until initiation of subsequent new anticancer therapy (whichever occurs first). Patients will be followed for survival every 12 weeks (±2 weeks) until survival events (deaths) have been reported for 75% of enrolled patients or the study is terminated early by the Sponsor. Additional cohorts may be added in the future in case of new emerging efficacy data.
Interventions
DRUGFutibatinib
Futibatinib tablets were dosed orally every day on a continuous 28-day cycle
Sponsors
Taiho Oncology, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
The study consists of independent 3 cohorts (Cohorts A, B and C) and there is no difference in the treatment regimen between the cohorts
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Eastern Cooperative Oncology Group (ECOG) performance status 0 or 1 2. Known FGFR aberration status and tumor type that meet all of the criteria for 1 of the following cohorts: a. Cohort A i. Histologically-confirmed, locally-advanced, advanced, or metastatic solid tumors harboring a FGFR1-4 ii. Measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST) Version 1.1 iii. Had disease progression/recurrence after standard treatment for their cancer b. Cohort B i. Histologically-confirmed, locally-advanced, advanced, or metastatic gastric or gastroesophageal junction cancer harboring a FGFR2 amplification. ii. Measurable disease per RECIST 1.1 iii. Received at least 2 prior systemic regimens for advanced/metastatic disease iv. Experienced disease progression/recurrence during or after the most recent prior systemic treatment for advanced/metastatic gastric cancer or GEJ cancer c. Cohort C i. Confirmed myeloid or lymphoid neoplasms as defined by WHO criteria with a FGFR1 rearrangement ii. Not a candidate for hematological stem cell transplant (HSCT) or relapsed after HSCT and donor lymphocyte infusion, and progressed and not a candidate for other therapies
Exclusion criteria
1. History and/or current evidence of any of the following disorders: 1. Non-tumor related alteration of the calcium-phosphorus homeostasis that is considered clinically significant in the opinion of the Investigator 2. Ectopic mineralization/calcification including, but not limited to, soft tissue, kidneys, intestine, or myocardia and lung, considered clinically significant in the opinion of the Investigator 3. Retinal or corneal disorder confirmed by retinal/corneal examination and considered clinically significant in the opinion of the Investigator. 2. Prior treatment with an FGFR inhibitor 3. Brain metastases that are untreated or clinically or radiologically unstable (that is, have been stable for \<1 month)
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B | At the end of every 2 cycles until disease progression (Up to 31 months) | ORR was defined as the percentage of participants experiencing a best overall response of partial response (PR) or complete response (CR) (per Response Evaluation Criteria in Solid Tumors, RECIST version 1.1), based on IRC of radiological images. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place. |
| Complete Response (CR) Rate in Cohort C | At the end of every 2 cycles until disease progression (Up to 31 months) | CR rate was defined as the percentage of participants who achieved a CR (per response criteria for myeloid or lymphoid neoplasm) based on investigator assessment of imaging, peripheral blood, and bone marrow. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| DOR Based on Investigator Assessment in Cohorts A, B and C | At the end of every 2 cycles until disease progression (Up to 31 months) | DOR was defined as the time from the first documentation of response (CR or PR in based on Investigator Assessment) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. DOR was estimated using the Kaplan-Meier method. |
| Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C | At the end of every 2 cycles until disease progression (Up to 31 months) | PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on IRC), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure. |
| PFS Based on Investigator Review in Cohorts A, B and C | At the end of every 2 cycles until disease progression (Up to 31 months) | PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on Investigator Review), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure. |
| Overall Survival (OS) in Cohorts A, B and C | Up to 31 months | OS was defined as the time from the date of first dose to the death date. Participants without a documented death date were censored on the last date they were known to be alive. The OS was presented using a Kaplan-Meier estimate. The 95% CI for median OS was provided using the Kaplan-Meier procedure. |
| Disease Control Rate (DCR) Based on IRC in Cohort A and B | At the end of every 2 cycles until disease progression (Up to 31 months) | DCR was defined as the percentage of participants experiencing a best overall response of stable disease (SD), PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. Percentages were rounded off to the nearest single decimal place. |
| DCR Based on Investigator Review in Cohort A and B | At the end of every 2 cycles until disease progression (Up to 31 months) | DCR was defined as the percentage of participants experiencing a best overall response of SD, PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. Percentages were rounded off to the nearest single decimal place. |
