Research is active.
You have options.
Scientists and doctors around the world are studying new treatments for pheochromocytoma and paraganglioma (PPGL) right now. Below you’ll find every active, enrolling, and recently completed clinical study — in plain language.
1
FDA-approved targeted therapy (2025)
17+
Active or enrolling studies worldwide
8
Distinct treatment approaches in trials
700+
Total enrollment slots across all trials
✓ FDA APPROVED · MAY 2025
Belzutifan (Welireg®) — the first targeted pill approved specifically for advanced 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 HIF-2α, a protein that helps tumors grow — especially relevant for people with SDHx or VHL gene changes. Available now for ages 12+.
→ Ask your doctor: “Am I a candidate for belzutifan?”
Clinical Trials You May Be Able to Join
What is a clinical trial? A research study that tests whether a new medicine is safe and effective. Joining a trial gives you access to cutting-edge treatments, expert medical oversight — and helps future patients too. Participation is always voluntary and you can leave at any time.
HIF-2α Inhibitors
Targeted Pills Blocking Tumor Growth Signals
Phase 2
Opening soon
Casdatifan + Cabozantinib — next-generation HIF-2α inhibitor combo
Casdatifan is a new HIF-2α blocker (same target as belzutifan, potentially more potent) combined with cabozantinib, which cuts off tumor blood supply. Being studied specifically for PPGL at MD Anderson Cancer Center under Dr. Glover. As of May 2026, this trial has not yet been registered on ClinicalTrials.gov — contact PPA for the latest enrollment details.
Who may qualify: Advanced PPGL — eligibility criteria to be confirmed at registration
HIF-2α + TKI combo
MD Anderson Cancer Center
Arcus Biosciences™
NCT # pending ClinicalTrials.gov registration · Arcus Biosciences™ · MD Anderson Cancer Center, Houston TX
ClinicalTrials.gov link will be added once registered — contact pheopara.org for updates
Phase 2
Enrolling now
Belzutifan (Welireg®) — ongoing PPGL cohort at major cancer centers
The trial that led to FDA approval. Continues to enroll patients at major cancer centers. Joining gives access to expert monitoring and the FDA-approved drug.
Who may qualify: Advanced or metastatic PPGL or PanNET, ages 12+, HIF-2α-related tumors (SDHx, VHL)
Oral pill once dailyAges 12+
Cedars-Sinai, U Iowa, Johns Hopkins, Vanderbilt, MDACC, NCI
NCT04924075 · Merck® · Multicenter US
New Targeted Medicines
Oral Pills & Injectable Treatments
Phase 2
Enrolling now
ONC206 / JZP3507 — oral pill targeting PPGL tumor energy production
Works differently from belzutifan — targets DRD2 receptor on tumor cells and disrupts how they produce energy (OXPHOS). A two-stage Phase 2 trial designed specifically for PPGL.
Who may qualify: Unresectable or advanced PPGL, prior treatment or ineligible for surgery, BP ≤150/90 mmHg
Oral pillDRD2 / Imipridone classUCLA Health + US multicenter
NCT07282587 · Jazz Pharmaceuticals® · UCLA Health + sites
Phase 2
Enrolling now
Temozolomide + Olaparib (A021804) — chemo plus DNA repair blocker
Temozolomide is a chemotherapy pill. Olaparib blocks tumor DNA repair. Together they may be more effective — especially for SDHB mutations. Randomized: compares the combination to temozolomide alone.
Who may qualify: Advanced PPGL, any prior treatment ok, no specific gene mutation required
RandomizedSDHB mutation benefitMultiple US sites
NCT04394858 · NCI / NCTN / ACOG · Multiple US sites
Phase 2
Enrolling now
Axitinib — cutting off tumor blood supply at the NIH
Axitinib blocks proteins (VEGFR) that tumors use to grow new blood vessels, starving them of nutrients. Oral pill given twice daily at NIH Clinical Center in Bethesda.
Who may qualify: Advanced or metastatic PPGL, ages 18+, any prior treatment
TKI · Anti-angiogenicOral pillNIH Clinical Center
NCT03839498 · NCI / Antonio Fojo MD · NIH Clinical Center, Bethesda MD
Phase 2
Enrolling now
LAMPARA — Lanreotide monthly injection to slow tumor growth
Lanreotide is a somatostatin analog that can slow how fast tumors grow without necessarily shrinking them. Monthly injection for up to a year with possible extension at the NIH.
