Paraganglioma Clinical Trials and Treatments

Paragangliomas are neuroendocrine tumors arising from chromaffin cells in extra-adrenal locations including the carotid bifurcation (carotid body tumor), jugular bulb, sympathetic chain, retroperitoneum, thorax, and bladder. Approximately 30-40% are hereditary, primarily caused by mutations in genes regulating succinate dehydrogenase (SDH complex) including SDHA, SDHB, SDHC, SDHD, and SDHF. Sporadic paragangliomas also associate with mutations in NF1, RET, VHL, PHD1, and PHD2 genes.

The most common symptoms of Paraganglioma include Hypertension and hypertensive episodes, Palpitations and tachycardia, Diaphoresis and flushing, Headaches and chest/abdominal pain, Metastatic disease in malignant forms, among others. Paraganglioma affects approximately 1 in 300,000 to 1,000,000; ~30% are hereditary. Symptoms can range from mild to severe and may vary between individuals.

Trial Friend lists all active clinical trials for Paraganglioma from ClinicalTrials.gov, with eligibility criteria translated into plain English. Patients can filter by trial phase and recruitment status, search by location, and use the Match Me tool to score trials against their profile. Genetic testing identifies SDH mutations, RET, NF1, and VHL alterations, guiding surveillance of mutation carriers and family members. Surgical resection remains primary therapy for localized disease, with meticulous dissection to preserve cranial nerves in head/neck paragangliomas.

Yes. As of September 2026, Trial Friend lists 1 FDA-approved drug used to treat Paraganglioma: Welireg (belzutifan), first approved in 2025.

Each has its own page with what the label covers, side effects and how it is given, and the Treatments section of this page adds any further approved drugs found in FDA labeling data.

Paraganglioma is caused by variants in any of many genes, most often SDHB, SDHD, SDHC, SDHAF2, SDHA and VHL. SDHD and SDHAF2 disease shows paternal-only transmission; SDHB carriers have the highest metastatic risk.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it. The condition affects approximately 1 in 300,000 to 1,000,000; ~30% are hereditary. A multigene panel identifies the cause in most families.

Yes. Paraganglioma can be caused by variants in many different genes (SDHB, SDHD, SDHC, SDHAF2, SDHA and VHL are among the most common), so testing is usually a multigene panel or exome sequencing rather than one gene at a time. SDHD and SDHAF2 disease shows paternal-only transmission; SDHB carriers have the highest metastatic risk.

It is autosomal dominant, so each child of an affected parent has a 1 in 2 chance of inheriting it.

Knowing the exact gene matters: it decides which trials a patient qualifies for and which relatives should be tested. The labs currently offering the test are listed in the Genetic Testing section of this page, pulled live from the NIH Genetic Testing Registry. A genetic counselor can help interpret the result and decide which relatives should be tested.

Eligibility for Paraganglioma clinical trials depends on factors like age, diagnosis, treatment history, and overall health. Each trial sets its own inclusion and exclusion criteria. Trial Friend translates these requirements from medical language into plain English so patients can review them more easily. The Match Me tool can score your profile against active trials to help identify studies that may be a fit.

Participating in a Paraganglioma clinical trial typically involves scheduled visits, medical tests, and close monitoring by a research team. Depending on the study phase, you may receive an investigational treatment, a placebo, or the current standard of care. Trials may last from a few weeks to several years. Study participation is voluntary, and you can withdraw at any time.