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.
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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
26
Rutgers Cancer Institute of New Jersey
New Brunswick, New Jersey, United States
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.
Time frame: Up to 4 weeks after end of study treatment
Frequency of toxicities associated with ONC201
Time frame: Up to 4 weeks after end of study treatment
Response rate of oral ONC201 in patients with advanced solid tumors
Time frame: 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.
Time frame: Up to 4 weeks of therapy
Clinical benefit rate (stable disease and partial disease)
Time frame: 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.
Time frame: Up to 4 weeks after end of study treatment
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