Belzutifan for teenagers with advanced or spreading solid tumors
Tests belzutifan in adolescents with advanced solid tumors, including pheochromocytoma and paraganglioma.
Plain-language title generated from the registry record
Registry title: A Study of Belzutifan in Adolescent Participants With Solid Tumors (MK-9999-01E/LIGHTBEAM-U01)
Key facts
- Where
- Milan, Rome, and Turin, ItalySee the 3 sites
- Phase
- Phase 2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 12 and 17 years old?
Source: NCT07700758, who can take part
Where in Italy
- Fondazione IRCCS - Istituto Nazionale dei TumoriMilan, ItalyNot yet recruiting
- Ospedale Pediatrico Bambino GesùRome, ItalyNot yet recruiting
- Azienda Ospedaliera Universitaria Citta Della Salute E Della Scienza Di TorinoTurin, ItalyNot yet recruiting
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Merck Sharp & Dohme LLC
- Trial code
- NCT07700758EU 2025-524515-37-00
- Record updated
- 10 November 2026
- Original description
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:Read full original descriptionShow less
LIGHTBEAM-U01 Substudy 01E: A Phase 2 Substudy to Evaluate the Safety and Efficacy of Belzutifan in Participants With Solid Tumors
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.
Inclusion: 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: 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 human immunodeficiency virus infection; 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 an active infection requiring systemic therapy; Has clinically significant cardiac disease or electrocardiogram indicating uncontrolled cardiac condition or has congenital long QT syndrome; 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 known active central nervous system metastases and/or carcinomatous meningitis.