Recruiting
Phase 2

Belzutifan

Sponsor:

Merck Sharp & Dohme LLC

Code:

NCT07700758

Conditions

Malignant Neoplasm

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Interventions

Belzutifan

Study Details

Brief summary:

Researchers are looking for new ways to treat adolescents with locally advanced, unresectable, or metastatic solid tumors. Participants were enrolled into pheochromocytoma/paraganglioma (PPGL), wild type gastrointestinal stromal tumor (wtGIST), and Von Hippel-Lindau (VHL) disease-associated localized tumors cohorts:

  • PPGL are rare cancers that start in cells that make hormones in the adrenal glands
  • wtGIST is a less common type of cancer that starts in the digestive tract. Wild type means it does not have certain gene mutations (changes)
  • VHL disease-associated localized tumors are rare tumors caused by a certain gene mutation that may be passed down from parents to children
  • Locally advanced means the cancer has spread into nearby tissue
  • Unresectable means the cancer cannot be removed by surgery
  • Metastatic means the cancer has spread to other parts of the body

The goal of the study is to learn about the safety of belzutifan and if people tolerate it.

Conditions

Malignant Neoplasm

Study ID

NCT07700758

Start date

Nov 23, 2026

Status verified date

Oct, 2026

Completion date

Jan 2, 2034

Anticipated

Primary completion date

Jan 2, 2034

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Inclusion Criteria:

The main inclusion criteria include but are not limited to the following:

  • Has a diagnosis of one of the following: locally advanced, unresectable, or metastatic pheochromocytoma/paraganglioma or wild-type gastrointestinal stromal tumors, or localized tumors associated with von Hippel-Lindau disease
  • Has measurable disease per RECIST 1.1

Exclusion Criteria:

The main exclusion criteria include but are not limited to the following:

  • Has a pulse oximeter reading <92% at rest, requires intermittent supplemental oxygen, or requires chronic supplemental oxygen
  • Has clinically significant cardiac disease or electrocardiogram indicating uncontrolled cardiac condition or has congenital long QT syndrome
  • Has a history of human immunodeficiency virus infection
  • Has received prior treatment with any hypoxia inducible factor-2α inhibitor, including belzutifan
  • Has had an allogenic tissue/solid organ transplant
  • Has a history of autologous stem cell transplant within 6 months of start of study intervention
  • Has known additional malignancy that is progressing or has required active treatment within the past 2 years
  • Has known active central nervous system metastases and/or carcinomatous meningitis
  • Has an active infection requiring systemic therapy

Study Design

Enrollment

15 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Belzutifan

Participants will receive belzutifan 80 mg (body weight <40 kg) or 120 mg (body weight ≥40 kg) orally once daily for approximately 2 years.

Interventions

Belzutifan

Administered once daily via oral tablet

Primary outcome measure

  • Number of Participants Who Experience One or More Adverse Events (AEs) [ Time Frame: Up to approximately 5 years ]
  • Number of Participants Who Discontinue Study Intervention Due to an AE [ Time Frame: Up to approximately 5 years ]

Central Contacts and Locations

Central contacts

Toll Free Number

1-888-577-8839Trialsites@merck.com

Locations

University of Iowa ( Site 5017)

Recruiting

Iowa City, Iowa, United States, 52242

Contacts

Study Coordinator

319-356-2296

More Information

Sponsor

Merck Sharp & Dohme LLC

Last update posted

Oct 5, 2026

Last verified

Oct, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-06. This information was provided to ClinicalTrials.gov by Merck Sharp & Dohme LLC on 2026-10-05. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.