Advanced Pheochromocytoma and ParagangliomaPhase 2NCT07282587
Study of ONC206 (JZP3507) in Advanced Pheochromocytoma and Paraganglioma
A Phase 2 study testing ONC206, ONC206: Dose 1 and ONC206: Dose 2 in people with Advanced Pheochromocytoma and Paraganglioma and PCPG, run by Jazz Pharmaceuticals at 9 sites in United States.
Trial summary
This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Advanced Pheochromocytoma and Paraganglioma, PCPG
- What is being tested?
- ONC206, ONC206: Dose 1 and ONC206: Dose 2
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 9 sitesUnited States
- Study dates
- 2 Jan 2026to 15 Dec 2028
- Phase
- Phase 2
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Has histologically confirmed pheochromocytoma or paraganglioma that is unresectable as determined by the Investigator.
- Has failed, is not a candidate for, or has declined standard of care treatment for PCPG. There is no limit on the number of prior systemic therapies.
- Must have measurable disease per RECIST v1.1, as assessed by the Investigator.
- Has adequately controlled blood pressure defined as blood pressure ≤150/90 mmHg and with no change in antihypertensive medications (for participants with concomitant hypertension) for at least 14 days before the first dose of study treatment.
- Is ≥18 years of age.
- Is able to swallow oral tablets.
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- Has an Eastern Cooperative Oncology Group (ECOG) Performance Status of 0 to 2, assessed within 7 days before the first dose of study treatment.
- Has laboratory test results meeting the following parameters within 14 days before the first dose of study treatment
- Has an expected survival of at least 12 weeks, as predicted by the physician.
- Has pharmacologic control of catecholamine-associated symptoms if participant has functional disease.
Who cannot join
- Has known hypersensitivity to ONC206 or any excipient used in the ONC206 study treatment formulation.
- Has active cardiac disease/condition including any of the following:
- Corrected QT interval (QTc) >480 msec (based on the mean from triplicate electrocardiogram [ECGs] performed during Screening).
- History of documented congestive heart failure (New York Heart Association function classification III-IV).
- Unstable angina, acute myocardial infarction, or arterial bypass or percutaneous transluminal coronary angioplasty within 6 months before the first dose of study treatment.
- Has previous exposure to ONC206 or dordaviprone (ONC201) from any source.
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- Has a known additional malignancy that is progressing or has required active treatment within the past 2 years. Exceptions include participants with basal cell carcinoma of the skin, squamous cell carcinoma of the skin that has undergone potentially curative therapy or in situ cervical cancer, or Von Hippel-Lindau disease-associated tumors that do not require immediate surgery or intervention.
- Has received any of the following interventions within the specified time periods before the first dose of study treatment or plans to receive any of the following interventions during study participation:
- a. Any prior anticancer therapy or investigational agents within 4 weeks or 5 half-lives, whichever is shorter. Note: Denosumab and zoledronic acid are permissible.
- i. Any treatment with somatostatin analog or lanreotide within 21 days before the baseline Positron Emission Tomography (PET) scan.
- b. Strong cytochrome P450 (CYP) inhibitors within 14 days. c. Strong CYP inducers within 14 days. d. Any radiotherapy within 14 days. e. Any major surgery, open biopsy or significant traumatic injury within 1 month (30 days).
- Is pregnant, breastfeeding, or planning to become pregnant while receiving study treatment or within 3 months after the last dose.
- Has uncontrolled intercurrent illness or any other medical, psychiatric, or social condition that, in the opinion of the Investigator, may interfere with participant safety or the ability to comply with study requirements.
- Has unresolved toxicities from previous locoregional, systemic, or any other therapies, defined as toxicities (other than Grade ≤2 neuropathy or alopecia) not yet resolved to the National Cancer Institute Common Terminology Criteria for Adverse Events Grade ≤1, or baseline and considered clinically significant; consult with Medical Monitor.
- Has an active infection that requires systemic therapy.
Trial locations and contacts
9 sites in 1 country. Contact the study team or a site near you to ask about taking part.
9 sites in 1 country
United States9 sites, 9 recruiting
- Recruiting
U of Michigan
Ann Arbor, Michigan, 48109
- Recruiting
U of Colorado
Aurora, Colorado, 80045
- Recruiting
UCLA
Los Angeles, California, 90095
- Not yet recruiting
Columbia University Medical Center
New York, New York, 10032
- Recruiting
Stanford
Palo Alto, California, 94604
- Recruiting
PENN
Philadelphia, Pennsylvania, 19104
- Recruiting
Mayo-Rochester
Rochester, Minnesota, 55905
- Recruiting
UTAH
Salt Lake City, Utah, 84112
- Recruiting
Washington University
St Louis, Missouri, 63110
About this study
Official title: A Phase 2 Study of ONC206 in Advanced Pheochromocytoma and Paraganglioma
This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).
