Belzutifan for Von Hippel-Lindau Syndrome
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial explores the effectiveness of belzutifan for individuals with advanced solid tumors or tumors associated with von Hippel-Lindau (VHL) syndrome. Researchers aim to determine how long patients live after starting belzutifan, which blocks a protein that aids tumor growth. The trial is open only to those already enrolled in specific belzutifan studies. Participants must have advanced solid tumors or VHL-related tumors and be actively receiving treatment in a prior belzutifan study. As a Phase 3 trial, this study serves as the final step before potential FDA approval, offering participants an opportunity to contribute to the validation of a promising treatment.
Do I have to stop taking my current medications for the trial?
The trial information does not specify if you need to stop taking your current medications. Since this is an extension trial for those already on belzutifan, you may need to continue any medications related to the parent study.
Is there any evidence suggesting that this trial's treatments are likely to be safe?
A previous study found that belzutifan, a drug for certain tumors, was generally safe for people with von Hippel-Lindau (VHL) disease. Research has shown that while many patients experienced side effects, adjusting the dose often managed these issues. Anemia, a condition characterized by a lack of healthy red blood cells, was the most common side effect.
The FDA has already approved belzutifan for treating tumors related to VHL disease, indicating its safety for these conditions. Long-term studies also support its safety, showing that patients can continue using it with manageable side effects. Overall, evidence suggests that belzutifan is generally well-tolerated in patients with VHL disease.12345Why are researchers excited about this trial's treatments?
Belzutifan is unique because it targets hypoxia-inducible factor-2 alpha (HIF-2α), a protein involved in tumor growth in Von Hippel-Lindau (VHL) syndrome. Unlike current treatments like surgical removal of tumors or VEGF inhibitors that mainly manage symptoms, belzutifan directly interferes with the molecular pathway driving the condition. Researchers are excited about its potential to offer a more targeted and less invasive approach, possibly leading to fewer side effects and improved quality of life for patients with VHL syndrome.
What evidence suggests that this trial's treatments could be effective for VHL-related tumors?
Research has shown that belzutifan effectively treats tumors linked to Von Hippel-Lindau (VHL) disease. In earlier studies, up to 90% of patients experienced significant tumor shrinkage, particularly in kidney cancer. This trial may administer belzutifan as monotherapy or in combination therapy. Belzutifan blocks a protein that aids tumor growth, helping to control and stabilize tumors over time. It has also slowed disease progression with manageable side effects. These findings highlight belzutifan's potential as a promising and non-invasive treatment for individuals with VHL-related tumors.678910
Who Is on the Research Team?
Medical Director
Principal Investigator
Merck Sharp & Dohme LLC
Are You a Good Fit for This Trial?
This trial is for people with advanced solid tumors or VHL-related tumors who are already taking belzutifan as part of another clinical study. Participants must be actively receiving treatment and not have any serious ongoing side effects from the previous study.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants continue the same dose and frequency of belzutifan or non-belzutifan therapy as in the parent study until progressive disease, unacceptable toxicity, withdrawal of consent, death, investigator decision, or study termination
Follow-up
Participants are monitored for overall survival and adverse events
What Are the Treatments Tested in This Trial?
Interventions
- Belzutifan
Trial Overview
The study looks at the long-term safety and effectiveness of belzutifan, a medicine that blocks tumor growth, in people continuing from earlier trials. Other cancer drugs like cabozantinib, everolimus, palbociclib, nivolumab, or lenvatinib may also be involved depending on prior treatments.
How Is the Trial Designed?
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Treatment groups
Experimental Treatment
Active Control
Participants on active treatment assigned to a belzutifan combination therapy in a parent study are transitioned to this extension study. Participants will continue the same dose and frequency of intervention as they were receiving in the parent study at the time of the transition. Treatment will continue until PD, unacceptable toxicity, withdrawal of consent, death, investigator decision, or study termination.
Participants on active treatment assigned to belzutifan monotherapy in a parent study are transitioned to this extension study. Participants will continue the same dose and frequency of intervention as they were receiving in the parent study at the time of the transition. Treatment will continue until progressive disease (PD), unacceptable toxicity, withdrawal of consent, death, investigator decision, or study termination.
Participants on active treatment assigned to a non-belzutifan therapy in a parent study are transitioned to this extension study. Participants will continue the same dose and frequency of intervention as they were receiving in the parent study at the time of the transition. Treatment will continue until PD, unacceptable toxicity, withdrawal of consent, death, investigator decision, or study termination.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Merck Sharp & Dohme LLC
Lead Sponsor
Chirfi Guindo
Merck Sharp & Dohme LLC
Chief Marketing Officer since 2022
Degree in Engineering from Ecole Centrale de Paris, MBA from New York University Stern School of Business
Robert M. Davis
Merck Sharp & Dohme LLC
Chief Executive Officer since 2021
JD from Northwestern University Pritzker School of Law, MBA from Northwestern University Kellogg Graduate School of Management, Bachelor's in Finance from Miami University
Citations
Belzutifan for patients with Von Hippel-Lindau (VHL) disease ...
The findings from this study demonstrate that belzutifan is an effective treatment for managing advanced manifestations of VHL disease.
2.
onclive.com
onclive.com/view/belzutifan-maintains-high-durable-responses-in-vhl-disease-associated-neoplasms-at-5-yearsBelzutifan Maintains High, Durable Responses in VHL ...
After approximately 5 years of follow-up, high ORRs of 70%, 50% and 90% were achieved by patients with VHL disease–associated RCC, CNS ...
Phase 2 study of belzutifan (MK-6482), an oral hypoxia- ...
Belzutifan demonstrates clinical benefit and has a favorable safety profile in patients with VHL disease–associated ccRCC, pNETs, and hemangioblastomas.
Belzutifan Demonstrates Promise in Von Hippel-Lindau ...
Over a median treatment duration of 16.4 months (range, 3.6-26 months), belzutifan effectively controlled tumor growth and stabilized disease.
MK-6482 as a potential treatment for von Hippel-Lindau ... - PMC
In terms of efficacy, 24% of the patients had a partial response, and 56% had stable disease. The clinical activity was observed across IMDC risk categories. At ...
Long-term Safety and Efficacy of Belzutifan in Von Hippel ...
This study aims to report a single-center experience with belzutifan in patients with VHL and to provide clinical data on the safety and ...
Long-term safety of belzutifan in von Hippel-Lindau ...
Following treatment initiation, 8% required additional procedures. Conclusions: These findings provide long-term safety data on belzutifan in ...
The safety profile of belzutifan in renal tumors - Oxford Academic
Belzutifan is a HIF-2ɑ inhibitor approved for the treatment of tumors in von Hippel-Lindau (VHL) syndrome and sporadic metastatic clear cell renal cell ...
The safety profile of belzutifan in renal tumors: real-world ...
Belzutifan is a HIF-2ɑ inhibitor approved for the treatment of tumors in von Hippel-Lindau (VHL) syndrome and sporadic metastatic clear cell ...
10.
renalandurologynews.com
renalandurologynews.com/reports/cancer-belzutifan-durable-response-von-hippel-lindau-treatment-risk/Belzutifan Provides Durable Response in Von Hippel ...
“After 5 years of follow-up, belzutifan continues to demonstrate durable antitumor activity and a manageable safety profile, consistent with ...
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