PH 2 Pemigatinib in SDH-deficient GIST
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorDana-Farber Cancer Institute
About this trial
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)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
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.
Age ≥18 years
Disqualifiers
Participants who are receiving any other investigational agents.
History of allergic reactions attributed to compounds of similar chemical or biologic composition to pemigatinib (INCB054828).
Concomitant administration with sensitive substrates/narrow therapeutic index drugs of CYP3A4, P-gp BCRP, MATE1, and MATE2K, and strong inhibitors and inducers of CYP3A4 should be avoided. Use caution with strong inhibitors and inducers of P-gp. Because the lists of these agents are constantly changing, it is important to regularly consult a frequently-updated medical reference. As part of the enrollment/informed consent procedures, the participant will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the participant is considering a new over-the-counter medicine or herbal product. (See Appendix C Participant Drug Information Handout and Wallet Card).
Participants with uncontrolled intercurrent illness.
Trial design
Design model
Single group
Treatments tested in this trial
Pemigatinib
DrugA selective Fibroblast Growth Factor Receptors inhibitor * Tablet taken orally
Treatment groups
24 Participants
are divided into 1 treatment groupGroup A: PEMIGATINIBExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Objective radiographic response rate
The objective response rate is defined as the rate of participants with a complete response or partial response, calculated using RECIST 1.1.
Time frame
From first dose of pemigatinib until the date of objective response per RECIST 1.1 through study completion, an average of 1 yearSecondary outcomes
1
Progression-free survival (PFS)
Progression-Free Survival (PFS) based on the Kaplan-Meier method is defined as the time from randomization (or registration) to the earlier of progression or death due to any cause. Participants alive without disease progression are censored at the earliest date of last disease evaluation or off treatment date.
Time frame
Participants alive without disease progression are censored at the earliest date of last disease evaluation or off treatment date] average of 2yrs.2
Incidence of Grade 3 or Higher Treatment-Related Toxicity
Number and proportion of participants experiencing at least one Grade 3, 4, or 5 treatment related adverse event, as defined and graded by NCI CTCAE v5.0. Events will be summarized overall and by type of event.
Time frame
From first dose of pemigatinib through 30 days after last dose of pemigatinib (approximately up to 36 months total on study time).Sponsors and contacts
Click on the lead sponsor to view all of their trials.
Dana-Farber Cancer Institute
Lead sponsor
Gateway for Cancer Research
Collaborator
Incyte Corporation
Collaborator
Source ClinicalTrials.gov