Who may qualify: Advanced or metastatic PPGL, ages 18+, any prior treatment
Monthly injectionSomatostatin analogNIH Clinical Center
NCT03946527 · NCI / Ipsen® · NIH Clinical Center, Bethesda MD
Phase 2
Enrolling now
Anlotinib — oral pill before surgery to shrink unresectable tumors
Cuts off tumor blood supply. Used before surgery to shrink tumors that may be too large or complex to safely remove — potentially making surgery possible.
Who may qualify: Locally advanced or unresectable PPGL, ages 18+, no prior anti-vascular TKI
NeoadjuvantOral pillChina-based
NCT05883085 · Beijing Cancer Hospital
Phase 2
Enrolling now
Temozolomide neoadjuvant — chemotherapy pill before surgery
Uses temozolomide before surgery to shrink tumors with the goal of making an unresectable tumor removable.
Who may qualify: Locally advanced or unresectable PPGL, ages 10+
NeoadjuvantOral pillChina-based
NCT05885386 · Beijing Cancer Hospital
Targeted Radiation (Theranostics)
Scan First, Then Treat — Radiation That Seeks Out PPGL Tumors
How theranostics works: A radioactive particle is attached to a molecule that seeks out PPGL tumor cells — delivering targeted radiation while largely sparing healthy tissue. A special imaging scan (DOTATATE PET or MIBG scan) is needed first to confirm your tumors would respond.
Phase 2
Enrolling now
Lutathera® (177Lu-DOTATATE) — FDA-approved targeted radiation studied for PPGL 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. 4 infusions, 2 months apart, at NIH Clinical Center.
Who may qualify: Metastatic or inoperable PPGL, ages 18+, DOTATATE PET scan positive
SSTR2-targeted4 infusionsNIH Clinical Center, Bethesda MD
NCT03206060 · NCI / Frank Lin MD · NIH Clinical Center, Bethesda MD
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 patients, one for post-Lutathera® patients.
Who may qualify: SSTR+ advanced PPGL or NETs, ages 18+ — including post-Lutathera® patients
Alpha particle therapyPost-Lutathera® optionMultiple US sites
NCT06479811 / NCT06427798 · Perspective Therapeutics™ / NCI
Phase 2
Active — long-running
Azedra® (131I-MIBG) — FDA-approved MIBG therapy, ongoing trial at MSKCC
Azedra® is FDA-approved for adults with PPGL. This MSKCC trial continues to enroll patients including children (ages 1+). An MIBG scan first confirms your tumors would respond.
Who may qualify: MIBG-avid recurrent or progressive PPGL, ages 1+, MIBG scan positive
MIBG-targetedAges 1+Memorial Sloan Kettering, New York NY
NCT00107289 · MSKCC / Lantheus® · New York, NY
Phase 1
Enrolling now (Japan)
[211At]MABG — first-ever alpha-emitting MIBG analog trial
An experimental alpha-emitting version of MIBG therapy — potentially more powerful than standard MIBG. The first-ever human trial of this approach, currently enrolling in Japan.
Who may qualify: Metastatic or unresectable PPGL, ages 18+, MIBG scan positive (Japan sites only)
Alpha therapyFirst-in-humanJapan
jRCT2021220012 · Investigator-initiated · Japan
Immunotherapy & Novel Biologics
Harnessing the Immune System
Phase 2
Enrolling now
Penpulimab (Anti-PD-1) — immune checkpoint therapy after prior treatment
Releases a “brake” on the immune system, allowing it to better recognize and attack PPGL tumor cells. For patients whose PPGL has progressed after prior treatment.
Who may qualify: Metastatic PPGL, ages 18+, failed prior systemic treatment, no prior anti-PD-1/PD-L1
Anti-PD-1Post-treatmentChina-based
NCT05885399 · Beijing Cancer Hospital / Chia Tai Tianqing
Phase 2
Enrolling now
Anlotinib + Penpulimab — first-line combination for advanced PPGL
Combines anlotinib (cuts off tumor blood supply) with penpulimab (immune checkpoint) as a first treatment for advanced PPGL. Tests whether a two-pronged attack is more effective.
