Actively Recruiting
Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
Led by Jazz Pharmaceuticals · Updated on 2025-12-18
102
Participants Needed
1
Research Sites
318 weeks
Total Duration
On this page
Sponsors
J
Jazz Pharmaceuticals
Lead Sponsor
N
National Institutes of Health (NIH)
Collaborating Sponsor
AI-Summary
What this Trial Is About
The primary objective of this Phase 1, open-label, dose-escalation, and exploratory study is to evaluate the safety and tolerability profile (establish the maximum-tolerated dose) and evaluate the occurrence of dose-limiting toxicities (DLTs) following single weekly or multiple-day weekly dose regimens of single-agent, oral ONC206 in patients with recurrent, primary central nervous system (CNS) neoplasms.
CONDITIONS
Official Title
Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients aged 18 years or older with histologically confirmed recurrent primary CNS neoplasm
- Measurable recurrent disease after standard treatments with no available standard options
- Karnofsky Performance Score (KPS) of 70 or higher
- No investigational or approved cytotoxic chemotherapy within 28 days prior to first dose (42 days for nitrosoureas and bevacizumab)
- All prior therapy-related adverse events (Grade 2 or higher) resolved except alopecia or neuropathy; Grade 1 or 2 lymphopenia allowed
- No major surgery within 4 weeks prior to first dose and fully recovered from surgery
- Normal organ and marrow function as defined by specific blood counts and liver/kidney tests
- Availability of tumor tissue for biomarker analysis before first dose
- Ability to swallow oral capsules or liquids depending on formulation
- Ability to give informed consent personally
- Ability to tolerate MRI with contrast
- Negative COVID-19 test within 72 hours before first dose or prior infection more than 20 days before treatment
You will not qualify if you...
- History of allergic reactions to ONC206 or similar compounds
- Uncontrolled illness or infection, or psychiatric/social conditions limiting compliance
- Known HIV-positive patients on anti-retroviral therapy
- Active cardiac diseases including prolonged QT interval, arrhythmias requiring pacemaker, angina needing medication, valvular disease affecting function, or symptomatic pericarditis
- History of recent heart attack, congestive heart failure (NYHA III-IV), or cardiomyopathy
- Stroke within last 3 months
- Refractory epilepsy or generalized seizures in the last 28 days
- Significant gastrointestinal dysfunction affecting drug absorption
- Use of hematopoietic growth factors within 2 weeks prior to study
- Current use of therapeutic doses of warfarin or similar anticoagulants
- Use of strong CYP450 enzyme inhibitors or inducers within 14 days prior to first dose
- Pregnant or breastfeeding women
- Women of child-bearing potential with positive pregnancy test within 72 hours prior to first dose
- Use of other anti-cancer therapies during study
- Alcohol or substance abuse interfering with compliance or safety
- Any serious unstable medical or psychiatric conditions affecting safety or consent
- Women or men unwilling to use highly effective contraception during and 16 weeks after treatment
- Prior receipt of ONC201, placebo, or blinded study drug for H3 K27M-mutant diffuse glioma on or after January 1, 2023
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
National Institutes of Health
Bethesda, Maryland, United States, 20892
Actively Recruiting
Research Team
C
Clinical Trial Disclosure & Transparency
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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