A Phase 2 interventional study of Belzutifan in VHL - Von Hippel-Lindau Syndrome, VHL Gene Mutation and VHL Syndrome, sponsored by Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA). Active, not recruiting at 11 sites in 4 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2026-04-13.
Sponsored by Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) (part of Merck & Co., Inc) · Phase 2, Interventional, and Treatment
Among 33 studies in von Hippel-Lindau Disease with a site in United States — 8 open to participants now, 11 under way, 1 of them phase 3. von Hippel-Lindau Disease · United States.
Among the 33 studies in von Hippel-Lindau Disease with a site in United States, this one is the 6th largest by planned enrollment of the 23 interventional ones, is the only one that started in 2018, is one of 11 in phase 2, and was the 25th registered.
55 studies on the registry are indexed under von Hippel-Lindau Disease; 23 are open to participants now.
This study's planned enrollment of 50 is above the median of 28 across 31 interventional studies indexed under von Hippel-Lindau Disease.
Browse von Hippel-Lindau Disease studies →8 of the 33 studies in von Hippel-Lindau Disease with a site in United States are open now, in 12 countries. Each count lists the studies.
Of the 20 von Hippel-Lindau Disease studies recruiting now, 16 (80%) set no upper age limit and 0 (0%) stop at 75 or younger; 9 take children under 18; 5 take healthy volunteers.
This study: 18 Years and older · all sexes · no healthy volunteers.
The full table on the hub →33 studies; the count open now beside each. This study's row is marked.
First in 1997; this study’s year is marked. 1 started in 2018.
Median enrollment 15 across the 23 interventional studies, 300 across the 10 observational. Of the 22 that completed or were terminated, 9 (41%) have results posted.
6 of 33 have an industry lead sponsor. The 10 most frequent:
Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) is the lead sponsor of 7 studies on the registry; none are open to participants now.
Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) has 2 studies filed under von Hippel-Lindau Disease — fewer than ten, so there is no comparison to draw.
3 of the studies in von Hippel-Lindau Disease with a site in United States that are under way reach primary completion within a year, 0 of them phase 3. Phase 3 first, then by date:
| Study | Primary completion |
|---|---|
| NCT05955014M.D. Anderson Cancer CenterData Collection Protocol for Patients With Von Hippel Lindau Disease | 2026-12-31 |
| NCT03401788Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA)A Phase 2 Study of Belzutifan (PT2977, MK-6482) for the Treatment of Von Hippel Lindau (VHL) Disease-Associated Renal Cell Carcinoma (RCC) (MK-6482-004) | 2027-04-29 |
| NCT05737602Massachusetts General HospitalPromoting Stress Management and Resilience Among Individuals With Von Hippel- Lindau Disease | 2027-05-01 |
Among the 33 studies in von Hippel-Lindau Disease with a site in United States, from their registry updates:
Nothing recorded among them since 2026-09-25.
This study is designed to investigate belzutifan as a treatment for VHL disease associated RCC.
This open-label Phase 2 study will evaluate the efficacy and safety of belzutifan in participants with VHL disease who have at least 1 measurable RCC tumor. Belzutifan will be administered orally and treatment will be continuous. Participants will be evaluated radiologically approximately 12 weeks after initiation of treatment and every 12 weeks thereafter while continuing in the study for a minimum of 3 years and then every 24 weeks or more frequently if clinically indicated. Changes in VHL disease-associated non-RCC tumors will also be evaluated.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Investigational drug or device
A drug or device being studied in a clinical trial.
It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Metastases
Cancer that has spread from where it began to other parts of the body. “Metastatic” cancer is cancer that has spread this way.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Participants receive 120 mg belzutifan orally once daily. Participants may continue to receive belzutifan in the absence of unacceptable treatment related toxicity or unequivocal disease progression.
Drug: Belzutifan
120 mg once daily (three 40 mg oral tablets once daily).
Also known as: PT2977, MK-6482, WELIREG™
Objective Response Rate (ORR) in VHL Disease-Associated RCC Tumors
ORR is defined as the percentage of participants in the analysis population who have a best confirmed response of Complete Response (CR: Disappearance of all target lesions) or a Partial Response (PR: At least a 30% decrease in the sum of diameters of target lesions) per Response Evaluation Criteria in Solid Tumors version 1.1 (RECIST 1.1). ORR will be assessed by independent review committee (ICR) for the primary analysis.
Time frame: Up to approximately 4 years
Duration of Response (DOR) in VHL Disease-Associated RCC Tumors
DOR is defined as the interval from the first documented evidence of a CR (disappearance of all target lesions) or a PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1 until Progressive Disease (PD) or death due to any cause, whichever occurs first, in participants demonstrating a best confirmed response of CR or PR. Per RECIST 1.1, PD is defined as at least a 20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD.
Time frame: Up to approximately 4 years
Time to Response (TTR) in VHL Disease-Associated RCC Tumors
TTR is defined as the interval from the start of study treatment to the first documentation of a response per RECIST 1.1, and calculated for participants with a best confirmed response of CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions).
Time frame: Up to approximately 4 years
Progression-free Survival (PFS) in VHL Disease-Associated RCC Tumors
PFS is defined as the interval from the start of study treatment to the first documented PD or death from any cause, whichever occurs first. Per RECIST 1.1, PD is defined as at least a 20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD.
Time frame: Up to approximately 4 years
Time to Surgery (TTS) in VHL Disease-Associated RCC Tumors
TTS was defined as the interval from the start of study treatment to the date of surgery.
Time frame: Up to approximately 4 years
ORR in VHL Disease-Associated Non-RCC Tumors
ORR is defined as the percentage of participants in the analysis population who have a best confirmed response of CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1.
Time frame: Up to approximately 4 years
DOR in VHL Disease-Associated Non-RCC Tumors
DOR is defined as the interval from the first documented evidence of a CR (disappearance of all target lesions) or a PR (at least a 30% decrease in the sum of diameters of target lesions) per RECIST 1.1 until PD or death due to any cause, whichever occurs first, in participants demonstrating a best confirmed response of CR or PR. Per RECIST 1.1, PD is defined as at least a 20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD.
Time frame: Up to approximately 4 years
TTR in VHL Disease-Associated Non-RCC Tumors
TTR is defined as the interval from the start of study treatment to the first documentation of a response per RECIST 1.1, and calculated for participants with a best confirmed response of CR (disappearance of all target lesions) or PR (at least a 30% decrease in the sum of diameters of target lesions).
Time frame: Up to approximately 4 years
PFS in VHL Disease-Associated Non-RCC Tumors
PFS is defined as the interval from the start of study treatment to the first documented PD or death from any cause, whichever occurs first. Per RECIST 1.1, PD is defined as at least a 20% increase in the sum of diameters of target lesions. In addition to the relative increase of 20%, the sum must also demonstrate an absolute increase of at least 5 mm. The appearance of one or more new lesions is also considered PD.
Time frame: Up to approximately 4 years
TTS in VHL Disease-Associated Non-RCC Tumors
TTS was defined as the interval from the start of study treatment to the date of surgery.
Time frame: Up to approximately 4 years
Number of Participants Experiencing an Adverse Event (AE)
An AE is any untoward medical occurrence in a participant regardless of its causal relationship to study treatment. An AE can be any unfavorable and unintended sign (including any clinically significant abnormal laboratory test result), symptom, or disease temporally associated with the use of the study drug, whether or not it is considered to be study drug related.
Time frame: Up to approximately 4 years
Number of Participants Experiencing a Serious Adverse Event (SAE)
An SAE is any AE occurring at any dose and regardless of causality that meets any of the following criteria: results in death, is life-threatening, requires inpatient hospitalization or prolongation of existing hospitalization, results in persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, is a congenital anomaly or birth defect in an offspring of a participant taking study drug, or is an important medical event.
Time frame: Up to approximately 4 years
Belzutifan Plasma Concentration
Blood samples for the determination of belzutifan concentration will be collected at pre-specified timepoints before and after treatment administration.
Time frame: Weeks 1 and 3: pre-dose, 2 and 5 hours post-dose, Week 5, 9, and 13: post-dose.
Belzutifan Metabolite Plasma Concentration
Blood samples for the determination of belzutifan metabolite concentration will be collected at pre-specified timepoints before and after treatment administration.
Time frame: Weeks 1 and 3: pre-dose, 2 and 5 hours post-dose, Week 5, 9, and 13: post-dose.
Plan to share: Yes — http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf
Data sharing details ↗ · engagezone.msd.com
Started May 2018 (actual). Primary completion is estimated for Apr 2027, 107 months after the start.
How these figures are worked out, and which studies are left out of them.
This study is active, not recruiting, as verified in Apr 2026. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Belzutifan
Peloton Therapeutics, Inc., a subsidiary of Merck & Co., Inc. (Rahway, New Jersey USA) →