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Nucleoside Analog

Azacitidine for Acute Myeloid Leukemia

Phase 1
Waitlist Available
Led By Jeffrey E. Rubnitz, MD, PhD
Research Sponsored by St. Jude Children's Research Hospital
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Patients in all categories above must have ≥ 5% blasts in the bone marrow as assessed by morphology or ≥ 1 blasts in the bone marrow as assessed by flow cytometry. However, if an adequate bone marrow sample cannot be obtained, patients may be enrolled if there is unequivocal evidence of leukemia with ≥ 5% blasts in the peripheral blood. In addition, patients in all categories must not be eligible to undergo curative therapy, such as immediate SCT, because of disease burden, time needed to identify a stem cell donor, or other reasons.
AST (SGOT) and ALT (SGPT) ≤ 4 x ULN
Timeline
Screening 3 weeks
Treatment Varies
Follow Up 28 days after start of therapy
Awards & highlights

Study Summary

This trial will test the safety and best dose of venetoclax when given with or without idarubicin to pediatric patients with acute myeloid leukemia (AML) that did not respond to treatment or has come back after treatment.

Eligible Conditions
  • Acute Myeloid Leukemia

Eligibility Criteria

Inclusion Criteria

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

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~28 days after start of therapy
This trial's timeline: 3 weeks for screening, Varies for treatment, and 28 days after start of therapy for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
Maximum tolerated combination (MTC)
Secondary outcome measures
Complete Remission (CR)
Complete remission with incomplete count recovery (CRi)

Trial Design

1Treatment groups
Experimental Treatment
Group I: TreatmentExperimental Treatment5 Interventions
In Part 1, venetoclax with cytarabine will initially be given at dose level 1 and escalated based on tolerability. Idarubicin will be given only at dose level 4. Note: Part 1 has been completed. Two expansion cohorts will be enrolled: Cohort A will be a group of 12 participants receiving the recommended phase 2 doses (RP2D) of venetoclax plus cytarabine. Cohort B will be a group of 12 participants receiving the RP2D of venetoclax plus cytarabine and idarubicin. Intrathecal Triple Therapy (ITMHA) will be given prior to cycle 1. Patients without evidence of central nervous system (CNS) leukemia will receive no further IT therapy during cycle 1. Patients with CNS disease will receive weekly ITMHA beginning on day 8 until the cerebrospinal fluid becomes free of leukemia. Cohort C: Participants will receive venetoclax PO on days 1-21, azacitidine IV on days 1-7, and cytarabine Q12H on days 8-11.
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Azacitidine
2012
Completed Phase 3
~1440
Venetoclax
2019
Completed Phase 3
~1990
Cytarabine
2016
Completed Phase 3
~3310
Intrathecal Triple Therapy
2017
Completed Phase 1
~70
Idarubicin
2014
Completed Phase 4
~4330

Find a Location

Who is running the clinical trial?

Gateway for Cancer ResearchOTHER
45 Previous Clinical Trials
2,508 Total Patients Enrolled
St. Jude Children's Research HospitalLead Sponsor
426 Previous Clinical Trials
5,306,543 Total Patients Enrolled
Jeffrey E. Rubnitz, MD, PhDPrincipal InvestigatorSt. Jude Children's Research Hospital
5 Previous Clinical Trials
346 Total Patients Enrolled

Frequently Asked Questions

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

How is Azacitidine commonly utilized in treatment?

"Azacitidine is often prescribed to treat synovitis, as well as ophthalmia, sympathetic disorder, full blood count deficiencies and induction chemotherapy."

Answered by AI

Are minors permitted to participate in the present research study?

"According to the requirements for this experiment, all participants must be between 2 and 20 years of age."

Answered by AI

What prior research has been carried out involving Azacitidine?

"Presently, there are 155 Phase 3 trials and 722 active clinical investigations of Azacitidine. While most testing is taking place in Edmonton, Alberta; numerous international locales have established clinical studies for this drug."

Answered by AI

Are there currently any vacancies in this research project?

"Correct. Data hosted on clinicaltrials.gov affirms that this trial is still recruiting volunteers, having been posted initially in July 2017 and last updated in late July 2022. A total of 72 patients are needed from 2 different medical institutions."

Answered by AI

Who qualifies for inclusion in this clinical trial?

"To qualify for this clinical investigation, prospective participants must have a diagnosis of leukemia and be between 2 to 20 years old. The trial is currently aiming to enrol 72 patients in total."

Answered by AI

Does Azacitidine pose any significant risks to users?

"The safety of Azacitidine is tentatively rated at 1, as this Phase 1 trial has only provided a small amount of evidence for both its efficacy and security."

Answered by AI

How many participants are currently participating in this investigation?

"Affirmative. Clinicaltrials.gov records show that this investigation, which first appeared online on July 11th 2017, is still recruiting participants as of the last edit made on July 25th 2022. 72 individuals must be recruited from two individual sites for full data collection to occur."

Answered by AI
~8 spots leftby Mar 2025