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Could a daily pill tame rare cancers in teens? new trial investigates

NCT ID NCT07700758

Disease control Sponsor: Merck Sharp & Dohme LLC Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jul 14, 2026 · Last updated Sep 01, 2026 · Updated 7 times

Summary

This phase 2 trial tests the safety of belzutifan, a daily oral tablet, in adolescents with rare solid tumors that cannot be surgically removed or have spread. The study includes three tumor types: pheochromocytoma/paraganglioma (PPGL), wild-type gastrointestinal stromal tumors (wtGIST), and von Hippel-Lindau (VHL) disease-associated tumors. Researchers aim to see how many participants experience side effects and whether they can tolerate the drug.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
belzutifan, an oral tablet taken once daily
What this could lead to
If successful, this trial could show that belzutifan is a safe and tolerable treatment option for adolescents with certain rare solid tumors.
What could go wrong
This is an early-phase, small study (15 participants) focused on safety, not yet on how well the drug works. The results may not apply to all patients or tumor types.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 15 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Nov 2026

An estimate. Start dates often move.

Expected to finish

Jan 2034

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

12 to 17 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

cancer gastrointestinal stromal tumor neoplasm pheochromocytoma-paraganglioma von Hippel-Lindau disease

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Malignant neoplasm

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Study contacts

  • Contact

    Email: •••••@•••••

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