Pheochromocytoma & paraganglioma research
Research is active.
You have options.
Scientists and doctors around the world are studying new treatments for pheochromocytoma and paraganglioma (PPGL) right now. This page shows what's being tested — and how you might be able to participate.
1
Treatment FDA-approved in 2025
6
Active, enrolling treatment trials
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Treatment approaches being explored
Already approved — available now. Ask your doctor about this today.
FDA approved · May 2025
Belzutifan (brand name: Welireg®) — a daily pill for advanced PPGL
The first targeted oral medicine approved specifically for advanced or metastatic PPGL. In the clinical trial, 26 out of 100 patients saw their tumors shrink, and 85 out of 100 had their disease stay stable or improve. Works by blocking a protein (HIF-2α) that helps tumors grow — especially relevant for people with SDHx or VHL gene changes. Available for ages 12+.
→ Ask your doctor: “Am I a candidate for belzutifan?”
Clinical trials you may be able to join
A clinical trial is a research study that tests whether a new medicine is safe and effective. Joining gives you access to cutting-edge treatments and expert medical oversight. Participation is always voluntary and you can leave at any time. All trials below are verified actively recruiting as of July 2026.
HIF-2α inhibitors
Phase 2
Enrolling now
Belzutifan (Welireg®) — ongoing PPGL cohort at major cancer centers
The trial that led to FDA approval continues to enroll. Joining gives access to expert monitoring and the FDA-approved drug at leading cancer centers nationwide.
Advanced / metastatic PPGL
Ages 12+
SDHx or VHL
NCT04924075 · Merck · Cedars-Sinai, U Iowa, Johns Hopkins, Vanderbilt, MDACC, NCI
Learn more →
New targeted medicines
Phase 2
Enrolling now
ONC206 / JZP3507 — oral pill targeting PPGL tumor energy production
Works differently from belzutifan — targets the DRD2 receptor on tumor cells and disrupts how they produce energy. A two-stage Phase 2 trial designed specifically for PPGL.
Unresectable / advanced PPGL
Oral pill
No prior tx required
NCT07282587 · Jazz Pharmaceuticals · UCLA Health + US multicenter
Learn more →
Targeted radiation (theranostics)
These treatments attach a radioactive particle to a molecule that seeks out PPGL tumor cells — delivering targeted radiation while sparing healthy tissue. A special scan confirms your tumors would respond before treatment begins.
Phase 2
Enrolling now
Lutathera® (177Lu-DOTATATE) — targeted radiation at the NIH
Already FDA-approved for some gut tumors. This NCI trial studies it specifically for PPGL. A DOTATATE PET scan confirms if your tumor would respond. Four infusions, 8 weeks apart, at NIH. No cost to participate.
Metastatic or inoperable
DOTATATE scan required
Ages 18+
NCT03206060 · NCI · NIH Clinical Center, Bethesda MD
Learn more →
Phase 1/2
Enrolling now
VMT-α-NET — next-generation alpha particle therapy, including post-Lutathera® option
Uses alpha particles — denser radiation that may work better for resistant tumors, including those that have already had Lutathera®. Two separate trials: one for first-time, one for post-Lutathera® patients.
SSTR+ PPGL
Post-Lutathera® option
Ages 18+
NCT05636618 / NCT06479811 · Perspective Therapeutics / NCI · Multiple US sites
Learn more →
Novel biologics
Phase 1
Enrolling now
ADCT-701 — “guided missile” antibody targeting DLK1 on PPGL tumor cells
DLK1 is found on nearly 100% of PPGL tumors tested. This first-in-human antibody-drug conjugate (ADC) delivers a cancer-killing payload directly to DLK1-expressing tumor cells at the NIH Clinical Center. No cost to participate.
