NCT03932643

ONC-201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant

A Phase 1 Trial of ONC-201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After an Allogeneic Hematopoietic Stem Cell Transplant

• Status: Completed
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: University of Nebraska · Academic / Other
• Sex: All
• Age: 19 Years
• Healthy volunteers: Not accepted

Summary

This is a single-center Phase 1 trial of 20 patients with AML/MDS. Eligible patients will be enrolled following an informed consent between 6-20 weeks after allogeneic hematopoietic stem cell transplant. Patients will receive weekly oral ONC 201 for a total of 52 weeks.

Detailed description

This is a single-center Phase 1 trial of 20 participants with AML/MDS. Eligible participants will be enrolled following an informed consent between 6-20 weeks after allogeneic hematopoietic stem cell transplant. Participants will receive weekly oral ONC-201 for a total of 52 weeks. The objectives of the study are: 1. To determine the safety and preliminary efficacy of ONC-201 maintenance therapy among partcipants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), who undergo allogeneic hematopoietic stem cell transplant. Participants will be monitored for toxicities (using Common Terminology Criteria for Adverse Events, CTCAE version 5.0), quality of life \[Functional Assessment of Cancer Therapy-Bone Marrow Transplant (FACT-BMT)\], and immunologic changes. We will also examine changes in functional status (Karnofsky Performance Scale (KPS), instrumental activities of daily living and short physical performance battery), rates of disease relapse and mortality.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	ONC-201	ONC-201 Capsules, 125 mg Oral ONC-201 at various dose levels will be given at weekly intervals for up to 13 cycles (52 weeks); 4-week therapy will be considered 1 cycle.

Timeline

• Start date: 2019-07-30
• Primary completion: 2025-03-27
• Completion: 2025-03-27
• First posted: 2019-05-01
• Last updated: 2026-01-20

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study