Continuation of Oral ONC201 in Treating Patients With Advanced Solid Tumors

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

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.

Study Overview

• Status: Completed
• Conditions: Unspecified Adult Solid Tumor
• Intervention / Treatment: Other: laboratory biomarker analysis; Other: pharmacological study; Drug: Oral ONC201

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.

• Study Type: Interventional
• Enrollment (Actual): 26
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • New Jersey
    • New Brunswick, New Jersey, United States, 08903
      • Rutgers Cancer Institute of New Jersey

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

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

What is the study measuring?

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

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Changes in serum biomarkers of therapeutic response	Descriptive statistics will be provided.	Baseline to up to 4 weeks after end of study treatment
Changes in molecular targets of oral ONC201	Descriptive statistics will be provided.	Baseline to up to 4 weeks after end of study treatment

Collaborators and Investigators

• Sponsor: Rutgers, The State University of New Jersey
• Collaborators: National Cancer Institute (NCI); Rutgers Cancer Institute of New Jersey; Oncoceutics, Inc.

Publications and helpful links

General Publications

• Stein MN, Malhotra J, Tarapore RS, Malhotra U, Silk AW, Chan N, Rodriguez L, Aisner J, Aiken RD, Mayer T, Haffty BG, Newman JH, Aspromonte SM, Bommareddy PK, Estupinian R, Chesson CB, Sadimin ET, Li S, Medina DJ, Saunders T, Frankel M, Kareddula A, Damare S, Wesolowsky E, Gabel C, El-Deiry WS, Prabhu VV, Allen JE, Stogniew M, Oster W, Bertino JR, Libutti SK, Mehnert JM, Zloza A. Safety and enhanced immunostimulatory activity of the DRD2 antagonist ONC201 in advanced solid tumor patients with weekly oral administration. J Immunother Cancer. 2019 May 22;7(1):136. doi: 10.1186/s40425-019-0599-8.

Study record dates

Study Major Dates

• Study Start (Actual): February 20, 2015
• Primary Completion (Actual): October 18, 2018
• Study Completion (Actual): October 23, 2018

Study Registration Dates

• First Submitted: October 28, 2014
• First Submitted That Met QC Criteria: December 23, 2014
• First Posted (Estimate): December 24, 2014

Study Record Updates

• Last Update Posted (Actual): May 22, 2019
• Last Update Submitted That Met QC Criteria: May 20, 2019
• Last Verified: May 1, 2019

More Information

Terms related to this study

• Keywords: protocol specific
• Additional Relevant MeSH Terms: Neoplasms; Antineoplastic Agents; TIC10 compound
• Other Study ID Numbers: 051407 (Other Identifier: Rutgers Cancer Institute of New Jersey); P30CA072720 (U.S. NIH Grant/Contract); NCI-2014-02043 (Registry Identifier: CTRP (Clinical Trial Reporting Program))