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Endocrine & Hormonal

Pheochromocytoma Clinical Trials and Treatments

Also called Chromaffin Cell Tumor, Paraganglioma (extra-adrenal)

Pheochromocytoma is a neuroendocrine tumor arising from chromaffin cells, most commonly in the adrenal medulla (90%), though extra-adrenal tumors (paragangliomas) occur in 10%. The tumor produces catecholamines (epinephrine and norepinephrine) that cause the characteristic symptoms.

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About Pheochromocytoma

Pheochromocytoma is a neuroendocrine tumor arising from chromaffin cells, most commonly in the adrenal medulla (90%), though extra-adrenal tumors (paragangliomas) occur in 10%. The tumor produces catecholamines (epinephrine and norepinephrine) that cause the characteristic symptoms. Episodic catecholamine release causes sudden hypertensive attacks with severe headache, sweating, palpitations, and anxiety.

Attacks can be triggered by abdominal pressure, exercise, micturition, foods containing tyramine, or occur spontaneously. The rule of 10s classically applied: 10% bilateral, 10% familial, 10% malignant, 10% extra-adrenal, 10% in children. However, newer genetic and imaging data suggest higher rates of hereditary disease. Sustained hypertension or episodic hypertension with normal blood pressure between attacks both occur.

Untreated tumors increase risk of myocardial infarction, stroke, cardiomyopathy, and sudden cardiac death. Long-term catecholamine excess can cause irreversible complications. About 10% are malignant with metastatic potential, particularly SDH mutation-associated tumors. Genetic testing is recommended for all patients to identify hereditary syndromes.

Common Symptoms of Pheochromocytoma

Recognizing the signs of Pheochromocytoma early can lead to faster diagnosis and better outcomes. Symptoms may vary in severity from person to person. If you or a loved one are experiencing any of the following, consider speaking with a specialist.

  • Severe hypertension, often episodic or paroxysmal
  • Intense headaches during attacks
  • Profuse sweating
  • Palpitations and chest or abdominal pain
  • Tremor, anxiety, and sense of impending doom
  • Syncope (fainting) or hypertensive crisis with stroke risk

Who Pheochromocytoma Affects

Pheochromocytoma can develop at any age but is most common in the fourth and fifth decades of life. It affects males and females roughly equally. The disease occurs across all racial and ethnic groups. About 30-40% of cases are hereditary, associated with MEN2, NF1, VHL, or SDHA/B/C/D syndromes. Hereditary cases often present at younger ages and may be bilateral or extra-adrenal.

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FDA-Approved Treatments for Pheochromocytoma

There is currently 1 FDA-approved medication for Pheochromocytoma. These therapies represent the current standard of care and may be used alongside or compared against investigational treatments in active clinical trials.

Source: openFDA drug labeling data. This list may not include all treatments. Always consult your doctor.

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Help Paying for Pheochromocytoma Treatment

Charity funds and drugmaker programs for Pheochromocytoma, checked at the source. Pick your insurance to see what fits.

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Charity funds

No charity fund for this condition is open right now. Funds reopen when they receive new donations; the foundations let you sign up to be told when one opens.

Status as each foundation showed it on September 28, 2026.
Drugmaker programs
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Side Effect Explorer

Real-world side effect reports from the FDA Adverse Event Reporting System (FAERS). Includes both FDA-approved drugs and investigational therapies from active clinical trials. Click any drug to see what patients reported.

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Genetic Testing

Genetic testing can confirm a diagnosis, guide treatment decisions, and identify family members who may be at risk.

The gene pages for RET and NF1 list every condition Orphanet links to the gene and the open trials that name it.

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Trusted Pheochromocytoma Resources

Reputable organizations and medical references for learning more about Pheochromocytoma, including disease registries, foundation resources, and clinical guidelines.

Active Clinical Trials for Pheochromocytoma

Use this Pheochromocytoma clinical trial finder to see the 12 studies recruiting patients and 3 opening soon in the United States and worldwide, with eligibility criteria in plain English. These studies play a critical role in advancing care for endocrine & hormonal conditions and may offer access to treatments not yet widely available. Each trial below is sourced directly from ClinicalTrials.gov, with eligibility criteria translated into plain English to help patients and caregivers evaluate whether a study may be a fit.

