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Not yet recruiting NCT07700758

A Study of Belzutifan in Adolescent Participants With Solid Tumors (MK-9999-01E/LIGHTBEAM-U01)

Phase II Interventional Malignant Neoplasm

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Belzutifan.
Who it may be relevant to
Registry conditions: Malignant Neoplasm. Basic parameters: 12 years — 17 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
Center list to be confirmed — check the primary protocol.
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

LIGHTBEAM-U01 Substudy 01E: A Phase 2 Substudy to Evaluate the Safety and Efficacy of Belzutifan in Participants With Solid Tumors

Overview

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.

Interventions

  • Drug Belzutifan
    Administered once daily via oral tablet

Primary outcome measures

  • 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]
Secondary outcome measures (5)
  • Area Under the Concentration-Time Curve From Time 0 to 24 hours of Belzutifan [Time frame: At designated timepoints (up to 5 weeks)]
  • Minimum Plasma Concentration (Cmin) of Belzutifan [Time frame: At designated timepoints (up to 5 weeks)]
  • Maximum Plasma Concentration (Cmax) of Belzutifan [Time frame: At designated timepoints (up to 5 weeks)]
  • Objective Response Rate (ORR) [Time frame: Up to approximately 5 years]
  • Duration of Response (DOR) [Time frame: Up to approximately 5 years]

Eligibility criteria

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
N/A
Model
Single group
Masking
Open label
Primary purpose
Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Identifiers

NCT: NCT07700758 · 9999-01E · U1111-1330-9370 · 2025-524515-37-00 · MK-9999-01E · LIGHTBEAM-U01

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