NCT03929211

CPI-613 and Hydroxychloroquine for Patients With High Risk Myelodysplastic Syndrome

A Phase I/II Study of CPI-613 and Hydroxychloroquine for Patients With High Risk MDS Who Have Failed Hypomethylating Therapy

• Status: Withdrawn
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: Wake Forest University Health Sciences · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a phase 1/2 study of the combination of CPI-613 and hydroxychloroquine for the treatment of high risk myelodysplastic syndrome patients who have failed a hypomethylating agent.

Detailed description

Primary Objective(s): * To determine the Maximum Tolerated Dose (MTD) of the combination of CPI-613 and Hydroxychloroquine therapy for patients with high risk MDS who have failed hypomethylating therapy. * To determine the overall response rate (complete remission (CR), marrow CR, partial remission (PR), Hematologic improvement (HI)) of high risk MDS patients who have failed hypomethylating agents, treated with the combination of CPI-613 and the maximally tolerated dose of hydroxychloroquine Secondary Objective(s): * To assess the safety of the combination * To assess progression-free-survival (PFS) * To assess the overall survival of MDS patients who have failed hypomethylating agents treated with the combination of CPI-613 and hydroxychloroquine defined as the time from enrolment on study to death from any cause. * To assess any changes in the frequency of blood transfusions OUTLINE: This is a phase I, dose-escalation study of hydroxychloroquine, followed by a phase II study. Patients receive hydroxychloroquine orally (PO) and 6,8-bis(benzylthio)octanoic acid intravenously (IV) over 2 hours on days 1-5. Treatments repeat every 14 days for 2 cycles in the absence of disease progression or unacceptable toxicity. Beginning cycle 3, patients receive hydroxychloroquine PO and 6,8-bis(benzylthio)octanoic acid IV over 2 hours on days 1-5. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. After completion of study treatment, patients are followed up for 5 years.

Conditions

• Myelodysplastic Syndromes
• Progressive Disease

Interventions

Type	Name	Description
DRUG	CPI-613	Given intravenously, CPI-613 dose will be 2,000 mg/m² and will not escalate.
DRUG	Hydroxychloroquine	Given by mouth, hydroxychloroquine will be dose escalated from 600 mg to 1,200 mg orally given 2 hours before the CPI-613 infusion on days 1-5 of every 28 day cycle in a 3+3 dose escalation design.

Timeline

• Start date: 2021-05-01
• Primary completion: 2022-09-01
• Completion: 2027-07-01
• First posted: 2019-04-26
• Last updated: 2021-04-14

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study