Advanced or Metastatic Solid Tumors, FGFR Mutations, FGFR Translocations
Conditions
Keywords
Advanced solid tumor, metastatic solid tumor, FGFR inhibitor, FGFR mutation, FGFR translocation
Brief summary
The purpose of this study is to evaluate the efficacy and safety of pemigatinib in participants with previously treated locally advanced/metastatic or surgically unresectable solid tumors harboring activating FGFR mutations or translocations.
Interventions
DRUGPemigatinib
Pemigatinib administered orally once daily.
Sponsors
Incyte Corporation
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants will be assigned to 1 of 2 cohorts (Cohort A: FGFR1-3 in-frame fusions or FGFR2 intron 17 rearrangements; Cohort B: Known/predicted activating point mutations in FGFR1-3 \[excluding kinase domain\]) but 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
* Histologically or cytologically confirmed solid tumor malignancy that is advanced or metastatic (Stage IIIB or IV) or is surgically unresectable. * Radiographically measurable disease (per RECIST v1.1 or RANO for primary brain tumors). * Documentation of an FGFR1-3 gene mutation or translocation. * Objective disease progression after at least 1 prior therapy. * Not eligible or able to participate in any other Incyte-sponsored clinical trial.
Exclusion criteria
* Advanced/metastatic bladder cancer or advanced/metastatic cholangiocarcinoma. * Prior receipt of a selective FGFR inhibitor. * Current evidence of clinically significant corneal or retinal disorder. * History of calcium and phosphate hemostasis disorder or systemic mineral imbalance with ectopic calcification of soft tissues.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Objective Response Rate (ORR) | Up to approximately 6 months | Defined as the proportion of participants in each cohort who achieve a complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Progression-free Survival (PFS) | Up to approximately 6 months | Defined as the time from first dose until progressive disease (per RECIST v1.1 or Response Assessment in Neuro-Oncology \[RANO\]) or death (whichever is first) in each cohort. |
| Duration of Response (DOR) | Up to approximately 6 months | Defined as the time from the date of first assessment of CR or PR until the date of the first progressive disease (per RECIST v1.1 or RANO) or death (whichever is first) in each cohort. |
| Disease Control Rate (DCR) | Up to approximately 6 months | Defined as the proportion of participants who achieved best overall response of CR, PR, or stable disease per RECIST v1.1 or RANO. |
| Overall Survival (OS) | Up to approximately 6 months | Defined as the time from first dose of study drug to death of any cause in each cohort. |
| Number of Treatment-related Adverse Events | Up to approximately 6 months | Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment. |
Countries
United States
Participant flow
Recruitment details
Approximately 50 participants were planned for enrollment. However, only 1 participant was enrolled in the study (assigned to Cohort A) and data was analyzed for safety. The study was closed for lack of enrollment in 2021.
Pre-assignment details
A total of 1 participant (assigned to Cohort A) was enrolled in the study, received at least 1 dose of pemigatinib, and was included in the safety population. The participant withdrew consent after Cycle 2+. As a result, there was no efficacy evaluable population.
Participants by arm
| Arm | Count |
|---|---|
| Cohort A: Participants With FGFR1-3 In-frame Fusions or FGFR2 Intron 17 Rearrangements Participants will receive 13.5mg QD Pemigatinib | 1 |
| Cohort B: Participants With Known/Predicted Activating Point Mutations in FGFGR1-3 Participants will receive 13.5mg QD Pemigatinib | 0 |
| Total | 1 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 |
|---|---|---|---|
| Overall Study | Withdrawal by Subject | 1 | 0 |
Baseline characteristics
| Characteristic | Cohort A: Participants With FGFR1-3 In-frame Fusions or FGFR2 Intron 17 Rearrangements | Cohort B: Participants With Known/Predicted Activating Point Mutations in FGFGR1-3 | Total |
|---|---|---|---|
| Age, Categorical <=18 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical >=65 years | 0 Participants | 0 Participants | 0 Participants |
| Age, Categorical Between 18 and 65 years | 1 Participants | 0 Participants | 1 Participants |
| Race (NIH/OMB) American Indian or Alaska Native | NA Participants | — | NA Participants |
| Race (NIH/OMB) Asian | NA Participants | — | NA Participants |
| Race (NIH/OMB) Black or African American | NA Participants | — | NA Participants |
| Race (NIH/OMB) More than one race | NA Participants | — | NA Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | NA Participants | — | NA Participants |
| Race (NIH/OMB) Unknown or Not Reported | NA Participants | — | NA Participants |
| Race (NIH/OMB) White | NA Participants | — | NA Participants |
| Sex: Female, Male Female | NA Participants | — | NA Participants |
| Sex: Female, Male Male | NA Participants | — | NA Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk |
|---|---|---|
| deaths Total, all-cause mortality | 0 / 1 | 0 / 0 |
| other Total, other adverse events | 1 / 1 | 0 / 0 |
| serious Total, serious adverse events | 0 / 1 | 0 / 0 |
Outcome results
Primary
Objective Response Rate (ORR)
Defined as the proportion of participants in each cohort who achieve a complete response (CR) or partial response (PR) based on Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.
Secondary
Disease Control Rate (DCR)
Defined as the proportion of participants who achieved best overall response of CR, PR, or stable disease per RECIST v1.1 or RANO.
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.
Secondary
Duration of Response (DOR)
Defined as the time from the date of first assessment of CR or PR until the date of the first progressive disease (per RECIST v1.1 or RANO) or death (whichever is first) in each cohort.
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.
Secondary
Number of Treatment-related Adverse Events
Adverse events reported for the first time or worsening of a pre-existing event after first dose of study drug/treatment.
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.
Secondary
Overall Survival (OS)
Defined as the time from first dose of study drug to death of any cause in each cohort.
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.
Secondary
Progression-free Survival (PFS)
Defined as the time from first dose until progressive disease (per RECIST v1.1 or Response Assessment in Neuro-Oncology \[RANO\]) or death (whichever is first) in each cohort.
Time frame: Up to approximately 6 months
Population: Due to low participant enrollment (n=1), efficacy analyses were not conducted.