NCT02863991

Oral ONC201 in Relapsed/Refractory Multiple Myeloma

• Status: Terminated
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Jazz Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a Phase 1/2 open-label study of ONC201 administered orally once every week in combination with dexamethasone in adults with relapsed/refractory multiple myeloma. The primary objective of this study was to evaluate the antitumor efficacy of ONC201. Note: This study was completed by predecessor company, Oncoceutics, Inc.

Detailed description

In Phase 1 of the study, patients were to receive 375 or 625 mg ONC201 once every week in combination with dexamethasone using a 3+3 dose escalation design to evaluate up to 625 mg ONC201 weekly with 20 mg dexamethasone. In Phase 2 of the study, patients were to receive 625 mg ONC201 once every week. Dexamethasone was to be administered at a dose determined in Phase 1. A treatment cycle was defined as 3 weeks. The dose-limiting toxicity window was defined as the first 3 weeks of treatment (i.e., 1 cycle). Patients may have continued treatment with ONC201 until disease progression, occurrence of an unacceptable adverse event, intercurrent illness or changes in the patient's condition rendered the patient unacceptable to continue, patient decision to withdraw from the study, or discontinuation of the study by the Sponsor. Assessments of tumor response were conducted using the International Myeloma Working Group response criteria. Safety was assessed through the reporting of adverse events, measurement of vital signs, electrocardiograms, and clinical laboratory results. Before the study was terminated, a total of 17 patients were enrolled and treated with ONC201: 2 patients received 375 mg ONC201 and 15 patients received 625 mg ONC201.

Conditions

• Multiple Myeloma

Interventions

Type	Name	Description
DRUG	ONC201	375 mg or 625 mg ONC201
DRUG	Dexamethasone	20 mg dexamethasone

Timeline

• Start date: 2017-04-19
• Primary completion: 2019-09-17
• Completion: 2019-12-16
• First posted: 2016-08-11
• Last updated: 2024-07-03
• Results posted: 2024-07-03

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study