Continuation of Oral ONC201 in Treating Patients With Advanced Solid Tumors

Study Overview
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Brief Summary
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This phase I trial studies the long-term side effects of the continuation of oral ONC201 in treating patients with solid tumors that have spread to other places in the body who have previously benefited from receiving this drug. Oral ONC201 may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth.

Detailed Description
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PRIMARY OBJECTIVES: I. To evaluate the long-term safety and tolerability of ONC201 (oral ONC201) administered orally in patients with advanced cancers. SECONDARY OBJECTIVES: I. To characterize pharmacokinetics of ONC201. II. To assess serum biomarkers of therapeutic response to ONC201. III. To assess preliminary antitumor activity of ONC201 as a single agent in advanced solid tumors. OUTLINE: Patients receive oral ONC201 orally (PO) on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 4 weeks.

Official Title
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A Continuation Clinical Trial of Every Three-Week Dosing of Oral ONC201 in Patients With Advanced Solid Tumors

Conditions
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Unspecified Adult Solid Tumor

Intervention / Treatment
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* Drug: Oral ONC201
* Other: pharmacological study
* Other: laboratory biomarker analysis


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Patient has completed participation in one of the ONC201 protocols, has not shown tumor progression while on study treatment, and has tolerated the study drug without unacceptable toxicities Patient has not met criteria for withdrawal from the base protocol Patient is willing to comply with protocol requirements and procedures, and keep scheduled study visits Patient agrees to practice effective contraception during the entire study period unless documentation of infertility exists Patient is able to understand and is willing to sign the informed consent form Exclusion Criteria: Patient is receiving concomitant standard and/or investigational anticancer therapy; local palliative radiotherapy is permissible upon discussion with the principal investigator Patient has alcohol or substance abuse which, in the opinion of the investigator, would interfere with compliance or safety Patient has any condition or laboratory abnormality which, in the opinion of the investigator, would pose additional risk in administering the study drug to the patient

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: Treatment (oral ONC201)<br>Patients receive oral ONC201 PO on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity. | Drug: Oral ONC201<br>* Given PO<br>* Other names: TIC10;Other: pharmacological study<br>* Correlative studies<br>* Other names: pharmacological studies;Other: laboratory biomarker analysis<br>* Correlative studies<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of adverse experiences from oral ONC201 using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 | An adverse experience is defined as any unintended or abnormal clinical observation that is not of benefit to the patient. Either the condition was not present prior to exposure to the study therapy, or it has worsened in intensity or frequency following exposure to the study therapy. Descriptive statistics will be provided. | Up to 4 weeks after end of study treatment | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Frequency of toxicities associated with ONC201 |  | Up to 4 weeks after end of study treatment | 
| Response rate of oral ONC201 in patients with advanced solid tumors |  | At 3 months | 
| Pharmacokinetic parameters | The oral ONC201 concentration, and the corresponding time, area under the curve, elimination half-life, total body clearance and volume of distribution will be determined for each patient concentration-time data using a non-compartmental model. Descriptive statistics will be provided. | Up to 4 weeks of therapy | 
| Clinical benefit rate (stable disease and partial disease) |  | At 3 months | 
| Time to progressive disease | Descriptive statistics on continuous data will include means, medians, standard deviations, and ranges will be provided to estimate clinical benefit rate. | Up to 4 weeks after end of study treatment | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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protocol specific