Belzutifan for Advanced Pheochromocytoma or Paraganglioma.
Camilo Jimenez, Mikkel Andreassen, Alice Durand...
https://pubmed.ncbi.nlm.nih.gov/41124218/Actively Recruiting
Led by Merck Sharp & Dohme LLC · Updated on 2026-08-03
355
Participants Needed
84
Research Sites
N/A
Total Duration
Researchers are evaluating the safety and effectiveness of belzutifan as a single therapy for people with advanced pheochromocytomaparaganglioma, pancreatic neuroendocrine tumors, von Hippel-Lindau disease-related tumors, advanced wild-type gastrointestinal stromal tumors, or other advanced solid tumors with genetic changes related to hypoxia inducible factor-2 alpha. This phase 2 study focuses on measuring the objective response rate using standardized criteria reviewed by an independent central panel. Participants will receive oral belzutifan at a dose of 120 mg once daily. Treatment continues until the disease progresses or the participant chooses to stop. The study does not include a comparison group and follows a single-group design. The study period can last up to about 5.5 years to assess treatment effects and safety over time. During the study, participants will have regular assessments including imaging scans reviewed centrally to evaluate tumor response. Researchers will track outcomes such as duration of response, time to response, disease control rate, progression-free survival, overall survival, and any adverse events experienced. Safety and treatment tolerability will be monitored throughout the study. Participants are expected to remain in the study for several years to gather long-term data on belzutifans effects.
CONDITIONS
Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)
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Merck Sharp & Dohme LLC
Lead Sponsor
This table compares participation in this trial with other options available for this condition. The information is drawn from published research and the trial’s study details and is provided for educational purposes only.
| Factors | Belzutifan (investigational) | Prior standard systemic care (contextual) |
|---|---|---|
| What the approach is | An oral HIF‑2α inhibitor taken daily (120 mg) until disease progression or discontinuation as studied in the trial | Prior to belzutifan approval, treatment options included systemic therapies such as radiolabeled iobenguane I‑131 when available, chemotherapy or targeted agents used off‑label for advanced PPGL |
| Role in trial | Single‑group investigational treatment arm evaluated for efficacy and safety | Not part of this trial; represents care delivered previously outside trial |
| Primary use or purpose | Treated advanced PPGL and related tumors by inhibiting HIF‑2α pathway | Used systemically to control tumor growth or symptoms in advanced PPGL when surgery was not feasible |
| Administration or performance | Orally once daily at 120 mg until progression or unacceptable toxicity | Varied by agent—for example, iobenguane I‑131 delivered intravenously as radiotherapy; chemotherapies infused according to regimen—specific schedules varied |
| Evidence status | Phase 2 data show ~26 % objective response rate, durable responses, manageable safety, and blood‑pressure reductions | Evidence limited; systemic approaches had variable effectiveness and lacked consistent response data |
| Important risks | Treatment‑related adverse events were common; notable risks included anemia, fatigue, elevated liver enzymes, dyspnea; serious events in a minority | Depends on prior therapy used (e.g., hematologic toxicity for chemotherapy; radiation exposure for iobenguane I‑131); safety profiles varied by treatment |
| Important limitations | Single‑arm design without randomized control; long‑term outcomes and broader population efficacy remain under study | Lacked approved, disease‑specific systemic therapies; available treatments were extrapolated, inconsistent, and not tailored to HIF‑2α driven disease |
Disclaimer: This comparison is created with the help of artificial intelligence(AI) and may contain errors or missing information. It is not medical advice and does not mean one option is better or safer than another. Clinical trials are still being studied, and joining a trial is your choice. Please consult your healthcare professional before making any decisions.
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Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to approximately 5.5 years
Participants take oral belzutifan 120 mg once daily until their disease progresses or they choose to stop.
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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Camilo Jimenez, Mikkel Andreassen, Alice Durand...
https://pubmed.ncbi.nlm.nih.gov/41124218/Merck Sharp & Dohme LLC
Lead Sponsor