MSD Belzutifan PAS

Withdrawn

• Conditions: Von Hippel Lindau Disease

• Registration Number: NCT06554730
• Lead Sponsor: Merck Sharp & Dohme LLC

Brief 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 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 Timeline

• Study Start: 2024-06-30
• Study First Submitted: 2024-08-12
• Study First Submitted QC: 2024-08-12
• Study First Posted: 2024-08-15
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-26
• Last Update Posted: 2024-11-29
• Primary Completion: 2030-12-31
• Study Completion: 2030-12-31

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: 100

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Procedure	Through study completion	Proportion of patients who undergo surgery or other tumor reductive procedure.

Trial Locations

Locations (1)

MD Anderson Cancer Center; 🇺🇸 Houston, Texas, United States