Von Hippel Lindau DiseaseNCT06554730
MSD Belzutifan PAS
An observational study of Von Hippel Lindau Disease, run by Merck Sharp & Dohme LLC.
Trial summary
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice. This study has been registered and will be maintained through close out on the RWD Catalogues Observational Registry - https://catalogues.ema.europa.eu/ under the EU PAS… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Von Hippel Lindau Disease
- What is being tested?
- Belzutifan
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 30 Jun 2024to 31 Dec 2030
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Study population: MD Anderson Cancer Center
Who may be able to join
- Greater than or equal to 18 years of age
- Diagnosed with VHL disease based on a germline test or clinical diagnosis
- A decision has been made by the treating physician (at his/her discrestion) to intitate the first belzutifan treatment (i.e., belzutifan 'new users'), within the prescribing conditions of the approved product label
- Signed informed consent prior to or withing 30 days after the first inititaion of belzutifan
Who cannot join
- Anti-cancer systemic therapy within 2 weeks prior to the index date
- Unable to consent to participate in the study
- History of VHL disease-related metastasis or advanced cancer
About this study
Official title: Non-interventional Post-authorization Study of Belzutifan in Adult Patients With Von Hippel-Lindau Disease-associated Renal Cell Carcinoma, Pancreatic Neuroendocrine Tumor and/or Central Nervous System Hemangioblastoma (MK-6482-026)
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice.
This study has been registered and will be maintained through close out on the RWD Catalogues Observational Registry - https://catalogues.ema.europa.eu/ under the EU PAS number:
EUPAS108114
Read the detailed description
Primary Objective
- Among new users of belzutifan with VHL disease-associated RCC, to describe the proportion of patients who undergo at least one renal tumor reductive surgery (e.g., nephrectomy) or locally directed therapy (e.g., radiofrequency ablation).
- Among new users of belzutifan with VHL disease-associated CNS hemangioblastoma, to describe the proportion of patients who undergo at least one CNS tumor reductive surgery (e.g., craniectomy) or locally directed therapy (e.g., radiation therapy).
Secondary Objectives
Among all new users of belzutifan to describe:
- Proportion of patients with treatment emergent SAEs, including the nature of these events.
Treatment patterns including the:
- Duration of therapy
- Proportion of patients who discontinued treatment, time to treatment discontinuation, and summary of reasons for discontinuation
- Proportion of patients who interrupted treatment, time to treatment interruption, duration of treatment interruption, and summary of reasons for treatment interruption
- Proportion of patients with dose reductions, and reason for dose reduction.
- Among new users of belzutifan with VHL disease-associated RCC and, separately, VHL disease-associated CNS hemangioblastoma, to describe:
- Proportion of patients who develop metastatic disease (for RCC only)
- Proportion of patients with occurrence of new VHL disease-associated tumor or tumor type
- Conditions
- Von Hippel Lindau Disease
- Being tested
- Belzutifan
- Keywords
- Belzutifan, VHL
- Medical subject headings
- von Hippel-Lindau Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BelzutifanOther
There is no intervention for this study. This study will be collecting data from the EMR of consenting subjects who are taking Belzutifan.
What is being measured
The primary outcome is the main question the study is designed to answer.
Procedure
Proportion of patients who undergo surgery or other tumor reductive procedure.
Measured: Through study completion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Aug 2024
- Study start30 Jun 2024
- Primary completion31 Dec 2030 (estimated)
- Study completion31 Dec 2030 (estimated)
Why it stopped: Study is registered /active on RWD Catalogues (https://catalogues.ema.europa.eu) - Please refer to EU PAS number: EUPAS108114
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Hippel Lindau DiseaseStudies of the same condition, open and under way.
