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Continuation of Oral ONC201 in Treating Patients With Advanced Solid Tumors

Phase 1
Completed
Conditions
Unspecified Adult Solid Tumor
Interventions
Drug: Oral ONC201
Other: pharmacological study
Other: laboratory biomarker analysis
Registration Number
NCT02324621
Lead Sponsor
Rutgers, The State University of New Jersey
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.

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
26
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

Study & Design

Study Type
INTERVENTIONAL
Study Design
SINGLE_GROUP
Arm && Interventions
GroupInterventionDescription
Treatment (oral ONC201)Oral ONC201Patients receive oral ONC201 PO on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Treatment (oral ONC201)pharmacological studyPatients receive oral ONC201 PO on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Treatment (oral ONC201)laboratory biomarker analysisPatients receive oral ONC201 PO on day 1. Courses repeat every 21 days in the absence of disease progression or unacceptable toxicity.
Primary Outcome Measures
NameTimeMethod
Incidence of adverse experiences from oral ONC201 using National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) version 4.0Up 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 Outcome Measures
NameTimeMethod
Frequency of toxicities associated with ONC201Up to 4 weeks after end of study treatment
Pharmacokinetic parametersUp 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.

Response rate of oral ONC201 in patients with advanced solid tumorsAt 3 months
Clinical benefit rate (stable disease and partial disease)At 3 months
Time to progressive diseaseUp 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.

Trial Locations

Locations (1)

Rutgers Cancer Institute of New Jersey

🇺🇸

New Brunswick, New Jersey, United States

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