Who may qualify: Advanced PPGL, ages 18+, treatment-naïve, no prior anti-PD-1/PD-L1
TKI + Anti-PD-1 comboFirst-lineChina-based
NCT06429397 · Beijing Cancer Hospital / Chia Tai Tianqing
Phase 1/2
Enrolling now
EO2401 Vaccine + Nivolumab — first therapeutic vaccine ever studied in PPGL
Trains the immune system to recognize and attack tumor cells, combined with nivolumab to keep that response active. For PPGL and adrenal cancer (ACC) patients. This is the first therapeutic vaccine ever studied specifically in PPGL.
Who may qualify: Advanced or metastatic PPGL or ACC, ages 18+
Tumor neoantigen vaccineAnti-PD-1 combo
First PPGL vaccine trialMD Anderson + sites
NCT04187404 · Enterome™ / MD Anderson Cancer Center
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 — the highest rate of any cancer type. This early-stage antibody-drug conjugate (ADC) trial delivers a cancer-killing payload directly to DLK1-expressing tumor cells.
Who may qualify: Advanced PPGL, NETs, ACC, or SCLC, ages 18+, ECOG ≤2, exhausted standard options
Antibody-drug conjugateDLK1-targeted
Dose-finding studyNIH / NCI
Phase 1 FIH · ADC Therapeutics™ / NCI · NIH Clinical Center, Bethesda MD
Observational
Natural History Study — Gateway to NCI Treatment Trials
Observational
Enrolling now
NCI Natural History of Neuroendocrine Neoplasms — gateway to NCI treatment trials
Not a treatment trial — collects tissue and blood samples and follows patients over time. Enrolling here is often the entry point that qualifies you for NCI treatment trials listed on this page.
Who may qualify: Any patient with a neuroendocrine neoplasm including PPGL, any stage, any prior treatment
No treatment involvedGateway to NCI trials
Unlimited enrollmentNIH Clinical Center
NCT05237934 · NCI / CCR · NIH Clinical Center, Bethesda MD
Recently Completed
Results That Are Changing PPGL Care
Completed
Published — Lancet 2024
FIRSTMAPPP — Sunitinib vs. Placebo in Malignant Progressive PPGL
First ever randomized placebo-controlled trial in PPGL. Result: 8.9 months median progression-free survival vs. 3.6 months for placebo. Now informs standard of care.
TKI vs placeboPublished Lancet 2024Gustave Roussy (EU-led)
NCT02602080 · Gustave Roussy / INCa · Eric Baudin MD
Completed
Published — Lancet Oncology 2024
The Natalie Trial — Cabozantinib in Metastatic PPGL (Jimenez et al.)
Phase 2 trial of cabozantinib in advanced PPGL at MD Anderson Cancer Center. Published Lancet Oncology 2024. Established cabozantinib as an active agent in PPGL.
TKI · VEGFR/MET/AXLPublished Lancet Oncology 2024MD Anderson Cancer Center
NCT02302833 · NCI / Jaydira Del Rivero MD · MD Anderson Cancer Center
Not sure where to start?
Your situation helps determine which trials you may qualify for. Use this as a starting point to talk to your doctor.
Advanced or metastatic PPGL
Ask about belzutifan (Welireg®, approved now), Lutathera® (if DOTATATE PET positive), or temozolomide + olaparib — especially if you have an SDHB mutation.
No treatment yet
Ask about the anlotinib neoadjuvant trial (if surgery is the goal) or the lanreotide LAMPARA trial (if your tumor is slow-growing).
Known gene mutation (SDHx, VHL, RET)
Share genetic results with trial coordinators — it speeds up eligibility screening. Belzutifan (Welireg®), ONC206, and casdatifan are especially relevant for SDHx tumors.
Already had Lutathera®
Ask about VMT-α-NET — the next-generation alpha particle therapy specifically designed for post-Lutathera® patients.
Your next steps
1
Ask your doctor: “Are there clinical trials I qualify for?” — share the trial numbers listed on this page.
2
Search all PPGL trials directly at
clinicaltrials.gov or use the links on each card above.
3
Ask about DOTATATE PET scan and genetic testing if you haven’t had them — both open up more options.
4
Connect with the PPA patient community at
pheopara.org — others have navigated this and can share what helped.
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