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ONC201 Maintenance Therapy for Acute Myeloid Leukemia

Phase 1

Waitlist Available

Led By Vijaya R Bhatt, MBBS

Research Sponsored by University of Nebraska

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Absolute neutrophil count (ANC) greater than 1000/µL without the use of granulocyte colony stimulating factor in the past 2 weeks, and platelet count 50,000/µL without platelet transfusion in the past 2 weeks.

Disease status: <5% bone marrow blast at the time of enrollment

Must not have

Use of prednisone at a dose of ≥0.25 mg/kg/day (or equivalent dose of another glucocorticoid) at the time of enrollment

Treatment with any other investigational agent, device, or procedure, within 21 days (or 5 half-lives, whichever is greater)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up after one month of treatment

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new drug for people with AML or MDS who have had a stem cell transplant. The drug is taken orally, once a week for a year.

Who is the study for?

Adults over 19 with a history of high-risk Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS), post stem cell transplant, can join this trial. They must have stable blood counts, be able to take oral meds, and not be pregnant or planning pregnancy. Exclusions include severe organ dysfunction, uncontrolled infections or heart/lung conditions, certain drug sensitivities, and recent graft-versus-host disease.

What is being tested?

The study tests ONC201 as maintenance therapy for AML/MDS patients after a stem cell transplant. It's an oral medication taken weekly for one year to see if it helps prevent cancer from returning.

What are the potential side effects?

While specific side effects of ONC201 are not listed here, common ones may include fatigue, nausea, diarrhea, liver enzyme changes and potential risk of infection due to immune system impact.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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My white blood cell and platelet counts are within safe ranges without recent medical help.

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My bone marrow has less than 5% cancer cells.

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I can take pills by mouth.

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I am 19 years old or older and live in Nebraska.

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My AML is considered high-risk based on specific genetic features or my treatment history.

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My MDS is high-risk based on specific genetic changes or lack of response to treatment.

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I am mostly able to care for myself.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I am currently taking a high dose of prednisone or a similar medication.

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I haven't been part of any other research studies for at least 21 days.

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I do not have severe heart or lung conditions that are not under control.

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I have HIV or active hepatitis B or C.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ after one month of treatment

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~after one month of treatment

This trial's timeline: 3 weeks for screening, Varies for treatment, and after one month of treatment for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Grade ≥3 toxicities

Rate of dose limiting toxicities during the first cycle

Secondary study objectives

Number of toxicities (all grades) during the duration of maintenance therapy with ONC 201

The rate of relapse

The rate of relapse-free survival

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: ONC-201 TreatmentExperimental Treatment1 Intervention

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.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

ONC-201

2015

Completed Phase 1

~40

0 / 11Eligibility criteria met