| CR+ Complete Response With Incomplete Hematological Recovery (CRi) Rate in Cohort C | Up to 31 months | CR+CRi rate was defined as the percentage of participants who achieved a CR or CRi. |
| ORR Based on Investigator Assessment in Cohorts A and B | At the end of every 2 cycles until disease progression (Up to 31 months) | ORR was defined as the percentage of participants experiencing a best overall response of PR or CR (per RECIST 1.1), based on investigator assessment. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place. |
| Duration of CR+CRi in Cohort C | Up to 31 months | Duration of CR+CRi was defined as the time from the first documentation of CR or CRi to the first documentation of disease relapse or death due to any cause, whichever occurs first. |
| Complete Cytogenetic Response (CCyR) Rate in Cohort C | Up to 31 months | CCyR rate was defined as the percentage of participants who achieved a CCyR. |
| Partial Cytogenetic Response (PCyR) Rate in Cohort C | Up to 31 months | PCyR rate was defined as the percentage of participants who achieved a PCyR. |
| Relapse-free Survival (RFS) in Cohort C | Up to 31 months | RFS was defined as the time from first documentation of CR to the date of death (any cause) or disease relapse or death due to any cause, whichever occurs first. |
| Event-free Survival (EFS) in Cohort C | Up to 31 months | EFS was defined as the time from first dose of study therapy to treatment failure, disease relapse after CR, or participant death due to any cause, whichever occurs first. |
| Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C | From the first dose of study drug up to 30 days after the last dose (Up to 31 months) | An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant and does not necessarily have a causal relationship with the study drug. A treatment-emergent AE (TEAE) is defined as an AE that is starting or worsening at the time of or after the first dose of study drug administration and within 30 days after the last dose of study drug, and does not necessarily have a causal relationship to the use of the study drug. |
| Duration of CR in Cohort C | Up to 31 months | Duration of CR was defined as the time from the first documentation of CR to the first documentation of recurrence or death due to any cause, whichever occurs first. |
| Duration of Response (DOR) Based on IRC in Cohorts A, B and C | At the end of every 2 cycles until disease progression (Up to 31 months) | DOR was defined as the time from the first documentation of response (CR or PR in based on IRC) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. DOR was estimated using the Kaplan-Meier method. |
Countries
Belgium, France, Germany, Hong Kong, Italy, Japan, Netherlands, Portugal, Singapore, South Korea, Spain, Sweden, Turkey (Türkiye), United Kingdom, United States
Participant flow
Recruitment details
Participants took part in the study from 05 August 2020 to 11 November 2024.
Pre-assignment details
A total of 115 participants were enrolled in Cohort A and Cohort B to receive futibatinib; no participants were enrolled in Cohort C.
Participants by arm
| Arm | Count |
|---|---|
| Futibatinib (Cohort A) Participants with advanced or metastatic solid tumors harboring FGFR1-4 rearrangements received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 841 days. | 87 |
| Futibatinib (Cohort B) Participants with advanced or metastatic solid gastric or GEJ cancer harboring FGFR2 amplification received futibatinib 20 mg, oral tablets, once a day on a continuous 28-day cycle up to a maximum of 297 days. | 28 |
| Total | 115 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Overall Study | Death | 54 | 24 | 0 |
| Overall Study | Study Terminated by Sponsor | 25 | 3 | 0 |
| Overall Study | Withdrawal of consent | 8 | 1 | 0 |
Baseline characteristics
| Characteristic | Futibatinib (Cohort A) | Futibatinib (Cohort B) | Total |
|---|---|---|---|
| Age, Continuous | 60.0 years STANDARD_DEVIATION 12.47 | 55.6 years STANDARD_DEVIATION 13.77 | 58.9 years STANDARD_DEVIATION 12.87 |
| Race/Ethnicity, Customized Ethnicity Hispanic or Latino | 3 Participants | 1 Participants | 4 Participants |
| Race/Ethnicity, Customized Ethnicity Not Hispanic or Latino | 62 Participants | 26 Participants | 88 Participants |
| Race/Ethnicity, Customized Ethnicity Unknown | 22 Participants | 1 Participants | 23 Participants |
| Race/Ethnicity, Customized Race Asian | 36 Participants | 24 Participants | 60 Participants |
| Race/Ethnicity, Customized Race Black or African American | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Caucasian/White | 29 Participants | 3 Participants | 32 Participants |
| Race/Ethnicity, Customized Race Other | 1 Participants | 0 Participants | 1 Participants |
| Race/Ethnicity, Customized Race Unknown | 20 Participants | 1 Participants | 21 Participants |
| Sex: Female, Male Female | 47 Participants | 16 Participants | 63 Participants |
| Sex: Female, Male Male | 40 Participants | 12 Participants | 52 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 54 / 87 | 24 / 28 |
| other Total, other adverse events | 86 / 87 | 27 / 28 |
| serious Total, serious adverse events | 23 / 87 | 6 / 28 |
Outcome results
Primary
Complete Response (CR) Rate in Cohort C
CR rate was defined as the percentage of participants who achieved a CR (per response criteria for myeloid or lymphoid neoplasm) based on investigator assessment of imaging, peripheral blood, and bone marrow. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: No participants were enrolled in Cohort C, hence no data was collected.
