Active and recruiting clinical trials for pheochromocytoma and paraganglioma (PPGL). Sourced from the National Cancer Institute (NCI) and the NCI Center for Cancer Research (CCR). Some trials are held at NCI-Designated Cancer Centers and may be supported by other organizations.
Learn more about types and phases of clinical trials, what to expect, or use the NCI’s Steps to Find a Clinical Trial.
Jonathan Pascual is a Nurse Practitioner living with metastatic paraganglioma who dedicates himself to patient education and raising critical funds for the PPGL community. Hear what he has to say about clinical trials.
Jonathan also invites you to join him at JP’s Backyard Ultra/Hike — The Final Edition, September 26, 2026 in Napa, California. Walk, hike or run alongside the PPGL community and help fund the research and patient resources this page exists to support.
ONC206 (JZP3507) is an oral imipridone that works as a ClpP agonist and DRD2 antagonist — disrupting how tumor cells produce energy. This is a two-stage Phase 2 trial designed specifically for PPGL. Stage 1 uses 150mg given twice daily on three consecutive days per week; Stage 2 refines dosing based on early signals. All participants receive ONC206 (no placebo).
NCT07282587 · NCI-2026-00132 · 10 study sites (8 active, 2 projected) · Sponsor: Jazz Pharmaceuticals®
Casdatifan is an investigational HIF-2α inhibitor (same target as belzutifan/Welireg®, potentially more potent) being studied in combination with cabozantinib, a TKI that blocks tumor blood supply. Arcus’s existing PEAK-1 Phase 3 trial evaluates casdatifan + cabozantinib in kidney cancer (ccRCC), establishing the combination’s safety profile. A dedicated PPGL trial at MD Anderson Cancer Center is anticipated based on PPA’s engagement with Arcus; no investigator of record has been announced. HIF-2α inhibition is scientifically well-supported in PPGL, particularly for SDHx and VHL-related tumors.
NCT # not yet assigned · Arcus Biosciences™ · MD Anderson Cancer Center, Houston TX · Investigator of record TBD · Contact PPA for updates
First-in-human, open-label, multicenter study of CRN09682, an antibody-drug conjugate that binds somatostatin receptor type 2 (SST2) on the tumor surface and releases the cell-killing payload MMAE inside the tumor cell. The dose escalation phase finds the maximum tolerated dose; an expansion phase then enrolls additional patients at that dose to look for early signs of tumor response. Tumors must show somatostatin receptor uptake on SSR imaging (such as a DOTATATE PET scan) to qualify.
NCT07129252 · Started November 2025, estimated completion August 2029 · Recruiting in the United States and Spain · U.S. sites include UCSF (San Francisco, CA), City of Hope (Duarte, CA), UC Irvine Health (Orange, CA), Newport Beach, CA — additional sites opening
Belzutifan is an oral HIF-2α inhibitor already approved (as Welireg®) for VHL-associated tumors and advanced kidney cancer. This global Phase 2 study includes a dedicated advanced PPGL cohort and measures how many patients’ tumors shrink, assessed by independent central review. Because belzutifan is already an approved medicine, this is one of the most likely paths to a PPGL treatment patients can eventually get outside a trial — particularly relevant for SDHx- and VHL-related disease, where HIF-2α is the driver.
NCT04924075 · MK-6482-015 · Sites across North America, Europe, and Asia-Pacific — check ClinicalTrials.gov for the site nearest you
ADCT-701 is an antibody-drug conjugate (ADC) that delivers a cancer-killing payload directly to DLK1, a protein found at high levels in pheochromocytoma and paraganglioma tumors in pre-clinical studies. Very early-stage safety and dose-finding study. For patients who have exhausted standard treatment options.
NCT06041516 · IRB001547 · NIH Clinical Center, Bethesda, Maryland
Lutathera® is FDA-approved for some gut tumors. This NCI trial studies it specifically for PPGL. A DOTATATE PET scan first confirms whether your tumors would respond. Given as 4 infusions, 8 weeks apart, at the NIH Clinical Center.
