PEMIGIST: Phase 2 Study of Pemigatinib (INCB054828) in the Treatment of Patients With Advanced SDH-deficient Gastrointestinal Stromal Tumor (GIST)

PEMIGIST: Phase 2 Study of Pemigatinib (INCB054828) in the Treatment of Patients With Advanced SDH-deficient Gastrointestinal Stromal Tumor (GIST)

Gastrointestinal Stromal Tumor

Recruiting

2

Start Date 06/01/2026

Age of Trial (yrs) -.2

First-line

SDH-directed

Block FGFR Pathway

Specifically GIST and only GIST

25-666

Dana-Farber Cancer Institute

Suzanne George, MD 617-632-5204 SGEORGE2@PARTNERS.ORG

Oral

Brief Summary
The purpose of this study is to determine how effective the drug pemigatinib is as a treatment option for advanced succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors(GIST). This study will also assess the side effects associated with pemigatinib and evaluate its tolerability.

The name of the study drug involved in this study is:

• Pemigatinib (INCB054828)

Detailed Description
This is a single-arm, open-label Phase 2 trial evaluating the clinical efficacy of pemigatinib (INCB054828) in patients with advanced succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors.

The U.S. Food and Drug Administration (FDA) has not approved pemigatinib for advanced succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors but it has been approved for other uses.

The research study procedures include screening for eligibility, study treatment in-clinic visits, physical exam, routine blood tests, pregnancy test, Electrocardiogram (ECG),Imaging Scans, Tumor sample, Data and biological specimen collection.

Eligible participants will receive oral pemigatinib (INCB054828) continuously in 21-day cycles until disease progression or unacceptable toxicity. Follow up will begin 30 days after treatment stops.

It is expected that about 24 people will take part in this research study.

Incyte, a biopharmaceutical company, is supporting this research study by providing the study drug.

Official Title
PEMIGIST: Phase 2 Study of Pemigatinib (INCB054828) in the Treatment of Patients With Advanced SDH-deficient Gastrointestinal Stromal Tumor (GIST)

Eligibility Criteria (Partial - See NCT number link for full criteria)
Description
Inclusion Criteria:

Participant must have histologically confirmed SDH-deficient GIST. Participants must have locally advanced or metastatic disease that is not amenable to surgery.
Participants must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter to be recorded for non-nodal lesions and short axis for nodal lesions) as ≥20 mm (≥2 cm) by chest x-ray or as ≥10 mm (≥1 cm) with CT scan, MRI, or calipers by clinical exam. See Section 12 (Measurement of Effect) for the evaluation of measurable disease.
Participants must have radiographically documented progressive disease prior to study enrollment as per investigator assessment.