Study of ONC206 (JZP3507) in Advanced Pheochromocytoma and Paraganglioma

NCT07282587 | Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: ONC206-002
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 9

Brief Summary

This is a two-stage Phase 2 trial evaluating the efficacy and safety of ONC206 in participants with Pheochromocytoma and Paraganglioma (PCPG).

Conditions

Advanced Pheochromocytoma and Paraganglioma; PCPG

Keywords

PCPG; ONC206; JZP3507

Outcome Measures

Primary Outcomes (1)

• 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.

  Up to 36 months.

Secondary Outcomes (18)

• Duration of Response (DOR)

  Up to 36 months.

• Time to Response (TTR)

  Up to 36 months.

• Disease Control Rate (DCR)

  Up to 36 months.

• Progression Free Survival (PFS) by RECIST v1.1

  Up to 36 months.

• Overall Survival (OS)

  Up to 36 months.

Study Arms (3)

Stage 1 Participants

EXPERIMENTAL

150 mg ONC206 BID TIW

Drug: ONC206

Stage 2: Dose 1

EXPERIMENTAL

Participants receiving ONC206 at dose (To be Determined \[TBD\] post stage 1).

Drug: ONC206: Dose 1

Stage 2: Dose 2

EXPERIMENTAL

Participants receiving ONC206 at dose (TBD post stage 1).

Drug: ONC206: Dose 2

Interventions

ONC206 DRUG

150 mg BID TIW

Stage 1 Participants

ONC206: Dose 1 DRUG

Stage 2: Dose 1

ONC206: Dose 2 DRUG

Stage 2: Dose 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.
• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Jazz Pharmaceuticals: lead

Study Sites (9)

UCLA

Los Angeles, California, 90095, United States

RECRUITING

Stanford

Palo Alto, California, 94604, United States

RECRUITING

U of Colorado

Aurora, Colorado, 80045, United States

NOT YET RECRUITING

U of Michigan

Ann Arbor, Michigan, 48109, United States

NOT YET RECRUITING

Mayo-Rochester

Rochester, Minnesota, 55905, United States

RECRUITING

Washington University

St Louis, Missouri, 63110, United States

RECRUITING

Columbia University Medical Center

New York, New York, 10032, United States

NOT YET RECRUITING

PENN

Philadelphia, Pennsylvania, 19104, United States

RECRUITING

UTAH

Salt Lake City, Utah, 84112, United States

NOT YET RECRUITING

MeSH Terms

Conditions

Paraganglioma

Interventions

ONC206

Condition Hierarchy (Ancestors)

Neuroendocrine Tumors; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms by Histologic Type; Neoplasms; Neoplasms, Nerve Tissue

Central Study Contacts

Clinical Trial Disclosure & Transparency

CONTACT

215-832-3750; ClinicalTrialDisclosure@jazzpharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 5, 2025
• First Posted: December 15, 2025
• Study Start: January 2, 2026
• Primary Completion (Estimated): December 15, 2028
• Study Completion (Estimated): December 15, 2028
• Last Updated: February 27, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will share

Locations

US (9)