Unspecified Adult Solid Tumor
Conditions
Keywords
protocol specific
Brief summary
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
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.
Interventions
DRUGOral ONC201
Given PO
OTHERpharmacological study
Correlative studies
OTHERlaboratory biomarker analysis
Correlative studies
Sponsors
National Cancer Institute (NCI)
Rutgers Cancer Institute of New Jersey
Oncoceutics, Inc.
Rutgers, The State University of New Jersey
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of adverse experiences from oral ONC201 using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 | Up to 4 weeks after end of study treatment | 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. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Time to progressive disease | Up to 4 weeks after end of study treatment | Descriptive statistics on continuous data will include means, medians, standard deviations, and ranges will be provided to estimate clinical benefit rate. |
| Pharmacokinetic parameters | Up to 4 weeks of therapy | 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. |
| Clinical benefit rate (stable disease and partial disease) | At 3 months | — |
| 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 | — |
Other
| Measure | Time frame | Description |
|---|---|---|
| Changes in molecular targets of oral ONC201 | Baseline to up to 4 weeks after end of study treatment | Descriptive statistics will be provided. |
| Changes in serum biomarkers of therapeutic response | Baseline to up to 4 weeks after end of study treatment | Descriptive statistics will be provided. |
Countries
United States
Outcome results
None listed