- More trials run by Merck Sharp & Dohme LLCThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Merck Sharp & Dohme LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Merck Sharp & Dohme LLC
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06554730
- Sponsor's protocol ID
- 2023-0221
- Other IDs
- MK-6482-026, NCI-2024-06807, EUPAS108114
- Status last verified
- Nov 2024
- First submitted
- 12 Aug 2024
- Last updated
- 29 Nov 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-interventional Post-authorization Study of Belzutifan in Adult Patients With Von Hippel-Lindau Disease-associated Renal Cell Carcinoma, Pancreatic Neuroendocrine Tumor and/or Central Nervous System Hemangioblastoma (MK-6482-026)
- ClinicalTrials.gov ID
- NCT06554730
- Acronym / sponsor ID
- 2023-0221
- Conditions
- Von Hippel Lindau Disease
- Keywords
- Belzutifan, VHL
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: Study is registered /active on RWD Catalogues (https://catalogues.ema.europa.eu) - Please refer to EU PAS number: EUPAS108114
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Merck Sharp & Dohme LLCIndustry (pharma, biotech, devices)
- Study start
- 30 Jun 2024 (actual)
- Primary completion
- 31 Dec 2030 (estimated)
- Study completion
- 31 Dec 2030 (estimated)
- First posted
- 15 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective observational cohort study evaluating the effectiveness and safety of belzutifan treatment in routine clinical practice.
This study has been registered and will be maintained through close out on the RWD Catalogues Observational Registry - https://catalogues.ema.europa.eu/ under the EU PAS number:
EUPAS108114
Detailed description
Primary Objective
- Among new users of belzutifan with VHL disease-associated RCC, to describe the proportion of patients who undergo at least one renal tumor reductive surgery (e.g., nephrectomy) or locally directed therapy (e.g., radiofrequency ablation).
- Among new users of belzutifan with VHL disease-associated CNS hemangioblastoma, to describe the proportion of patients who undergo at least one CNS tumor reductive surgery (e.g., craniectomy) or locally directed therapy (e.g., radiation therapy).
Secondary Objectives
Among all new users of belzutifan to describe:
- Proportion of patients with treatment emergent SAEs, including the nature of these events.
Treatment patterns including the:
- Duration of therapy
- Proportion of patients who discontinued treatment, time to treatment discontinuation, and summary of reasons for discontinuation
- Proportion of patients who interrupted treatment, time to treatment interruption, duration of treatment interruption, and summary of reasons for treatment interruption
- Proportion of patients with dose reductions, and reason for dose reduction.
- Among new users of belzutifan with VHL disease-associated RCC and, separately, VHL disease-associated CNS hemangioblastoma, to describe:
- Proportion of patients who develop metastatic disease (for RCC only)
- Proportion of patients with occurrence of new VHL disease-associated tumor or tumor type
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- MD Anderson Cancer Center
Key inclusion criteria (4 listed)
- Greater than or equal to 18 years of age
- Diagnosed with VHL disease based on a germline test or clinical diagnosis
- A decision has been made by the treating physician (at his/her discrestion) to intitate the first belzutifan treatment (i.e., belzutifan 'new users'), within the prescribing conditions of the approved product label
- Signed informed consent prior to or withing 30 days after the first inititaion of belzutifan
Key exclusion criteria (3 listed)
- Anti-cancer systemic therapy within 2 weeks prior to the index date
- Unable to consent to participate in the study
- History of VHL disease-related metastasis or advanced cancer
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BelzutifanThere is no intervention for this study. This study will be collecting data from the EMR of consenting subjects who are taking Belzutifan. | Other | – |
Primary outcome
Procedure
Time frame: Through study completion
Proportion of patients who undergo surgery or other tumor reductive procedure.
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Nov 2024.
- HMA-EMA Catalogues of real-world data: EU PAS number: EUPAS108114Link provided by the sponsor.
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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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Merck Sharp & Dohme LLC
Questions about this trial
What is this clinical trial studying?
An observational study of Von Hippel Lindau Disease, run by Merck Sharp & Dohme LLC.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 29 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Procedure (time frame: Through study completion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Nov 2024.