This study is NOT currently recruiting participants.
Number
001715-C
Sponsoring Institute
National Cancer Institute (NCI)
Recruitment Detail
Type: Recruitment has not started Gender: Male & Female Min Age: 12 Years Max Age: 120 Years
Referral Letter Required
No
Population Exclusion(s)
Fetuses;Pregnant Women;Neonates
Keywords
HIF-2 alpha; Pheochromocytoma/paraganglioma; Pancreatic NET; Gastrointestinal Stromal Tumor
Recruitment Keyword(s)
None
Condition(s)
Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms, Nerve Tissue; Neoplasms, Connective Tissue; Neoplasms, Connective and Soft Tissue; Gastrointestinal Neoplasms; Digestive System Neoplasms; Digestive System Diseases; Gastrointestinal Diseases; Adenoma; Neoplasms, Glandular and Epithelial; Pancreatic Neoplasms; Neoplasms by Site; Endocrine Gland Neoplasms
Investigational Drug(s)
Belzutifan
Investigational Device(s)
Intervention(s)
Drug: Belzutifan
Supporting Site
National Cancer Institute
Pheochromocytomas and paraganglioma (PPGLs) are rare tumors of the adrenal glands and related nerves. About 2 to 8 people per million are diagnosed with PPGLs in the US each year. Between 15% and 20% of these tumors are cancerous.
SDH-deficient gastrointestinal stromal tumor, also known as SDH-deficient GIST, is a form of gastrointestinal malignancy that primarily affects the stomach or small intestine, with less common occurrences in the large intestine. Approximately 5% to 7.5% of GISTs are classified as "SDH-deficient" and primarily manifest in the stomach. SDH refers to succinate dehydrogenase, a critical protein involved in energy production within the body. SDH-deficient GISTs are predominantly observed in pediatric patients and certain young adults.
HIF-2-alpha serves as a crucial modulator of the cellular oxygen-signaling cascade. It plays a role in upregulating genes associated with cell growth, metastasis, and tumor progression. Activation of HIF-1-alpha has been closely linked with various types of malignancies.
Researchers want to know if an FDA-approved drug called belzutifan can help treat PPGLs, SDH-deficient GIST, HIF-2-alpha related-cancers.
Objective:
To test a drug (belzutifan) in people with PPGLs, GIST and HIF-2-alpha related-cancers.
Eligibility:
People aged 12 years and older with PPGLs, GIST and HIF-2-alpha related-cancers, and related cancers that have not responded to treatment.
Design:
Participants will be screened. They will have a physical exam, imaging scans, and blood tests. They will have tests of their heart function. Small pieces of tumor tissue will be collected. They will have a bone scan and an eye exam.
Belzutifan is a pill taken by mouth. Participants will take the drug at home once a day. They will write down their doses in a diary. They may also track their blood pressure at home.
Participants will visit the clinic 9 times in their first 6 months. Then they will have clinic visits every 4 weeks. Imaging scans, blood tests, and other tests will be repeated during the study. Some blood may be used for genetic testing.
Participants may remain in the study as long as the drug is helping them. If they stop taking the drug, they will have a follow-up visit after 4 weeks. Follow-up visits will continue every 8 to 12 weeks.
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INCLUSION CRITERIA: Male or female adults and adolescents >=12 years of age and weighing >=40 kg (cohort specific exceptions noted below) with one of the following indications: - Advanced (unresectable and/or metastatic) PPGL - Advanced pNET who have progressed on or after at least one prior systemic therapy, which includes an approved targeted agent (such as sunitinib or everolimus) - VHL disease-associated localized tumors - Advanced wt GIST - Advanced solid tumors with HIF-2-alpha related genetic alterations
Male or female adults and adolescents >=12 years of age and weighing >=40 kg (cohort specific exceptions noted below) with one of the following indications:
- Advanced (unresectable and/or metastatic) PPGL
- Advanced pNET who have progressed on or after at least one prior systemic therapy, which includes an approved targeted agent (such as sunitinib or everolimus)
- VHL disease-associated localized tumors
- Advanced wt GIST
- Advanced solid tumors with HIF-2-alpha related genetic alterations
Principal Investigator
Referral Contact
For more information: