ONC206 for Pheochromocytoma/Paraganglioma

Not yet recruiting at 9 trial locations

Overseen By Clinical Trial Disclosure & Transparency

Age: 18+

Sex: Any

Trial Phase: Phase 2

Sponsor: Jazz Pharmaceuticals

Must not be taking: Strong CYP inhibitors, inducers

Disqualifiers: Active cardiac disease, Additional malignancy, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ONC206 for individuals with pheochromocytoma and paraganglioma, tumors that cannot be surgically removed. The study explores ONC206's effectiveness and safety for patients. Different groups in the trial receive varying doses to determine the optimal one. Suitable candidates for this trial have been diagnosed with these specific tumors, have not responded to other treatments, and have stable blood pressure. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to significant findings.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you must not take strong CYP inhibitors or inducers within 14 days before starting the study. Also, there should be no change in your blood pressure medications for at least 14 days before the trial.

Is there any evidence suggesting that ONC206 is likely to be safe for humans?

Research has shown that ONC206 has been tested for safety in other conditions, particularly in patients with advanced brain tumors. Early studies examined various doses and schedules of this drug. These studies found that ONC206 was generally well-tolerated, with most participants not experiencing serious side effects.

The current trial is in Phase 2, indicating that ONC206 has already passed initial safety tests. This phase focuses more on the drug's effectiveness, though safety remains closely monitored. While specific data from this trial is not yet available, earlier research suggests that ONC206 may be safe for humans, though individual experiences can differ. It is important to consult a doctor to understand the potential risks and benefits. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for Pheochromocytoma/Paraganglioma?

Researchers are excited about ONC206 for pheochromocytoma and paraganglioma because it introduces a novel approach compared to traditional treatments like surgery, radiation, and chemotherapy. ONC206 works differently by targeting specific cancer cell pathways, potentially offering a more precise and effective treatment. This drug might offer a new hope for patients whose tumors are resistant to standard therapies, providing an option that could be both more effective and less invasive.

What evidence suggests that ONC206 might be an effective treatment for Pheochromocytoma and Paraganglioma?

Research has shown that ONC206, the investigational treatment in this trial, may help treat pheochromocytoma and paraganglioma (PCPG). Early studies found that ONC206 can stop cancer cells from growing by disrupting their energy production. It affects the mitochondria, the cell's power source. ONC206 has also been tested on other cancer types, where it demonstrated strong anti-cancer effects. While specific information on PCPG is still being gathered, these findings offer hope for its potential effectiveness. ^{1 3 5 6 7}

Are You a Good Fit for This Trial?

This trial is for adults with advanced pheochromocytoma or paraganglioma that can't be surgically removed. Participants must have tried other treatments without success, or are not suitable for them, and should have measurable disease. They need controlled blood pressure, the ability to swallow pills, a decent performance status (able to carry out daily activities), and stable lab results.

Inclusion Criteria

3. Must have measurable disease per RECIST v1.1, as assessed by the Investigator.

8. Has laboratory test results meeting the following parameters within 14 days before the first dose of study treatment

9. Has an expected survival of at least 12 weeks, as predicted by the physician.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Stage 1

Participants receive 150mg ONC206 twice daily, on three consecutive days per week in each 28-day cycle

Varies based on response

Treatment Stage 2

Participants are randomized to receive one of two ONC206 dose levels if Stage 1 meets the planned response

Varies based on response

Follow-up

Participants are monitored for safety and effectiveness after treatment

Up to 36 months

What Are the Treatments Tested in This Trial?

Interventions

• ONC206

Trial Overview

The study tests different doses of ONC206 in patients with Pheochromocytoma and Paraganglioma. It's a Phase 2 trial designed to see how effective and safe this treatment is. Patients will receive one of two dosing levels of ONC206 orally to determine its impact on their cancer.

How Is the Trial Designed?

Group I: Stage 2: Dose 2 Experimental Treatment 1 Intervention

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

Group II: Stage 2: Dose 1 Experimental Treatment 1 Intervention

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

Group III: Stage 1 Participants Experimental Treatment 1 Intervention

150 mg ONC206 BID TIW

Who Is Running the Clinical Trial?

Jazz Pharmaceuticals

Lead Sponsor

Trials

252

Recruited

35,100+

Bruce C. Cozadd

Jazz Pharmaceuticals

Chief Executive Officer since 2009

BA in Economics from Yale University, MBA from Stanford University

Dr. Austin

Jazz Pharmaceuticals

Chief Medical Officer since 2023

MD from the Royal College of Surgeons in Ireland

Citations

1.

clinicaltrials.gov

clinicaltrials.gov/study/NCT07282587

NCT07282587 | Study of ONC206 (JZP3507) in Advanced ...

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

2.

nanets.net

nanets.net/abstracts-archive/2025/2196-t7-narayan-trial-in-progress-a-phase-2-study-of-onc206-in-patients-with-advanced-pheochromocytoma-and-paraganglioma?format=html

Trial-in-Progress: A Phase 2 Study of ONC206 in Patients ...

This open-label, multicenter, two-stage Phase 2 clinical study will evaluate efficacy and safety of. ONC206 in adult patients with advanced PCPG who have ...

3.

mdpi.com

mdpi.com/1467-3045/47/6/451

Investigating the Effects of ONC206 Alone and in ...

Our results revealed that ONC206, alone or in combination with CDDP, exerts a potent anti-proliferative effect on both OVCAR-420 and SKOV-3 cells, as shown in ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04732065

Study Details | NCT04732065 | ONC206 for Treatment of ...

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline gliomas.

5.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC11267062/

ONC206 targeting ClpP induces mitochondrial dysfunction ...

Our data revealed the inhibitory effects of ONC206 on cell proliferation and the ability of ONC206 to induce mitochondrial dysfunction and ...

6.

smartpatients.com

smartpatients.com/trials/NCT07282587

Study of ONC206 (JZP3507) in Advanced ...

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

7.

clinicaltrials.gov

clinicaltrials.gov/study/NCT04732065

Study Details | NCT04732065 | ONC206 for Treatment of ...

This phase I trial studies the effects and best dose of ONC206 alone or in combination with radiation therapy in treating patients with diffuse midline ...