Trial Outcomes & Findings for Futibatinib and Pembrolizumab for Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer (NCT NCT04828486)
NCT ID: NCT04828486
Last Updated: 2025-11-18
Results Overview
PFS is defined as the length of time from study registration until disease progression. For the purposes of this study, 6 months is defined as 27 weeks. Percent of patients alive and progression free will be reported, estimated using the method of Kaplan-Meier. Progression will be evaluated in this study using Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
14 participants
Primary outcome timeframe
6 months
Results posted on
2025-11-18
Participant Flow
Participant milestones
| Measure |
Treatment (Futibatinib, Pembrolizumab)
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.\>
\> Futibatinib: Given PO\>
\> Pembrolizumab: Given IV\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Overall Study
STARTED
|
13
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
13
|
Reasons for withdrawal
| Measure |
Treatment (Futibatinib, Pembrolizumab)
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.\>
\> Futibatinib: Given PO\>
\> Pembrolizumab: Given IV\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Overall Study
Adverse Event
|
2
|
|
Overall Study
Death
|
1
|
|
Overall Study
Disease Progression
|
8
|
|
Overall Study
Other Complicating Disease
|
1
|
|
Overall Study
Withdrawal by Subject
|
1
|
Baseline Characteristics
Futibatinib and Pembrolizumab for Treatment of Advanced or Metastatic FGF19 Positive BCLC Stage A, B, or C Liver Cancer
Baseline characteristics by cohort
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.\>
\> Futibatinib: Given PO\>
\> Pembrolizumab: Given IV\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Age, Continuous
|
71.3 years
STANDARD_DEVIATION 7.61 • n=202 Participants
|
|
Sex: Female, Male
Female
|
6 Participants
n=202 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
13 Participants
n=202 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
White
|
13 Participants
n=202 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=202 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=202 Participants
|
|
Region of Enrollment
United States
|
13 participants
n=202 Participants
|
|
ECOG Performance Status
0
|
5 Participants
n=202 Participants
|
|
ECOG Performance Status
1
|
8 Participants
n=202 Participants
|
PRIMARY outcome
Timeframe: 6 monthsPFS is defined as the length of time from study registration until disease progression. For the purposes of this study, 6 months is defined as 27 weeks. Percent of patients alive and progression free will be reported, estimated using the method of Kaplan-Meier. Progression will be evaluated in this study using Response Evaluation Criteria in Solid Tumors (RECIST) guidelines (version 1.1).
Outcome measures
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
\>
\> Futibatinib: Given PO
\>
\> Pembrolizumab: Given IV
\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Progression-free Survival (PFS)
|
23.1 percentage of participants
Interval 8.6 to 62.3
|
SECONDARY outcome
Timeframe: 3 yearsORR defined as the number of evaluable patients achieving a response \[partial response or complete response per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1\] during treatment. Complete Response (CR): All of the following must be true: a. Disappearance of all target lesions. b. Each target lymph node must have reduction in short axis to \<1.0 cm. c. Normalization of tumor biomarkers. Partial Response (PR): At least a 30% decrease in PBSD (sum of the longest diameter for all target lesions plus the sum of the short axis of all the target lymph nodes at current evaluation) taking as reference the BSD (baseline sum of dimensions).
Outcome measures
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
\>
\> Futibatinib: Given PO
\>
\> Pembrolizumab: Given IV
\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Overall Response Rate (ORR)
|
0 Participants
|
SECONDARY outcome
Timeframe: 4 yearsOS is defined as the time from registration to death due to any cause. Median OS time and 95% CI will be reported.
Outcome measures
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
\>
\> Futibatinib: Given PO
\>
\> Pembrolizumab: Given IV
\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Overall Survival (OS)
|
55.7 weeks
Interval 47.3 to
Upper CI is not estimable due to a low number of events.
|
SECONDARY outcome
Timeframe: 4 yearsAdverse events will be evaluated per Common Terminology Criteria for Adverse Events (CTCAE) version 5.0 for each patient. Number of participants experiencing one or more grade 3+ adverse events will be reported.
Outcome measures
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
\>
\> Futibatinib: Given PO
\>
\> Pembrolizumab: Given IV
\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Incidence of Adverse Events
|
9 Participants
|
SECONDARY outcome
Timeframe: 3 yearsPopulation: Only patients that completed a baseline assessment and at least one post-baseline assessment were included in analysis
As measured by the global health domain of the European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30), version 3. Change in score between baseline and first re-staging will be calculated for each individual. Twenty-eight (28) questions are answered on a scale of 1-4 where 1=not at all, 2=a little, 3=quite a bit, and 4= very much. The final two questions are answered on a scale of 1-7 where 1= very poor and 7=excellent. Raw scores were then scored according to EORTC guidelines (Fayers PM, Aaronson NK, Bjordal K, Groenvold M, Curran D, Bottomley A on behalf of the EORTC Quality of Life Group. EORTC QLQ-C30 Scoring Manual (3rd edition). Brussels: EORTC, 2001.), so that scores are 0-100, with a higher score indicating a better quality of life.
