Actively Recruiting
Phase 2 Study of Pemigatinib (INCB054828) for Advanced SDH-deficient Gastrointestinal Stromal Tumor (GIST)
Led by Dana-Farber Cancer Institute · Updated on 2026-05-22
24
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
Sponsors
D
Dana-Farber Cancer Institute
Lead Sponsor
G
Gateway for Cancer Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the drug pemigatinib as a treatment option for advanced succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors (GIST) in this Phase 2 clinical trial. The study aims to determine how well pemigatinib works for this condition, assess side effects, and evaluate how well patients tolerate the drug. This trial is open-label and single-arm, meaning all participants will receive the study drug without a comparison group. Participants will take oral pemigatinib tablets once daily for the first 14 days of each 21-day cycle, followed by a 7-day break. Treatment will continue until the disease worsens, unacceptable side effects occur, or other criteria for stopping treatment are met. The study includes screening to confirm eligibility and regular in-clinic visits for treatment and monitoring. During the study, participants will undergo physical exams, routine blood tests, pregnancy tests, ECGs, imaging scans, and tumor sample collection. Researchers will collect data and biological specimens to monitor treatment response and safety. The main outcome measured is the objective radiographic response rate, with secondary outcomes including progression-free survival and treatment-related toxicity. Follow-up begins 30 days after treatment stops, and the study expects to enroll about 24 participants.
CONDITIONS
Brief Title
PH 2 Pemigatinib in SDH-deficient GIST
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Histologically confirmed SDH-deficient GIST with locally advanced or metastatic disease not suitable for surgery
- Measurable disease with at least one lesion measurable by scans or clinical exam
- Radiographically documented disease progression before enrollment
- Age 18 years or older
- ECOG performance status of 0 to 2
- Adequate organ and marrow function including hemoglobin > 8 g/dL, neutrophils ≥ 1,000/mcL, platelets ≥ 100,000/mcL, bilirubin ≤ 1.5 times upper limit of normal, AST/ALT ≤ 3 times upper limit of normal (≤ 5 times if liver metastases present), and GFR ≥ 60 mL/min/1.73 m2
- HIV-positive participants on effective non-interacting antiretroviral therapy with undetectable viral load
- Participants with chronic hepatitis B infection must have undetectable viral load on therapy
- Participants with hepatitis C infection must be treated and cured or have undetectable viral load if on treatment
- Treated brain metastases with no evidence of progression on follow-up imaging
- Disease-free from prior invasive cancers for more than 5 years except certain skin and cervical cancers
- Completed prior chemotherapy or radiotherapy at least 2 weeks (6 weeks for certain drugs) before enrollment
- Recovered from prior anti-cancer therapy side effects except hair loss
- Cardiac function class 2B or better by New York Heart Association criteria
- QTc interval below 450 msec
- Ability to comply with study protocol and swallow pills
- Negative pregnancy test for women of childbearing potential within 28 days before first dose and again within 7 days prior to treatment
- Ability and willingness to sign informed consent, including participants with impaired decision-making capacity with a legal representative
You will not qualify if you...
- Receiving any other investigational agents
- History of allergic reactions to drugs similar to pemigatinib
- Use of certain drugs that interact with CYP3A4, P-gp, BCRP, MATE transporters, and strong CYP3A4 inhibitors or inducers
- Uncontrolled illnesses
- Psychiatric or social conditions limiting compliance
- Women or men unwilling or unable to use contraception during and 4 months after treatment
- Previous treatment with an FGFR inhibitor
- History of severe vitamin D deficiency requiring high doses
- High calcium or phosphate levels unresponsive to treatment
- Eye conditions such as central serous retinopathy, retinal vein occlusion, wet age-related macular degeneration, diabetic retinopathy with macular edema, uncontrollable glaucoma, or corneal diseases
- Recent treatment with potent CYP3A4 inhibitors or inducers
- History of soft tissue calcifications linked to abnormal calcium or phosphate levels except for aging or injury-related calcifications
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person) for screening and enrollment
Duration - Repeated 21-day cycles until disease progression or unacceptable toxicity
Participants receive oral pemigatinib in 21-day cycles (2 weeks on, 1 week off) until disease progression or unacceptable toxicity.
In-clinic visits for treatment and assessments each cycle
Duration - Starts 30 days after treatment ends
Participants are followed for safety and disease status beginning 30 days after treatment stops.
Visits for follow-up assessments as scheduled
Trial Site Locations
Total: 1 location
1
Dana Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
S
Suzanne George, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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