Study identification

PURI

https://redirect.ema.europa.eu/resource/199007

EU PAS number

EUPAS108114

Study ID

199007

Official title and acronym

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)

DARWIN EU® study

No

Study countries

United States

Study description

Von Hippel-Lindau (VHL) disease is a rare autosomal dominant disease characterized by an increased prevalence of recurring benign and malignant tumors including renal cell carcinomas (RCCs), central nervous system (CNS) hemangioblastomas, and pancreatic neuroendocrine tumors (pNETs). The only systemic therapy approved for the treatment of certain patients with VHL disease-associated neoplasms is belzutifan (WELIREG®), which was initially approved by the United States (US) Food and Drug Administration (FDA) in August 2021 for the treatment of adult patients with VHL disease who require therapy for associated RCC, CNS hemangioblastoma, or pNET, not requiring immediate surgery. The primary aim of this registry is to further characterize the effectiveness of belzutifan for patients with von Hippel-Lindau (VHL) disease-associated renal cell carcinoma (RCC) and/or central nervous system (CNS) hemangioblastoma treated in real-world clinical practice. The primary effectiveness parameter includes tumor reductive procedures given the clinical importance of this parameter and association with subsequent morbidity and mortality of disease. Secondary aims are to describe potential serious adverse events (SAEs), occurrence of new VHL disease-associated tumors or tumor type, and metastasis during belzutifan use, and to evaluate treatment patterns.

Study status

Ongoing
Research institutions and networks

Institutions

Merck & Co.
First published:
01/02/2024
Institution

Contact details

Clinical Trials Disclosure Merck Sharp & Dohme LLC

Primary lead investigator
Study timelines

Date when funding contract was signed

Planned:
Actual:

Study start date

Planned:
Actual:

Data analysis start date

Planned:

Date of interim report, if expected

Planned:

Date of final study report

Planned:
Sources of funding
Pharmaceutical company and other private sector 

More details on funding

Merck Sharp & Dohme LLC
Study protocol
Initial protocol

MK-6482-026-00-v3-with-ha-approval-date_Protocol_redaction-final.pdf

English (1.91 MB - PDF)View document
Regulatory

Was the study required by a regulatory body?

Yes

Is the study required by a Risk Management Plan (RMP)?

Non-EU RMP only