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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up after one month of treatment
Awards & highlights

Study 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.Check my eligibility
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.See study design
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.

Timeline

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

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Grade ≥3 toxicities
Rate of dose limiting toxicities during the first cycle
Secondary outcome measures
Number of toxicities (all grades) during the duration of maintenance therapy with ONC 201
The rate of relapse
The rate of relapse-free survival

Side effects data

From 2021 Phase 2 trial • 30 Patients • NCT03394027
60%
Fatigue
40%
Alkaline phosphatase increased
40%
Aspartate aminotransferase increased
40%
Lymphocyte count decreased
40%
Dizziness
30%
Alanine aminotransferase increased
30%
Diarrhea
20%
Hypophosphatemia
20%
Abdominal pain
20%
Anemia
20%
Nausea
20%
Dyspnea
10%
Sepsis
10%
Lethargy
10%
Proteinuria
10%
Pain
10%
Vaginal discharge
10%
Paresthesia
10%
Pleural effusion
10%
Vaginal hemorrhage
10%
Edema limbs
10%
Flu like symptoms
10%
Gastroesophageal reflux disease
10%
Headache
10%
Atrial flutter
10%
Cardiac arrest
10%
Dehydration
10%
Neoplasms benign, malignant and unspecified (incl cysts and polyps) - Other, Death
10%
Sinus tachycardia
10%
Stroke
10%
Thromboembolic event
10%
Urinary tract infection
10%
Anorexia
10%
Arthralgia
10%
Back pain
10%
Constipation
10%
Lymphedema
10%
Malaise
10%
Mucosal infection
10%
Myalgia
10%
Rash maculo-papular
10%
Rhinorrhea
10%
Vascular access complication
10%
Weight loss
10%
Cough
10%
Creatinine increased
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 3-Endometrial Cancer (Female Only)
Cohort 1-Hormone Receptor (HR) + Breast Cancer (Male and Female)
Cohort 2-Triple Negative Breast Cancer (Male and Female)

Trial Design

1Treatment groups
Experimental Treatment
Group I: ONC201 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
ONC201
2017
Completed Phase 2
~60

Find a Location

Who is running the clinical trial?

University of NebraskaLead Sponsor
536 Previous Clinical Trials
1,143,902 Total Patients Enrolled
Vijaya BhattLead Sponsor
Vijaya R Bhatt, MBBSPrincipal InvestigatorUniversity of Nebraska

Media Library

ONC201 Clinical Trial Eligibility Overview. Trial Name: NCT03932643 — Phase 1
Myelodysplastic Syndrome Research Study Groups: ONC201 treatment
Myelodysplastic Syndrome Clinical Trial 2023: ONC201 Highlights & Side Effects. Trial Name: NCT03932643 — Phase 1
ONC201 2023 Treatment Timeline for Medical Study. Trial Name: NCT03932643 — Phase 1
Myelodysplastic Syndrome Patient Testimony for trial: Trial Name: NCT03932643 — Phase 1

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

What other research has been conducted on the efficacy of ONC201?

"Currently, there are 9 ongoing research projects for ONC201 with none of them in the final stage. Most studies take place in Miami, Florida; however, clinical trials covering this drug can be found at a total of 48 locations globally."

Answered by AI

Are there opportunities for individuals to register as participants in this research trial?

"Affirmative. Clinicaltrials.gov indicates that this investigation is currently searching for qualified individuals, with information last updated on August 4th 2022 since its initial posting of July 30 2019. This trial requires 20 subjects to be found from one centre."

Answered by AI

What is the current participation rate in this clinical research?

"Affirmative. Clinicaltrials.gov details that this clinical trial, which first appeared on July 30th 2019 is still actively recruiting patients. 20 volunteers are need to be enlisted from 1 site across the country."

Answered by AI

Has ONC201 been accepted by the FDA for medical use?

"Our team has assessed the safety of ONC201 to be a 1 due to it being in its initial phases, with few studies validating both efficacy and security."

Answered by AI

Is this trial a groundbreaking exploration of new therapies?

"For the past 6 years, ONC201 has been under scrutiny in clinical trials; beginning with a 120-person study sponsored by Oncoceutics Inc. after which Phase 1 & 2 drug approval was granted. Nine active studies are currently ongoing across 28 cities and 4 countries."

Answered by AI

Who else is applying?

What state do they live in?
Florida
What portion of applicants met pre-screening criteria?
Did not meet criteria
How many prior treatments have patients received?
1

Why did patients apply to this trial?

I have a bleed in my left eye which is interfering with my sight. I also have floaters in both eyes.
PatientReceived no prior treatments
Recent research and studies
clinicaltrials.govStudy
ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant
Vijaya Bhatt 2019. "ONC 201 Maintenance Therapy in Acute Myeloid Leukemia and Myelodysplastic Syndrome After Stem Cell Transplant". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03932643.
All > Mds > Myelodysplastic Syndrome
~1 spots leftby Aug 2024
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