Pheochromocytoma/Paraganglioma study: Belzutifan
Official title 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)
ClinicalTrials.gov ID: NCT04924075
What this study is testing
What is Belzutifan?
This is an investigational oral pill being studied as a potential treatment for advanced pheochromocytoma/paraganglioma. It is designed to block a specific protein called HIF-2α that helps tumors grow in low-oxygen environments.
Also referred to as MK-6482, Welireg.
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 study wants to see how well belzutifan works and how safe it is. The main goal is to measure the objective response rate using RECIST 1.1 criteria.
- Who it's for
- Adults who have advanced pheochromocytoma/paraganglioma, pancreatic neuroendocrine tumor, VHL disease‑associated tumors, advanced wild‑type GIST, or other solid tumors with HIF‑2α genetic changes.
- What's involved
- Participants will receive belzutifan alone. They will have regular clinic visits, blood tests, and imaging scans to monitor the tumor response and any side effects.
- Phase 2: a mid-size study of how well it works
- This is a blinded study.
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older
You may be able to join if
- Male and female participants at least 12 years of age (at least 18 years of age for Cohort B1)
- Diagnosis of one of the following: Advanced/metastatic pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumors (pNET), von...
- Have a diagnosis of VHL disease as determined by a germline test locally and/or clinical diagnosis
- Must be ≥18 years of age
- Has a life expectancy of at least 3 months The main
You likely can't join if
- Unable to swallow orally administered medication or has a disorder that might affect the absorption of belzutifan
- History of a second malignancy, unless potentially curative treatment has been completed with no evidence of malignancy for 2 years
- Any of the following: A pulse oximeter reading \<92% at rest, or requires intermittent supplemental oxygen, or requires chronic supplemental oxygen
- Clinically significant cardiac disease, including unstable angina, acute myocardial infarction, or arterial bypass (CABG) or Percutaneous...
- Received prior treatment (except somatostatin analogs) with chemotherapy, targeted therapy, biologics, or other investigational therapy within the...
The study team makes the final eligibility decision.
Where it is enrolling
- Los Angeles, California, United States
- Chicago, Illinois, United States
- Warrenville, Illinois, United States
- Iowa City, Iowa, United States
- Baltimore, Maryland, United States
- Bethesda, Maryland, United States
- Boston, Massachusetts, United States
- Ann Arbor, Michigan, United States
- Nashville, Tennessee, United States
- Houston, Texas, United States
- Randwick, New South Wales, Australia
- Parkville, Victoria, Australia
- Calgary, Alberta, Canada
- Toronto, Ontario, Canada
- Santiago, Region M. de Santiago, Chile
- Beijing, Beijing Municipality, China
- Strasbourg, Alsace, France
- Lyon, Auvergne-Rhône-Alpes, France
- Le Kremlin-Bicêtre, Paris, France
- Paris, France
+ 27 more site(s).
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, but 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, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
This study is enrolling now at Los Angeles, California, United States; Chicago, Illinois, United States; Warrenville, Illinois, United States; Iowa City, Iowa, United States; Baltimore, Maryland, United States; Bethesda, Maryland, United States and 41 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.