MSD Belzutifan PAS

November 26, 2024 updated by: Merck Sharp & Dohme LLC

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

Study Overview

Status

Withdrawn

Conditions

Von Hippel Lindau Disease

Intervention / Treatment

Other: Belzutifan

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 Type

Observational

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

United States
- Texas
  - Houston, Texas, United States, 77030
    - MD Anderson Cancer Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

Adult
Older Adult

Accepts Healthy Volunteers

Sampling Method

Non-Probability Sample

Study Population

MD Anderson Cancer Center

Description

Inclusion Criteria:

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

Exclusion Criteria:

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Procedure Time Frame: Through study completion	Proportion of patients who undergo surgery or other tumor reductive procedure.	Through study completion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Merck Sharp & Dohme LLC

Collaborators

Merck Sharp & Dohme LLC

Investigators

Study Director: Medical Director, Merck Sharp & Dohme LLC

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

HMA-EMA Catalogues of real-world data: EU PAS number: EUPAS108114

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 30, 2024

Primary Completion (Estimated)

December 31, 2030

Study Completion (Estimated)

December 31, 2030

Study Registration Dates

First Submitted

August 12, 2024

First Submitted That Met QC Criteria