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Small Molecule

ONC201 for Leukemia

Phase 1 & 2

Recruiting

Led By Gautam Borthakur, MBBS

Research Sponsored by M.D. Anderson Cancer Center

Eligibility Criteria Checklist

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

Must have

For Arms A, B, C, D, E patients must have relapsed or refractory acute leukemias or high-risk MDS for which no standard therapies are anticipated to result in a durable remission

Age >= 18 years

Must not have

Uncontrolled intercurrent illness

Active heart disease

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 63 days

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a new cancer drug to see if it works well and what the side effects are.

Who is the study for?

Adults over 18 with acute leukemia or high-risk myelodysplastic syndrome that's relapsed or refractory. They must be in fair health (ECOG 0-2), able to consent, and have no severe persistent side effects from past treatments. No recent heart issues, other cancer treatments within 3 years (except certain low-risk cases), or active infections like HIV/Hepatitis B/C.

What is being tested?

The trial is testing ONC201, a drug thought to block enzymes that cancer cells need to grow. It aims to find the safest dose and see how effective it is for patients whose disease has returned after treatment or hasn't responded at all.

What are the potential side effects?

Possible side effects of ONC201 include reactions related to stopping cancer cell growth which may affect normal cells too. Specific side effects aren't listed but generally could involve fatigue, nausea, liver enzyme changes, and others as seen with similar drugs.

Eligibility Criteria

Inclusion Criteria

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

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My leukemia or high-risk MDS has not responded to standard treatments.

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I am 18 years old or older.

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I can take care of myself and am up and about more than half of my waking hours.

Exclusion Criteria

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

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I do not have any unmanaged ongoing illnesses.

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I have an ongoing heart condition.

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My leukemia has spread to my brain or spinal cord.

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I am not taking strong CYP3A inhibitors.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 63 days

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~63 days

This trial's timeline: 3 weeks for screening, Varies for treatment, and 63 days for reporting.

Treatment Details

Study Objectives

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

Primary study objectives

Maximum Tolerated Dose (MTD) of ONOC201 in Relapsed or Refractory Acute Myelogenous Leukemia (AML), Myelodysplastic Syndrome (MDS) or Acute Lymphoblastic Leukemia (ALL)

Objective Response (OR) (Phase II)

Secondary study objectives

Objective Response (OR)

Awards & Highlights

No Placebo-Only Group

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

Trial Design

5Treatment groups

Experimental Treatment

Group I: Arm E (Akt/ERK inhibitor ONC201)Experimental Treatment1 Intervention

Patients receive Akt/ERK inhibitor ONC201 PO twice weekly. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity.

Group II: Arm D (Akt/ERK inhibitor ONC201)Experimental Treatment1 Intervention

Patients receive Akt/ERK inhibitor ONC201 PO QD. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Group III: Arm C (Akt/ERK inhibitor ONC201)Experimental Treatment1 Intervention

Patients receive Akt/ERK inhibitor ONC201 PO on the first two consecutive days of every week. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Group IV: Arm B (Akt/ERK inhibitor ONC201)Experimental Treatment1 Intervention

Patients receive Akt/ERK inhibitor ONC201 PO once every week. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

Group V: Arm A (Akt/ERK inhibitor ONC201)Experimental Treatment1 Intervention

Patients receive Akt/ERK inhibitor ONC201 PO once every 3 weeks. Cycles repeat every 21 days in the absence of disease progression or unacceptable toxicity.

0 / 7Eligibility criteria met