Clinical trial NCT06554730: MSD Belzutifan PAS
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)
Study type: OBSERVATIONAL
Overall status: WITHDRAWN
Conditions: Von Hippel Lindau Disease
Keywords: Belzutifan
VHL
Lead sponsor: Merck Sharp & Dohme LLC
Sponsor class: INDUSTRY
First posted: 2024-08-15
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: MD Anderson Cancer Center

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

Eligibility criteria: 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

Primary outcomes:
measure: Procedure
timeFrame: Through study completion
Secondary outcomes:
Other outcomes:
Locations:
Study officials:
affiliation: Merck Sharp & Dohme LLC
name: Medical Director
role: STUDY_DIRECTOR
References: