RecruitingPhase 2

Study of ONC206 (JZP3507) in Advanced Pheochromocytoma and Paraganglioma

United States90 participantsStarted 2026-01-02

Plain-language summary

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

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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 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:

Overall Response Rate (ORR) by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1

Timeframe: Up to 36 months.

NCT IDNCT07282587

SponsorJazz Pharmaceuticals

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2028-12-15

Clinical Trial Disclosure & Transparency

Who can participate

Age range18 Years

SexALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

✓. 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 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: