ONC201 Maintenance Therapy for Acute Myeloid Leukemia

Overseen By Marnee B Strege, RN

Age: 18+

Sex: Any

Trial Phase: Phase 1

Sponsor: University of Nebraska

Must not be taking: Dopamine antagonists

Disqualifiers: HIV, Hepatitis B/C, Cardiopulmonary, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: 52 weeks

20 Participants Needed

Trial Summary

Will I have to stop taking my current medications?

The trial does not specify if you need to stop taking your current medications. However, you cannot participate if you are on certain medications like high-dose prednisone or dopamine antagonists for psychotic disorders or Parkinson's disease. It's best to discuss your specific medications with the trial team.

What data supports the effectiveness of the drug ONC201 for treating acute myeloid leukemia?

Maintenance therapy for acute myeloid leukemia (AML) has shown benefits in prolonging remission, with recent studies indicating improved relapse-free survival rates when maintenance treatment is used. Although ONC201 is not specifically mentioned, the general effectiveness of maintenance therapies in AML suggests potential benefits. ^{1 2 3 4 5}

How does the drug ONC201 differ from other treatments for acute myeloid leukemia?

ONC201 is unique because it is being explored as a maintenance therapy for acute myeloid leukemia (AML), a setting where there is currently no standard treatment. Unlike traditional cytotoxic drugs, ONC201 may offer a novel approach by potentially targeting specific pathways involved in cancer cell survival, which could help reduce the risk of relapse after initial treatment. ^{1 2 6 7 8}

What is the purpose of this trial?

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.

Research Team

Vijaya R Bhatt, MBBS

Principal Investigator

University of Nebraska

Eligibility Criteria

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.

Inclusion Criteria

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

I have a history of AML or MDS.

My bone marrow has less than 5% cancer cells.

Exclusion Criteria

Any other condition that is judged by the physician to potentially interfere with compliance to the study protocol or pose a significant risk to the patient.

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

My liver tests are within twice the normal limit, except for high bilirubin due to Gilbert syndrome.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive weekly oral ONC-201 for a total of 52 weeks

52 weeks

Follow-up

Participants are monitored for safety, effectiveness, and relapse-free survival after treatment

up to 2 years

Treatment Details

Interventions

• ONC201

Trial Overview 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.

Participant Groups

Group I: ONC-201 Treatment Experimental Treatment 1 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.

Who Is Running the Clinical Trial?

University of Nebraska

Lead Sponsor

Trials

563

Recruited

1,147,000+

Vijaya Bhatt

Lead Sponsor

Trials

1

Recruited

20+

Findings from Research

Since 2017, the FDA has approved nine new agents for treating acute myeloid leukemia (AML), marking significant progress in AML therapy after decades of slow advancement.

Specific treatments for subsets of AML, such as all-trans retinoic acid and arsenic trioxide for acute promyelocytic leukemia, have led to impressive 10-year survival rates of over 80%, demonstrating the efficacy of targeted therapies.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Acute myeloid leukemia: Treatment and research outlook for 2021 and the MD Anderson approach. Kantarjian, HM., Kadia, TM., DiNardo, CD., et al.[2021]

Recent studies indicate that maintenance therapy for acute myeloid leukemia (AML) can be beneficial, particularly for high-risk patients or those with measurable residual disease (MRD), as shown in randomized trials.

The introduction of modern targeted agents, such as oral hypomethylating agents and immune checkpoint inhibitors, has renewed interest in maintenance therapy, emphasizing the need for standardized MRD measurement techniques in future research.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission. Shallis, RM., Podoltsev, NA.[2023]

References

1. United States pubmed.ncbi.nlm.nih.gov

Acute myeloid leukemia: Treatment and research outlook for 2021 and the MD Anderson approach. [2021]

2. Japan pubmed.ncbi.nlm.nih.gov

Acute myeloid leukemia in adults: is postconsolidation maintenance therapy necessary? [2007]

3. Netherlands pubmed.ncbi.nlm.nih.gov

What is the role of arsenic in newly diagnosed APL? [2018]

4. United States pubmed.ncbi.nlm.nih.gov

New strategies in acute promyelocytic leukemia: moving to an entirely oral, chemotherapy-free upfront management approach. [2021]

5. United States pubmed.ncbi.nlm.nih.gov

Maintenance therapy for acute myeloid leukemia: sustaining the pursuit for sustained remission. [2023]

6. Japan pubmed.ncbi.nlm.nih.gov

[Maintenance therapy for acute myeloid leukemia (including in patients who have undergone transplantation) including off-label use drugs in Japan]. [2022]

7. England pubmed.ncbi.nlm.nih.gov

AML and the art of remission maintenance. [2021]

8. England pubmed.ncbi.nlm.nih.gov

Maintenance therapy in acute myeloid leukemia after allogeneic hematopoietic stem cell transplantation. [2023]