ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant
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|ClinicalTrials.gov Identifier: NCT03932643|
Recruitment Status : Recruiting
First Posted : May 1, 2019
Last Update Posted : March 17, 2020
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia Myelodysplastic Syndromes||Drug: ONC201||Phase 1|
This is a single-center pilot study 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.
The objectives of the study are: 1. To determine the safety and preliminary efficacy of ONC 201 maintenance therapy among patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS), who undergo allogeneic hematopoietic stem cell transplant. 2. To investigate the impact of ONC 201 on reconstitution of NK and other immune cells.
Patients 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 specifically investigate the impact of ONC 201 on reconstitution of NK and other immune cells. 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.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||20 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Study of ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After an Allogeneic Hematopoietic Stem Cell Transplant|
|Actual Study Start Date :||July 30, 2019|
|Estimated Primary Completion Date :||July 2022|
|Estimated Study Completion Date :||July 2024|
Experimental: ONC201 treatment
A 3+3 dose escalation design will be followed. Given the safety profile in prior trials, A dose of 250 mg weekly will be the starting dose. The first 12-15 patients are expected to receive escalating doses of ONC 201, the remaining patients will go on the expansion cohort.
ONC201 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.
- The rate of dose limiting toxicities during the first cycle (among the dose escalating cohort) [ Time Frame: after one month of treatment ]
- The number of grade ≥3 toxicities [ Time Frame: during the first 3 cycles ot treatment (each cycle is 28 days) ]
- Number of toxicities (all grades) associated with the use of ONC 201 during the entire duration of maintenance therapy with ONC 201 [ Time Frame: upto 13 months after initiation of ONC 201 ]
- The rate of relapse [ Time Frame: upto 2 years after enrollment ]
- The rate of relapse-free survival [ Time Frame: upto 2 years after enrollment ]
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03932643
|Contact: Penny nurse firstname.lastname@example.org|
|Contact: Peggy Heiresemail@example.com|
|United States, Nebraska|
|University of Nebraska Medical Center||Recruiting|
|Omaha, Nebraska, United States, 68198|
|Principal Investigator:||Vijaya R Bhatt, MBBS||University of Nebraska|