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.
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 · 7 locations · 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 under Dr. Glover is anticipated based on PPA’s engagement with Arcus. 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 · Contact PPA for updates
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
Studies [212Pb]VMT-α-NET, an alpha-emitting radioparticle targeting SSTR2 receptors. Alpha particles deliver denser, shorter-range radiation than standard PRRT (Lutathera®). This trial is for patients who have not previously had targeted radioligand therapy. 13 locations nationwide.
NCT06479811 · IRB001709 · NCI-2023-06702 · 13 locations
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
This Memorial Sloan Kettering research study investigates I-131 MIBG therapy in recurrent or progressive PPGL. It is a research protocol — not a reference to commercial Azedra® availability. An MIBG scan first confirms your tumors would respond.
NCT00107289 · NCI-2016-00955 · Memorial Sloan Kettering Cancer Center
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
Phase 2 randomized trial comparing temozolomide plus olaparib to temozolomide alone in advanced neuroendocrine cancers including PPGL. 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 · Trial ID: 000303 · Co-PIs: Jaydira Del Rivero, M.D. (NCI) & Kimberly Perez, M.D. (Dana-Farber)
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 and co-discoverer of SDHB, HIF2A/EPAS1, and TMEM127 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 · Started August 2024
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). Randomizes patients to specialist follow-up vs. standard care follow-up.
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 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 Lutathera® if your tumors are DOTATATE PET positive. Belzutifan (Welireg®) is now FDA-approved — ask your doctor if you are a candidate. 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.
Share your genetic results with trial coordinators — it speeds up eligibility screening. Belzutifan and ONC206 are especially relevant. 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.
PPA is a small nonprofit run by a two-person staff. Your gift funds patient resources like this one, research advocacy, and our Centers of Excellence network — all at no cost to patients.
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