NCT03206060 · 17-C-0087 · NCI-2018-03458 · NIH Clinical Center
The company-sponsored, multicenter version of the [212Pb]VMT-α-NET program — the one with sites at cancer centers across the United States rather than only at NIH. It is a first-in-human dose-finding and dose-expansion study in up to roughly 300 adults with unresectable or metastatic SSTR2-expressing tumors who have not had prior peptide receptor radionuclide therapy (PRRT). Dose-finding enrolled mainly pancreatic and digestive-tract NETs; the sponsor has stated the study carries an enrollment allowance for pheochromocytoma/paraganglioma. Treatment is given as up to four administrations about 8 weeks apart. Interim data reported an objective response rate of 39% in the 5.0 mCi cohort with no dose-limiting toxicities.
NCT05636618 · Sponsor: Perspective Therapeutics™ · Multicenter, United States
Studies [212Pb]VMT-α-NET, an alpha-emitting radioparticle targeting SSTR2 receptors. Alpha particles deliver denser, shorter-range radiation than standard PRRT (Lutathera®). This is an NIH intramural Phase 1 study at the NIH Clinical Center; alongside PPGL it also enrolls SSTR-positive gastrointestinal neuroendocrine, small cell lung, renal cell and head and neck cancers. It is the companion study to the post-RLT trial below, for patients who have not previously had targeted radioligand therapy.
NCT06479811 · IRB001709 · NCI-2023-06702 · NIH Clinical Center, Bethesda, Maryland
The post-Lutathera® VMT-α-NET trial. Specifically designed for patients who have already received prior targeted radioligand therapy (e.g. Lutathera®). Alpha particles may be more effective for tumors that have progressed after standard PRRT.
NCT06427798 · IRB001711 · NCI-2024-04696 · NIH Clinical Center
RYZ401 is an investigational (not yet approved) targeted radiopharmaceutical that carries the alpha-emitting isotope actinium-225 to cells with somatostatin receptors (SSTRs), the same type of target used by Lutathera®. This open-label Phase 1 study has dose-escalation and dose-expansion parts and aims to find a safe dose and early signs of effectiveness. About 104 participants are planned.
NCT07165132 · UCLA protocol 25-2220 · Sponsor: RayzeBio, Inc. · Locations listed: Iowa City, IA; Lexington, KY; St. Louis, MO; Salt Lake City, UT; UCLA, Los Angeles, CA — site lists change, so check ClinicalTrials.gov for current sites
Ivonescimab is an investigational bispecific antibody that blocks both PD-1 (an immune checkpoint) and VEGF (a driver of tumor blood supply) in a single molecule — combining immunotherapy and anti-angiogenic activity. This Phase 2 study evaluates it in unresectable or metastatic adrenocortical carcinoma and in unresectable PPGL. Registered August 2026 and listed as not yet recruiting — confirm site activation before making plans.
NCT07743138 · Sponsor: M.D. Anderson Cancer Center with Summit Therapeutics™ · First posted August 2026
An open-access Memorial Sloan Kettering protocol offering targeted radiotherapy with I-131 MIBG in neuroblastoma and malignant chromaffin cell tumors (pheochromocytoma, paraganglioma) that are resistant to standard treatment. This is conventional I-131 MIBG under a research protocol — it is not commercial Azedra®. An MIBG scan first confirms your tumors would respond. Status verified by the site in May 2026.
NCT00107289 · MSKCC 04-148 · PI: Ellen M. Basu, MD, PhD · Memorial Sloan Kettering Cancer Center, New York NY
Studies a new PET scan tracer that detects PARP-1 in tumors. Could help identify which PPGL patients would benefit most from PARP inhibitor treatments like olaparib. University of Pennsylvania.
NCT05636540 · NCI-2023-05514 · University of Pennsylvania / Abramson Cancer Center
Alliance A021804 — a PPGL-specific randomized Phase 2 trial comparing dose-dense temozolomide plus olaparib to pulse-dose temozolomide alone in advanced (metastatic or unresectable) pheochromocytoma and paraganglioma. Enrollment is now complete. Results are expected to be analyzed and reported. This study may provide important data on the temozolomide and olaparib combination strategy, particularly for patients with SDHB mutations.
NCT04394858 · Alliance A021804 · Co-PIs: Jaydira Del Rivero, M.D. (NCI) & Kimberly J. Perez, M.D. (Dana-Farber Cancer Institute)
Studies pembrolizumab (anti-PD-1 immunotherapy) across multiple rare tumor types including PPGL. Currently temporarily closed to new enrollment — check the NCI listing for updates on reopening.