Primary
Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B
ORR was defined as the percentage of participants experiencing a best overall response of partial response (PR) or complete response (CR) (per Response Evaluation Criteria in Solid Tumors, RECIST version 1.1), based on IRC of radiological images. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 millimeters (mm). PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Futibatinib (Cohort A) | Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B | 6.9 percentage of participants |
| Futibatinib (Cohort B) | Objective Response Rate (ORR) Based on Independent Central Review (IRC) in Cohorts A and B | 17.9 percentage of participants |
Secondary
Complete Cytogenetic Response (CCyR) Rate in Cohort C
CCyR rate was defined as the percentage of participants who achieved a CCyR.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
CR+ Complete Response With Incomplete Hematological Recovery (CRi) Rate in Cohort C
CR+CRi rate was defined as the percentage of participants who achieved a CR or CRi.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
DCR Based on Investigator Review in Cohort A and B
DCR was defined as the percentage of participants experiencing a best overall response of SD, PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for PD, referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. Percentages were rounded off to the nearest single decimal place.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Futibatinib (Cohort A) | DCR Based on Investigator Review in Cohort A and B | 52.9 percentage of participants |
| Futibatinib (Cohort B) | DCR Based on Investigator Review in Cohort A and B | 64.3 percentage of participants |
Secondary
Disease Control Rate (DCR) Based on IRC in Cohort A and B
DCR was defined as the percentage of participants experiencing a best overall response of stable disease (SD), PR, or CR (per RECIST 1.1), based on IRC. SD was defined as neither sufficient shrinkage to qualify for PR nor sufficient increase to qualify for progressive disease (PD), referencing the smallest sum diameters while on study. PD was defined as at least a 20% increase in the sum of target lesion diameters from the smallest on study (including baseline), with an absolute increase of ≥ 5 mm, or the appearance of new lesions. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. Percentages were rounded off to the nearest single decimal place.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Futibatinib (Cohort A) | Disease Control Rate (DCR) Based on IRC in Cohort A and B | 37.9 percentage of participants |
| Futibatinib (Cohort B) | Disease Control Rate (DCR) Based on IRC in Cohort A and B | 50.0 percentage of participants |
Secondary
DOR Based on Investigator Assessment in Cohorts A, B and C
DOR was defined as the time from the first documentation of response (CR or PR in based on Investigator Assessment) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. DOR was estimated using the Kaplan-Meier method.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All responders population included all participants who received at least 1 dose of study drug and had a response. No participants were enrolled in Cohort C, hence no data was collected. Overall number of participants analyzed is the number of participants with events.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Futibatinib (Cohort A) | DOR Based on Investigator Assessment in Cohorts A, B and C | 5.6 months |
| Futibatinib (Cohort B) | DOR Based on Investigator Assessment in Cohorts A, B and C | 5.6 months |
Secondary
Duration of CR+CRi in Cohort C
Duration of CR+CRi was defined as the time from the first documentation of CR or CRi to the first documentation of disease relapse or death due to any cause, whichever occurs first.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
Duration of CR in Cohort C
Duration of CR was defined as the time from the first documentation of CR to the first documentation of recurrence or death due to any cause, whichever occurs first.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
Duration of Response (DOR) Based on IRC in Cohorts A, B and C
DOR was defined as the time from the first documentation of response (CR or PR in based on IRC) to the first documentation of objective tumor progression or death due to any cause, whichever occurs first. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. DOR was estimated using the Kaplan-Meier method.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All responders population included all participants who received at least 1 dose of study drug and had a response. No participants were enrolled in Cohort C, hence no data was collected. Overall number of participants analyzed is the number of participants with events.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Futibatinib (Cohort A) | Duration of Response (DOR) Based on IRC in Cohorts A, B and C | 5.6 months |
| Futibatinib (Cohort B) | Duration of Response (DOR) Based on IRC in Cohorts A, B and C | 3.9 months |
Secondary
Event-free Survival (EFS) in Cohort C
EFS was defined as the time from first dose of study therapy to treatment failure, disease relapse after CR, or participant death due to any cause, whichever occurs first.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C
An adverse event (AE) is defined as any untoward medical occurrence in a clinical study participant and does not necessarily have a causal relationship with the study drug. A treatment-emergent AE (TEAE) is defined as an AE that is starting or worsening at the time of or after the first dose of study drug administration and within 30 days after the last dose of study drug, and does not necessarily have a causal relationship to the use of the study drug.
Time frame: From the first dose of study drug up to 30 days after the last dose (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug. No participants were enrolled in Cohort C, hence no data was collected.
| Arm | Measure | Value (COUNT_OF_PARTICIPANTS) |
|---|---|---|
| Futibatinib (Cohort A) | Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C | 87 Participants |
| Futibatinib (Cohort B) | Number of Participants With Treatment Emergent Adverse Events (TEAEs) - Cohort A, B and C | 27 Participants |
Secondary
ORR Based on Investigator Assessment in Cohorts A and B
ORR was defined as the percentage of participants experiencing a best overall response of PR or CR (per RECIST 1.1), based on investigator assessment. CR was defined as disappearance of all target lesions. Any pathological lymph node must have reduction in short axis to \<10 mm. PR was defined as at least a 30% decrease in the sum of diameters of the target lesions, taking as a reference the baseline sum diameters. ORR was calculated based on the best overall response recorded from the start of treatment until progressive disease or start of subsequent new anticancer treatment. Percentages were rounded off to the nearest single decimal place.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug.
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| Futibatinib (Cohort A) | ORR Based on Investigator Assessment in Cohorts A and B | 9.2 percentage of participants |
| Futibatinib (Cohort B) | ORR Based on Investigator Assessment in Cohorts A and B | 10.7 percentage of participants |
Secondary
Overall Survival (OS) in Cohorts A, B and C
OS was defined as the time from the date of first dose to the death date. Participants without a documented death date were censored on the last date they were known to be alive. The OS was presented using a Kaplan-Meier estimate. The 95% CI for median OS was provided using the Kaplan-Meier procedure.
Time frame: Up to 31 months
Population: All treated population included all participants who received at least 1 dose of study drug. Overall number of participants analyzed are the number of participants with reported death. No participants were enrolled in Cohort C, hence no data was collected.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Futibatinib (Cohort A) | Overall Survival (OS) in Cohorts A, B and C | 11.1 months |
| Futibatinib (Cohort B) | Overall Survival (OS) in Cohorts A, B and C | 5.9 months |
Secondary
Partial Cytogenetic Response (PCyR) Rate in Cohort C
PCyR rate was defined as the percentage of participants who achieved a PCyR.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.
Secondary
PFS Based on Investigator Review in Cohorts A, B and C
PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on Investigator Review), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug. Overall number of participants analyzed are the number of participants with reported disease progression or death. No participants were enrolled in Cohort C, hence no data was collected.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Futibatinib (Cohort A) | PFS Based on Investigator Review in Cohorts A, B and C | 3.3 months |
| Futibatinib (Cohort B) | PFS Based on Investigator Review in Cohorts A, B and C | 3.3 months |
Secondary
Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C
PFS was defined as the time from first dose of the study therapy to the date of death (any cause) or disease progression (based on IRC), whichever occurs first. The PFS was analyzed using a Kaplan-Meier method, with PFS time being censored on the date of the last disease assessment. The 95% CI for median PFS was provided using the Kaplan-Meier procedure.
Time frame: At the end of every 2 cycles until disease progression (Up to 31 months)
Population: All treated population included all participants who received at least 1 dose of study drug. Overall number of participants analyzed are the number of participants with reported disease progression or death. No participants were enrolled in Cohort C, hence no data was collected.
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Futibatinib (Cohort A) | Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C | 1.9 months |
| Futibatinib (Cohort B) | Progression- Free Survival (PFS) Based on IRC in Cohorts A, B and C | 2.9 months |
Secondary
Relapse-free Survival (RFS) in Cohort C
RFS was defined as the time from first documentation of CR to the date of death (any cause) or disease relapse or death due to any cause, whichever occurs first.
Time frame: Up to 31 months
Population: No participants were enrolled in Cohort C, hence no data was collected.