Outcome measures
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=7 Participants
Patients receive futibatinib PO QD on days 1-21 for cycles 1-9, and days 1-42 for subsequent cycles and pembrolizumab IV over 30 minutes on day 1. Cycles repeat every 21 days for cycles 1-9 and every 42 days for subsequent cycles for up to 2 years in the absence of disease progression or unacceptable toxicity.
\>
\> Futibatinib: Given PO
\>
\> Pembrolizumab: Given IV
\>
\> Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Change in Quality of Life (QOL)
|
-10.68 score on a scale
Interval -27.78 to -2.39
|
Adverse Events
Treatment (Futibatinib, Pembrolizumab)
Serious events: 5 serious events
Other events: 12 other events
Deaths: 8 deaths
Serious adverse events
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 participants at risk
Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumor hemorrhage
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Renal and urinary disorders
Acute kidney injury
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Renal and urinary disorders
Renal calculi
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Renal and urinary disorders
Urinary retention
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnea
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Respiratory, thoracic and mediastinal disorders
Pleural effusion
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Gastrointestinal disorders
Nausea
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Gastrointestinal disorders
Vomiting
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
General disorders
Edema limbs
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Infections and infestations
Bacteremia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Investigations
Creatinine increased
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Blood and lymphatic system disorders
Anemia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Cardiac disorders
Myocarditis
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Gastrointestinal disorders
Abdominal pain
|
7.7%
1/13 • Number of events 1 • 4 years
|
Other adverse events
| Measure |
Treatment (Futibatinib, Pembrolizumab)
n=13 participants at risk
Quality-of-Life Assessment: Ancillary studies
|
|---|---|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Nervous system disorders
Dizziness
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Nervous system disorders
Memory impairment
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Nervous system disorders
Peripheral sensory neuropathy
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Psychiatric disorders
Confusion
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Renal and urinary disorders
Chronic kidney disease
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Skin and subcutaneous tissue disorders
Nail changes
|
23.1%
3/13 • Number of events 3 • 4 years
|
|
Skin and subcutaneous tissue disorders
Nail loss
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Skin and subcutaneous tissue disorders
Palmar-plantar erythrodysesthesia syndrm
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Skin and subcutaneous tissue disorders
Rash maculo-papular
|
38.5%
5/13 • Number of events 8 • 4 years
|
|
Vascular disorders
Hypertension
|
15.4%
2/13 • Number of events 8 • 4 years
|
|
Vascular disorders
Hypotension
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Musculoskeletal and connective tissue disorders
Joint range of motion decreased
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Musculoskeletal and connective tissue disorders
Muscle weakness lower limb
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Blood and lymphatic system disorders
Anemia
|
7.7%
1/13 • Number of events 7 • 4 years
|
|
Cardiac disorders
Sinus bradycardia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Eye disorders
Blurred vision
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Eye disorders
Dry eye
|
38.5%
5/13 • Number of events 6 • 4 years
|
|
Eye disorders
Eye pain
|
7.7%
1/13 • Number of events 2 • 4 years
|
|
Gastrointestinal disorders
Abdominal pain
|
23.1%
3/13 • Number of events 3 • 4 years
|
|
Gastrointestinal disorders
Constipation
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Gastrointestinal disorders
Diarrhea
|
53.8%
7/13 • Number of events 17 • 4 years
|
|
Gastrointestinal disorders
Dry mouth
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Gastrointestinal disorders
Gastrointestinal disorders - Oth spec
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Gastrointestinal disorders
Mucositis oral
|
38.5%
5/13 • Number of events 5 • 4 years
|
|
Gastrointestinal disorders
Nausea
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
General disorders
Edema limbs
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
General disorders
Fatigue
|
46.2%
6/13 • Number of events 10 • 4 years
|
|
Investigations
Alanine aminotransferase increased
|
38.5%
5/13 • Number of events 9 • 4 years
|
|
Investigations
Alkaline phosphatase increased
|
15.4%
2/13 • Number of events 4 • 4 years
|
|
Investigations
Aspartate aminotransferase increased
|
53.8%
7/13 • Number of events 15 • 4 years
|
|
Investigations
Blood bilirubin increased
|
15.4%
2/13 • Number of events 4 • 4 years
|
|
Investigations
Creatinine increased
|
15.4%
2/13 • Number of events 3 • 4 years
|
|
Investigations
Lymphocyte count decreased
|
23.1%
3/13 • Number of events 4 • 4 years
|
|
Investigations
Platelet count decreased
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Metabolism and nutrition disorders
Anorexia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Metabolism and nutrition disorders
Blood bicarbonate decreased
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Metabolism and nutrition disorders
Hypercalcemia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Metabolism and nutrition disorders
Hyperphosphatemia
|
76.9%
10/13 • Number of events 22 • 4 years
|
|
Metabolism and nutrition disorders
Hypokalemia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Metabolism and nutrition disorders
Hyponatremia
|
30.8%
4/13 • Number of events 4 • 4 years
|
|
Metabolism and nutrition disorders
Hypophosphatemia
|
15.4%
2/13 • Number of events 2 • 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.7%
1/13 • Number of events 1 • 4 years
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
7.7%
1/13 • Number of events 1 • 4 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place