Read the detailed description
Two-Stage Study to evaluate ONC206 as monotherapy doses:
Stage 1: Participants will receive 150mg ONC206 twice daily, on three consecutive days per week (BID TIW) in each 28-day cycle.
- Part A: Initial number of participants will be monitored.
- Part B: If a set number of participants have a response, then more participants will be enrolled and treated.
Stage 2: If Stage 1 meets the planned response, the study will proceed, and participants will be randomized 1:1 to receive 1 of 2 ONC206 dose levels.
- Conditions
- Advanced Pheochromocytoma and Paraganglioma, PCPG
- Being tested
- ONC206, ONC206: Dose 1, ONC206: Dose 2
- Keywords
- PCPG, ONC206, JZP3507
- Medical subject headings
- Paraganglioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ONC206Drug
150 mg BID TIW
ONC206: Dose 1Drug
ONC206: Dose 2Drug
| Study group | Role | What they receive |
|---|---|---|
| Stage 1 Participants | Experimental | 150 mg ONC206 BID TIW Receives: Drug: ONC206 |
| Stage 2: Dose 1 | Experimental | Participants receiving ONC206 at dose (To be Determined [TBD] post stage 1). Receives: Drug: ONC206: Dose 1 |
| Stage 2: Dose 2 | Experimental | Participants receiving ONC206 at dose (TBD post stage 1). Receives: Drug: ONC206: Dose 2 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1
ORR defined as the number of participants with a confirmed complete response (CR) or partial response during the study, as per RECIST v1.1.
Measured: Up to 36 months.
Secondary outcomes (18)
Duration of Response (DOR)
Measured: Up to 36 months.
Time to Response (TTR)
Measured: Up to 36 months.
Disease Control Rate (DCR)
Measured: Up to 36 months.
Progression Free Survival (PFS) by RECIST v1.1
Measured: Up to 36 months.
Overall Survival (OS)
Measured: Up to 36 months.
Change from Baseline in Antihypertensive Medication Dose
Measured: Up to 36 months.
Change from Baseline in Biochemical Response (Metanephrines/Disease Markers)
Measured: Up to 36 months.
Incidence of Adverse Events (AEs)
Measured: Up to 36 months.
Number of Participants with Clinically Significant Changes from Baseline in Clinical Laboratory Parameters
Measured: Up to 36 months.
Number of Participants with Grade 4+ Clinical Laboratory Parameters
Measured: Up to 36 months.
Number of Participants with Clinically Significant Changes from Baseline in Electrocardiogram (ECG) Parameters
Measured: Up to 36 months.
Maximum Observed Concentration of ONC206
Measured: Up to 36 months.
Time of Maximum Observed Concentration (Tmax) of ONC206
Measured: Up to 36 months.
Area Under the Concentration-Time Curve Extrapolated to Infinity (AUCinf)
Measured: Up to 36 months.
Area Under the Concentration Time Curve Over the Dosing Interval
Measured: Up to 36 months.
Terminal Half-Life (t1/2) of ONC206
Measured: Up to 36 months.
Change from Baseline in European Organization for the Research and Treatment of Cancer Quality of Life-Core Questionnaire (EORTC-QLQ-C30)
Measured: Up to 36 months.
Change from Baseline in Functional Assessment of Cancer Therapy - Bone Pain (FACT-BP)
Measured: Up to 36 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2025
- Study start2 Jan 2026
- Primary completion15 Dec 2028 (estimated)
- Study completion15 Dec 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Advanced Pheochromocytoma and ParagangliomaStudies of the same condition, open and under way.
- More trials run by Jazz PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Jazz Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07282587
- Sponsor's protocol ID
- ONC206-002
- Status last verified
- Oct 2026
- First submitted
- 5 Dec 2025
- Last updated
- 2 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: In accordance with ICMJE requirements, Jazz Pharmaceuticals may provide qualified external researchers access to individual participant data (IPD) and clinical trial data that underlie the results of this trial upon request. Qualified researchers can submit a request on https://www.jazzpharma.com/science/clinical-trial-data-sharing/ as outlined. Jazz Pharmaceuticals reserves the right not to consider a request. For inquiries about Jazz's data sharing policy contact clinicaldatasharing@jazzpharma.com.