TrialsSite mapPipeline timeline

Note: Trial recruitment statuses on ClinicalTrials.gov may not immediately reflect recent FDA decisions, sponsor announcements, or enrollment changes. Always confirm a trial's current status directly with the study coordinator before making plans.

44 active trials worldwide
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RECRUITINGPHASE1Recently updatedNCT06479811

[212Pb]VMT-Alpha-NET in Metastatic or Inoperable Somatostatin-Receptor Positive Gastrointestinal Neuroendocrine Tumors, Pheochromocytoma/Paragangliomas, Small Cell Lung, Renal Cell, and Head and Neck Cancers

Intervention: 68Ga-DOTATATE, [203Pb]VMT-alpha-NET, [212Pb]VMT-alpha-NET

Sponsor: National Cancer Institute (NCI)

Background:

Ages 18 Years – 120 Years1 location
Started Aug 2025Updated todayEst. Jan 2029 (~2y 3m)
RECRUITINGPHASE2Recently updatedNCT04924075

Belzutifan/MK-6482 for the Treatment of Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), Von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Solid Tumors With HIF-2α Related Genetic Alterations (MK-6482-015)

Intervention: Belzutifan

Sponsor: Merck Sharp & Dohme LLC

This is a study to evaluate the efficacy and safety of belzutifan monotherapy in participants with advanced pheochromocytoma/paraganglioma (PPGL), pancreatic neuroendocrine tumor (pNET), von Hippel-Lindau (VHL) disease-associated tumors, advanced wt (wild-type) gastrointestinal s...

Ages 12 Years+84 locations
Started Aug 2021Updated 4 days agoEst. Nov 2032 (~6y 1m)
RECRUITINGPHASE1Recently updatedNCT00004847

Diagnosis of Pheochromocytoma

Intervention: ([18F]-DOPA), ([18F]-6F-DA)

Sponsor: Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

The goal of this study is to develop better methods of diagnosis, localization, and treatment for pheochromocytomas. These tumors, which usually arise from the adrenal glands, are often difficult to detect with current methods. Pheochromocytomas release chemicals called catechola...

Ages 3 Years – 120 Years1 location
Started Mar 2000Updated 1 week agoEst. Nov 2048 (~22y 2m)
NOT YET RECRUITINGPHASE2Recently updatedNCT07743138

STOMP OUT: A Phase 2 Study To Evaluate The Effects Of Ivonescimab In Patients With Unresectable/Metastatic Adrenocortical Carcinoma (ACC) Or Unresectable Pheochromocytoma/Paraganglioma (PPGL)

Intervention: Ivonescimab

Sponsor: M.D. Anderson Cancer Center · Summit Therapeutics

To learn if ivonescimab can help to control previously treated, locally advanced or metastatic ACC or PPGL.

Ages 18 Years+1 location
Started Feb 2027Updated 1 week agoEst. May 2028 (~1y 7m)
RECRUITINGPHASE2Recently updatedNCT03206060

Lu-177-DOTATATE (Lutathera) in Therapy of Inoperable Pheochromocytoma/ Paraganglioma

Intervention: Lu-177-DOTATATE, Ga-68-DOTATATE

Sponsor: National Cancer Institute (NCI)

Background:

Ages 18 Years – 100 Years1 location
Started Oct 2017Updated 1 month agoEst. Jan 2030 (~3y 3m)
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Active trial locations181 cities in the US
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Trial Pipeline

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Data from ClinicalTrials.gov, U.S. National Library of Medicine.
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Patient Communities

Connect with other Pheochromocytoma patients, caregivers, and advocacy groups across Facebook groups, Reddit communities, and YouTube channels. These patient communities offer peer support, shared experiences, caregiver resources, and real-time discussion about Pheochromocytoma treatments, clinical trial participation, and day-to-day disease management.

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Related Endocrine & Hormonal Conditions

Other rare diseases in the endocrine & hormonal category. Patients with Pheochromocytoma may find relevant research, shared treatment pathways, or overlapping clinical trials among these related conditions.

Companies Developing Pheochromocytoma Treatments

6 pharmaceutical companies have Pheochromocytoma in their rare disease portfolio

Frequently Asked Questions About Pheochromocytoma