Phase 1 Rare Disease Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Adults aged 18 or older who have a brain or spinal cord tumour that has come back (recurred), with the diagnosis confirmed by a laboratory examination of tumour tissue. A wide range of tumour types are included — the trial site can confirm whether a specific tumour type qualifies.
• People whose tumour has returned and can be measured on a brain scan, who have already completed the standard treatments available for their tumour type, and for whom no further standard treatment options remain.
• People who are reasonably independent in daily life, as measured by a standard functioning score (called the Karnofsky Performance Score) of 70 or above. People with significant leg weakness who still manage most self-care tasks may also be considered eligible (confirm with trial site).
• People who have waited the required time since their last treatment before starting the study drug: at least 28 days after most chemotherapy; 42 days after certain chemotherapy types (nitrosoureas or bevacizumab); 14–28 days after targeted therapy; and 14 days after radiotherapy.
• People whose side effects from previous treatments have mostly resolved before starting the study drug (mild hair loss or nerve-related symptoms are acceptable, and mild-to-moderate low white blood cell counts may also be acceptable).
• People who have fully recovered from any major surgery completed at least 4 weeks before starting the study drug. Minor outpatient procedures do not require a waiting period.
• People whose blood, liver, and kidney function test results fall within the acceptable ranges specified by the trial (confirm with trial site for exact values).
• People who are able to provide a stored tumour tissue sample from a previous operation or biopsy, with enough tissue slides available for laboratory testing before starting the study drug.
• People who are able to swallow either capsules or liquids, depending on the dose and form of study drug assigned.
• People who are able to understand and sign a consent form personally — a family member or legal representative cannot provide this initial consent on someone else's behalf.
• People who are able to have an MRI scan with an intravenous contrast dye (gadolinium).
• People who have had a negative COVID-19 test within 72 hours before starting the study drug. People who had a confirmed COVID-19 infection within the past 90 days but recovered more than 20 days ago do not need to be re-tested.

Who may not be able to join:

• People who have had a previous allergic reaction to ONC201 or to medicines with a similar chemical make-up to ONC206.
• People with an uncontrolled ongoing infection, or mental health or personal circumstances that would make it difficult to follow the study requirements.
• People who are unwilling or unable to follow the study plan or work with the research team.
• People who are HIV-positive and currently taking combination anti-retroviral therapy, due to possible interactions with the study drug.
• People with certain active heart problems, including a prolonged QT interval on a heart tracing (ECG), chest pain requiring medication, certain abnormal heart rhythms, a need for a pacemaker, significant heart valve disease, or inflammation around the heart.
• People with a history of certain heart conditions, including a heart attack in the past 6 months, moderate-to-severe heart failure, or a diagnosed disease of the heart muscle.
• People who have had a stroke (either from a blocked or a burst blood vessel) in the past 3 months. Bleeding or circulation changes directly related to a recent brain operation are not an exclusion (confirm with trial site).
• People with seizures that are difficult to control. People who have had a generalised seizure in the 28 days before enrolment may not be eligible, unless the seizure occurred within 7 days of a brain operation. People with a prior history of seizures must have been on a stable dose of one or two seizure medicines for at least 14 days before enrolment.
• People with significant stomach or bowel problems that could interfere with how the study drug is absorbed, such as severe nausea, vomiting, diarrhoea, a condition affecting nutrient absorption, or prior removal of part of the small bowel.
• People who have received certain blood cell-stimulating injections (such as G-CSF) within 2 weeks before starting the study drug.
• People currently taking therapeutic doses of warfarin or similar blood-thinning medicines.
• People currently taking certain medicines that strongly affect a group of liver enzymes (CYP3A4, 2D6, 1A2, 2C9, and 2C19) within 14 days before starting the study drug, or who would need to continue these medicines during the study.
• People who are pregnant or breastfeeding.
• Women of childbearing potential who have a positive pregnancy test within 72 hours before starting the study drug.
• People currently receiving any other cancer treatment (standard or experimental) at the same time.
• People whose alcohol or substance use, in the investigator's view, would affect their safety or ability to follow the study plan.
• People with any other serious unstable medical or psychiatric condition that the investigators consider would put them at risk or affect their ability to participate.
• Women who are capable of becoming pregnant, or men capable of fathering a child, who do not agree to use highly effective contraception during the study and for 16 weeks after the final dose. Specific contraception requirements apply and should be confirmed with the trial site. Note: oral, implantable, or injectable hormonal contraceptives are not considered effective enough for this study due to possible drug interactions.
• People who have previously received ONC201 (or a placebo in an ONC201 trial) for a specific tumour type called H3 K27M-mutant diffuse glioma on or after 1 January 2023, or who have received ONC201 from any other source for that tumour type.

Important: Always verify eligibility with the trial site directly before applying.