NCI-2016-00545 · MD Anderson Cancer Center, Houston TX
A second MIBG therapy trial for MIBG-avid tumors including PPGL. Currently temporarily closed to both new enrollment and intervention. Check ClinicalTrials.gov for the most current status.
NCI-2016-01994 · NCT02378428
An NCI/NIH natural history study collecting tissue, blood, and clinical data from patients with neuroendocrine neoplasms including PPGL. Enrolling in this study can provide a pathway into other NCI treatment research at the NIH Clinical Center. No treatment involved.
NCT05237934 · NCI / CCR · NIH Clinical Center, Bethesda, Maryland
A PPGL-specific genetic study collecting tissue and blood samples to analyze hereditary causes including SDHx, VHL, RET, NF1, and other mutations. Open to patients with PPGL and their family members. 6 locations.
NCI-2025-03501 · 6 locations
Long-running NCI natural history study of VHL disease and associated tumors including PPGL. Particularly relevant for PPGL patients with VHL germline mutations. NIH Clinical Center.
NCI-2018-02236 · NIH Clinical Center, Bethesda, Maryland
A PPGL genetic study led by Dr. Patricia L.M. Dahia at UT Health San Antonio — one of the world’s leading PPGL genetics researchers, who identified the TMEM127 susceptibility gene and has contributed to the discovery and characterization of several other PPGL susceptibility genes. Collects samples and data from patients with PPGL and family members to identify hereditary mutations and understand how genetics drives tumor development. Separate from and complementary to the NCI genetic study listed above — enrolling at UT Health San Antonio.
NCT03160274 · PI: Patricia L.M. Dahia, MD, PhD · UT Health San Antonio, Texas
National registry for children and adolescents with cancer including PPGL. Collects biospecimens, clinical data, and patient-reported outcomes. Available at 239 locations nationwide.
NCI-2014-02057 · 239 locations nationwide
Penpulimab is an anti-PD-1 immune checkpoint inhibitor that may help the immune system recognize and attack PPGL tumor cells. This Phase 2 trial studies it in patients with metastatic PPGL who have already received and progressed on other systemic therapies. Given intravenously every 3 weeks. Beijing Cancer Hospital, China.
NCT05885399 · Beijing Cancer Hospital, Beijing, China
Compares two PET scan tracers for detecting PPGL tumors. Al18F-NOTA-LM3 is a new SSTR-targeted imaging agent that may offer advantages over the standard DOTATATE scan. Patients receive both scans and results are compared. Peking Union Medical College Hospital, Beijing.
NCT07288931 · Peking Union Medical College Hospital, Beijing, China
One of the most practical European routes to radioligand therapy for PPGL. Lutathera® is only approved for digestive-tract neuroendocrine tumors, which leaves PPGL patients unable to access PRRT through normal channels in many countries. This trial treats exactly those patients — PPGL, meningioma, lung/ovarian/renal NETs, and other SSTR-positive cancers outside the Lutathera® indication — with 177Lu-DOTATOC, a closely related radioligand, and measures tumor response and safety. IRST IRCCS “Dino Amadori,” Meldola, Italy.
NCT06045260 · Istituto Romagnolo per lo Studio dei Tumori “Dino Amadori” IRST IRCCS, Meldola, Italy
Nearly all PPGL treatment trials are adults-only, which leaves families of children and teenagers with almost nowhere to turn. This is one of the very few exceptions: it combines Lutathera® radioligand therapy with olaparib, a PARP inhibitor that blocks tumor cells from repairing the DNA damage the radiation causes, so the two may work better together than either alone. Paraganglioma and pheochromocytoma are named among the eligible tumor types. Fundación de investigación HM, Spain.
NCT06607692 · LUPARPED · Fundación de investigación HM, Spain
Directly relevant to anyone taking or considering belzutifan. Participants take belzutifan 120mg daily for five days while researchers measure how their adrenaline and noradrenaline production changes — tracking tyrosine, the substance that starts the catecholamine chain — before, during, and after. The answer matters practically: it shapes how symptoms and blood-pressure risk should be monitored on this drug, and whether catecholamine levels can be used to tell early on whether it is working. Cambridge University Hospitals NHS Foundation Trust.
NCT07680205 · Cambridge University Hospitals NHS Foundation Trust with the University of Cambridge, United Kingdom
Studies the role of the immune system in PPGL, comparing patients with hereditary vs. sporadic (non-inherited) tumors. A blood draw study — no treatment involved. Results aim to identify new immune-based therapy targets for PPGL. Radboud University Medical Center, Nijmegen, Netherlands.
NCT06444607 · Radboud University Medical Center, Nijmegen, Netherlands
A large European multicenter prospective cohort study following patients with PPGL (newly diagnosed, history of PPGL, or carriers of PPGL susceptibility gene mutations) over time. Studies follow-up care, genetic subtyping, quality of life, and long-term outcomes. Active sites in Zurich (Switzerland), Würzburg, and Dresden (Germany). Enrollment and observation run over roughly 10 years. This is an observational cohort and registry — participants are not randomized and receive no study treatment.
NCT03344016 · University Hospital Zurich (Switzerland) · University Hospital Würzburg (Germany) · University Hospital Dresden (Germany)
A randomized Phase 4 study examining whether alpha-adrenergic blockade — standard preparation given before surgery for pheochromocytoma — can safely be omitted in patients whose blood pressure is normal (normotensive). This question matters because alpha blockade carries its own side effects. Seoul National University Hospital, South Korea.
NCT05702944 · Seoul National University Hospital, Seoul, South Korea
As of August 25, 2026, our search found no active, recruiting PPGL-specific clinical trials registered on ClinicalTrials.gov in South America, the Middle East, or Central America. PPGL research expertise exists across these regions — particularly in Brazil and Israel — but no open enrollment studies appear in current registries. This may change as the global trial pipeline grows.
Your situation helps determine which trials may be right for you. Use this as a starting point when talking to your doctor.
Ask about ONC206 (oral pill — Jazz), or the NIH Lutathera® trial if your tumors are DOTATATE PET positive and your disease is sporadic or SDHx-related (VHL, NF1 and RET carriers are not eligible for that study). Belzutifan (Welireg®) is now FDA-approved — ask your doctor if you are a candidate. A newly registered Phase 2 of ivonescimab (NCT07743138, MD Anderson) is expected to open — ask whether it is enrolling yet. RYZ401 (NCT07165132) is a newer alpha-emitting radiopharmaceutical for SSTR-positive neuroendocrine tumors — RayzeBio has confirmed PPGL patients may be eligible, so ask the study team about your case. Note: temozolomide + olaparib enrollment is complete; results are pending.
Ask about the post-RLT VMT-α-NET trial (NCT06427798, Dr. Lin at NIH) — specifically designed for patients who have previously had targeted radioligand therapy. The two PRRT-naïve VMT-α-NET studies (NCT06479811 at NIH, NCT05636618 at multiple US sites) exclude anyone who has already had radioligand therapy. Note that RYZ401 (NCT07165132) excludes anyone who has had prior Lu-177.
Share your genetic results with trial coordinators — genotype decides eligibility on several studies. Belzutifan and ONC206 are especially relevant; note the NIH Lutathera® trial (NCT03206060) accepts sporadic and SDHx disease but excludes VHL, NF1 and RET. The VHL natural history study (NCI-2018-02236) is open to VHL patients.
Ask Dr. Del Rivero’s team at NIH about ADCT-701 (NCT06041516), the DLK1-targeted ADC. DLK1 is highly expressed in PPGL tumors in pre-clinical studies. Contact: anna.rivero@nih.gov or +1 240-858-7946.
Project: Every Child (NCI-2014-02057) is at 239 locations nationwide. Azedra® (MIBG, MSKCC) enrolls PPGL patients ages 1–21. Ask your pediatric oncologist about eligibility for any of these trials.
The PPGL genetic study (NCI-2025-03501) and VHL natural history study (NCI-2018-02236) accept patients with no treatment required and directly advance the science that leads to future treatments.
Ask your doctor: “Are there clinical trials I qualify for?” — share the trial numbers listed on this page.
Browse all NCI PPGL trials at cancer.gov/clinicaltrials or use the NCI and ClinicalTrials.gov links on each card above.
Ask about DOTATATE PET scan and genetic testing if you haven’t had them — both open up more treatment and trial options.
Connect with the PPA patient community at pheopara.org — others have navigated this and can share what helped them find the right trial or specialist.
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