Advanced PPGL, NETs, ACC
Ages 18+
NIH / NCI
NCT06041516 · NCI / ADC Therapeutics · NIH Clinical Center, Bethesda MD
Learn more →
Phase 2
Opening soon
Casdatifan + Cabozantinib — next-generation HIF-2α inhibitor + anti-angiogenic combo
A potent new HIF-2α blocker combined with cabozantinib, which cuts off tumor blood supply. Being studied specifically for PPGL at MD Anderson Cancer Center. NCT registration pending as of May 2026 — contact PPA for the latest enrollment details.
Advanced PPGL
MD Anderson Cancer Center
Arcus Biosciences
NCT # pending · Arcus Biosciences · MD Anderson Cancer Center, Houston TX
Contact PPA for updates →
Not sure where to start? Here’s a simple guide.
If your PPGL is advanced or has spread
Ask your doctor about belzutifan (Welireg®, approved now), Lutathera® if your tumors are DOTATATE PET positive, or ONC206 if your PPGL has progressed on prior treatment.
If you have a gene mutation (SDHx, VHL)
Share your genetic test results with trial coordinators — it speeds up eligibility screening. Belzutifan, ONC206, and casdatifan are especially relevant for SDHx-related tumors.
If you’ve already had Lutathera®
Ask about VMT-α-NET — the next-generation alpha particle therapy designed specifically for post-Lutathera® patients.
If you’ve exhausted standard options
Ask about ADCT-701 at the NIH Clinical Center — it targets DLK1, found on nearly all PPGL tumors. There is no cost to participate in NIH trials.
Your next steps
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Ask your doctor: “Are there clinical trials I qualify for?” — share this page or the NCT numbers listed here.
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Ask about a DOTATATE PET scan and genetic testing if you haven’t had them — both open up more options.
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Connect with the PPA patient community at
pheopara.org — others have navigated this and can help.
Clinical trial finder
Use the tool below to find a trial that may be right for you.
About clinical trials
The World Health Organization (WHO) defines a clinical trial as any research study that prospectively assigns human participants or groups of humans to one or more health-related interventions to evaluate the effects on health outcomes.
Participation in a clinical trial is an important tool in the development of better health outcomes for those with pheochromocytoma and paraganglioma (PPGL), especially those with progressive or metastatic disease. In this section, we explain the types of clinical trials and provide tools to help you decide if a clinical trial may be right for you.
The Pheo Para Alliance does not endorse any particular research study and is not responsible for the accuracy of the information provided by the investigator or the accuracy of the information provided by the searchable databases.
Types of clinical trials
Diagnostic trials
Determine better tests or procedures for diagnosing a particular disease or condition.
Natural history studies
Provide valuable information about how disease and health progress over time.
Prevention trials
Look for better ways to prevent disease or prevent disease from returning.
Quality of life trials
Explore ways to improve comfort and quality of life for people with chronic illness.
Screening trials
Test the best way to detect certain diseases or health conditions.
Treatment trials
Test new treatments, drug combinations, or new approaches to surgery or radiation therapy.
Phases of clinical trials
Phase I
20–80 people. Tests safety and identifies side effects.
Phase II
100–300 people. Evaluates effectiveness and further safety.
Phase III
1,000–3,000 people. Confirms effectiveness, monitors side effects, compares to standard treatment.
Phase IV
Post-approval. Tracks long-term safety, risks, benefits, and optimal use.
To learn more about clinical trials, visit the American Cancer Society’s “Clinical Trials: What You Need to Know”.
The Pheo Para Alliance does not endorse any particular research study and is not responsible for the accuracy of information provided by investigators or searchable databases. This page is for informational purposes only and does not constitute medical advice. Trial eligibility varies by individual — always consult your care team before making treatment decisions. Trial status changes frequently; verify current status at
clinicaltrials.gov. Welireg® and belzutifan are registered trademarks of Merck Sharp & Dohme LLC. Lutathera® is a registered trademark of Novartis AG. Jazz Pharmaceuticals® is a registered trademark of Jazz Pharmaceuticals plc. Perspective Therapeutics™ and Arcus Biosciences™ are trademarks of their respective owners. Sources: ClinicalTrials.gov, FDA.gov, NCI, pheopara.org. Updated July 2026.
