Belzutifan for Kidney Cancer

Not currently recruiting at 10 trial locations
Age: 18+
Sex: Any
Trial Phase: Phase 2
Sponsor: Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)
No Placebo GroupAll trial participants will receive the active study treatment (no placebo)
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 1 JurisdictionThis treatment is already approved in other countries

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests belzutifan, a medication for treating kidney cancer in people with von Hippel Lindau (VHL) disease. Researchers aim to determine if this treatment can manage renal cell carcinoma (RCC), a type of kidney tumor associated with VHL. The trial is open to individuals diagnosed with VHL disease who have at least one measurable kidney tumor that doesn't require immediate surgery. Participants should not have received previous treatment with belzutifan or similar medications. As a Phase 2 trial, the research measures the treatment's effectiveness in an initial, smaller group of people.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications, but you cannot have had any prior systemic anti-cancer therapy.

Is there any evidence suggesting that belzutifan is likely to be safe for humans?

Research shows that belzutifan is generally well-tolerated in people with tumors, including those related to von Hippel-Lindau (VHL) syndrome. Studies have found that most side effects are mild to moderate, such as fatigue or nausea. These are classified as grade 1 or 2, indicating they are less severe.

The FDA has already approved belzutifan for treating certain kidney tumors, which adds confidence in its safety. This approval indicates thorough testing to ensure its safety.

In summary, current data suggests belzutifan is safe, with mostly mild side effects reported.12345

Why do researchers think this study treatment might be promising?

Belzutifan is unique because it targets kidney cancer by inhibiting hypoxia-inducible factor-2 alpha (HIF-2α), a new approach compared to traditional treatments like tyrosine kinase inhibitors and immune checkpoint inhibitors. This mechanism of action is important because HIF-2α is a key player in cancer cell survival and growth under low oxygen conditions, which are common in tumors. Researchers are excited about belzutifan because its targeted action could lead to more effective treatments with potentially fewer side effects, offering hope for patients who may not respond well to existing therapies.

What evidence suggests that belzutifan might be an effective treatment for kidney cancer?

Research has shown that belzutifan, which participants in this trial will receive, may effectively treat kidney cancer related to von Hippel-Lindau (VHL) disease. Almost half of the patients experienced tumor shrinkage in this type of kidney cancer. The treatment is primarily associated with mild side effects. Additionally, the duration of benefit from the treatment was not determined, suggesting it may have long-lasting effects for many. This evidence supports belzutifan as a promising option for managing VHL-related kidney cancer.46789

Who Is on the Research Team?

MD

Medical Director

Principal Investigator

Merck Sharp & Dohme LLC

Are You a Good Fit for This Trial?

This trial is for individuals with von Hippel Lindau disease who have at least one measurable kidney cancer tumor but don't need immediate surgery. They shouldn't have used belzutifan or similar drugs before, had any cancer treatments like anti-VEGF therapy, require urgent tumor surgery, or have metastatic disease.

Inclusion Criteria

I have a measurable kidney cancer tumor that doesn't need immediate surgery.
I have been diagnosed with von Hippel Lindau disease.

Exclusion Criteria

I have been treated with belzutifan or a similar drug before.
My scans show cancer has spread to other parts of my body.
I have received treatments targeting cancer growth.
See 1 more

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive 120 mg belzutifan orally once daily. Treatment is continuous with radiological evaluations approximately every 12 weeks.

Minimum of 3 years
Radiological evaluations every 12 weeks

Follow-up

Participants are monitored for safety and effectiveness after treatment, with evaluations every 24 weeks or more frequently if clinically indicated.

Up to approximately 4 years

What Are the Treatments Tested in This Trial?

Interventions

  • Belzutifan
Trial Overview The study tests belzutifan as a potential treatment for kidney cancer associated with von Hippel Lindau disease. It aims to understand the effectiveness and safety of this medication in patients diagnosed with VHL-related renal cell carcinoma.
How Is the Trial Designed?
1Treatment groups
Experimental Treatment
Group I: Open Label BelzutifanExperimental Treatment1 Intervention

Belzutifan is already approved in United States for the following indications:

🇺🇸
Approved in United States as Welireg for:

Find a Clinic Near You

Who Is Running the Clinical Trial?

Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)

Lead Sponsor

Trials
8
Recruited
460+

Peloton Therapeutics, Inc.

Lead Sponsor

Trials
8
Recruited
460+

Published Research Related to This Trial

Belzutifan (Welireg™) is an oral medication that inhibits HIF-2α and has been approved in the USA for treating patients with von Hippel-Lindau (VHL) disease who have associated renal cell carcinoma (RCC) and other tumors, marking a significant advancement in targeted cancer therapy.
Clinical studies are ongoing to evaluate the efficacy of belzutifan in treating other solid tumors, including clear cell renal cell carcinoma (ccRCC) and pancreatic neuroendocrine tumors (pNET), indicating its potential for broader applications in oncology.
Belzutifan: First Approval.Deeks, ED.[2022]
The HIF-2α inhibitor, belzutifan, has shown effectiveness in treating advanced clear-cell renal cell carcinoma in the LITESPARK-005 and -003 trials, both as a standalone treatment and in combination with VEGFR tyrosine kinase inhibitors.
These promising results suggest that belzutifan could be used more broadly in cancer treatment, potentially extending its application beyond just von Hippel-Lindau disease-associated cancers.
Expanding "Practice-Changing" Belzutifan's Reach in RCC.[2023]
In a phase 2 study involving 52 patients with advanced clear cell renal cell carcinoma who had previously received immunotherapy, the combination of belzutifan and cabozantinib showed promising antitumour activity, with 30.8% of patients achieving a confirmed objective response, including one complete response.
The treatment was associated with manageable safety concerns, primarily hypertension (27% of patients), and serious treatment-related adverse events occurred in 29% of patients, indicating that while the combination therapy is effective, careful monitoring for side effects is necessary.
Belzutifan plus cabozantinib for patients with advanced clear cell renal cell carcinoma previously treated with immunotherapy: an open-label, single-arm, phase 2 study.Choueiri, TK., McDermott, DF., Merchan, J., et al.[2023]

Citations

59: Real-World efficacy and safety of belzutifan in sporadic ...The disease control rate was 64%. The median OS was 6.93 months (95% CI: 3.75-12.58). The median PFS was 3.09 months (95% CI 2.53-5.72). AEs of ...
Belzutifan for Renal Cell Carcinoma in von Hippel–Lindau ...Belzutifan was associated with predominantly grade 1 and 2 adverse events and showed activity in patients with renal cell carcinomas and non–renal cell ...
NCT04489771 | A Study of Belzutifan (MK-6482) in ...This study will compare the efficacy and safety of two doses of belzutifan in participants with advanced renal cell carcinoma (RCC) with clear cell ...
Efficacy Data for WELIREG® (belzutifan) | VHL DiseaseNearly half of patients achieved tumor reduction in VHL disease–associated RCC with WELIREG · MEDIAN DURATION OF RESPONSE WAS NOT REACHED · NCCN Clinical Practice ...
Merck & Co., Inc., Rahway, NJ, USA and ...Real-World Treatment Patterns and Clinical Outcomes Among Patients With Advanced Renal Cell Carcinoma. Clinical Genitourinary Cancer April 2024, ...
6.pubmed.ncbi.nlm.nih.govpubmed.ncbi.nlm.nih.gov/40920370/
The Safety Profile of Belzutifan in Renal TumorsThe efficacy and side effects of belzutifan are well-documented from clinical trials, however, real-world data examining the incidence and ...
The safety profile of belzutifan in renal tumors: real-world data ...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 ...
Merck and Eisai Announce WELIREG® (belzutifan) Plus ...The safety profiles of WELIREG and LENVIMA in this trial ... WELIREG is indicated for the treatment of adult patients with advanced renal cell carcinoma ...
Safety and efficacy profile of belzutifan, a hypoxia-inducible ...Belzutifan is a novel drug that inhibits HIF-2α which in turn results in anti-tumor effects of the drug against ccRCC.
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