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New treatment option for SDH Gene Mutation
Official title PH 2 Pemigatinib in SDH-deficient GIST
ClinicalTrials.gov ID: NCT07434843
What this study is testing
What is Pemigatinib?
Pemigatinib is an investigational medicine, given as a pill taken by mouth, being studied as a potential treatment for sdh gene mutation.
Also referred to as INCB054828.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 and older
You may be able to join if
- Participant must have histologically confirmed SDH-deficient GIST. Participants must have locally advanced or metastatic disease that is not amenable...
- Participants must have measurable disease, defined as at least one lesion that can be accurately measured in at least one dimension (longest diameter...
- Participants must have radiographically documented progressive disease prior to study enrollment as per investigator assessment.
- Age ≥18 years
- ECOG performance status ≤2
You likely can't join if
- 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...
- Participants with uncontrolled intercurrent illness.
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- Women of childbearing potential unwilling or unable to perform contraception from screening until 4 months after completion of pemigatinib...
See the full eligibility criteria
Who can join
- 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...
- Participants must have radiographically documented progressive disease prior to study enrollment as per investigator assessment.
- Age ≥18 years
- ECOG performance status ≤2
- Participants must have adequate organ and marrow function as defined below:
- Hemoglobin \> 8 g/dL. Patients with a hemoglobin of 7.5-8.0 g/dL may be eligible if the value is chronic, there is no clinical evidence of active bleeding, and eligibility is confirmed upon review and approval of the...
- Absolute neutrophil count ≥1,000/mcL
- platelets ≥100,000/mcL
- total bilirubin ≤1.5 × institutional upper limit of normal (ULN)
- AST(SGOT)/ALT(SGPT) ≤3.0 × institutional ULN (unless liver metastases are present in which case it must be ≤ 5 × ULN)
- glomerular filtration rate (GFR) ≥60 mL/min/1.73 m2 (using the CDK-EPI formula see Appendix B)
- Human immunodeficiency virus (HIV)-infected participants on effective non-CYP3A4 interacting (see Section 3.2.3) anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial.
- For participants with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated.
- Participants with a history of hepatitis C virus (HCV) infection must have been treated and cured. For participants with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV...
- Participants with treated brain metastases are eligible if follow-up brain imaging after central nervous system (CNS)-directed therapy shows no evidence of progression.
- Participants must be disease-free of prior invasive malignancies for \> 5 years with the exception of curatively-treated basal cell or squamous cell carcinoma of the skin or carcinoma in situ of the cervix. NOTE: If...
- Participants should have completed prior treatment for their cancer: chemotherapy or radiotherapy must have been completed for greater than 2 weeks (6 weeks for nitrosoureas or mitomycin C) prior to entering the study.
- Participants should have recovered from adverse events due to prior anti-cancer therapy (i.e.,have residual toxicities \> Grade 1) with the exception of alopecia.
- Participants with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association...
- Participants must have a QTc interval length of below 450 msec. QTc will be calculated via the Fridericia's formula.
- Participant is willing to comply with the protocol for the duration of the study including undergoing treatment and scheduled visits and examinations including follow up.
- Participant must be able to swallow and maintain pills.
- Women of childbearing potential must have a negative urine or serum pregnancy test within 28 days of initial dose of pemigatinib (INCB054828), and again within 7 days prior to treatment on day 1. If screening occurs...
- Ability to understand and the willingness to sign a written informed consent document. Participants with impaired decision-making capacity (IDMC) who have a legally authorized representative (LAR) and/or family member...
What rules you out
- 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...
- Participants with uncontrolled intercurrent illness.
- Participants with psychiatric illness/social situations that would limit compliance with study requirements.
- Women of childbearing potential unwilling or unable to perform contraception from screening until 4 months after completion of pemigatinib administration
- Men unwilling or unable to perform contraception from screening to until 4 months after completion of pemigatinib administration.
- Participants previously treated with a FGFR inhibitor.
- History of hypovitaminosis D requiring supraphysiologic doses (e.g., 50,000 UI/weekly or above). Vitamin D supplements are allowed.
- Participants with calcium and phosphate levels exceeding the upper limit of normal (ULN) despite medical intervention within 2 weeks prior to the first dose of pemigatinib.
- Current evidence of corneal or retinal abnormalities that may increase eye toxicity, including but not limited to:
- Currently suffering from central serous retinopathy (CSR) or retinal vein occlusion (RVO), or with relevant history
- Active wet age-related macular degeneration (wAMD)
- Diabetic retinopathy with macular edema
- Uncontrollable glaucoma
- Keratopathy, such as keratitis, keratoconjunctivitis, keratopathy, corneal abrasion, inflammation or ulceration
- Treatment with any potent CYP3A4 inhibitors or inducers or moderate CYP3A4 inducers within 14 days or 5 half-lives (whichever is longer) before the first dose of study drug.
- Participants with a history of soft tissue calcifications on imaging that are associated with documented abnormalities in calcium or phosphate levels except for soft tissue calcification due to aging, previous injury or...
The study team makes the final eligibility decision.
Where it's taking place
- Boston, Massachusetts, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Boston, Massachusetts, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.