A Collaboration Phase 2 Study of Venetoclax in Combination With Conventional Chemotherapy in Pediatric Patients With Acute Myeloid Leukemia

NCT05955261 Phase 2 SUSPENDED

This is a phase 2 study to test the hypothesis that venetoclax in combination with standard chemotherapy will be tolerable and active in pediatric patients with newly diagnosed acute myeloid leukemia (AML). Primary Objectives: * Establish the tolerability adding venetoclax to standard chemotherapy in pediatric patients with AML * Estimate the proportion of patients who become minimal residual disease (MRD) negative by flow cytometry after one course of venetoclax-based induction therapy Secondary Objectives: \- Estimate the rates of complete remission (CR), event-free survival (EFS), and overall survival (OS) in pediatric patients who receive venetoclax-based chemotherapy

Details

Lead sponsor	St. Jude Children's Research Hospital
Phase	Phase 2
Status	SUSPENDED
Enrolment	70
Start date	2023-07-25
Completion	2034-03

Conditions

• Acute Myeloid Leukemia

Interventions

• Venetoclax
• Azacitidine
• Cytarabine
• Gemtuzumab Ozogamicin
• Daunorubicin Hydrochloride
• Fludarabine Phosphate
• Idarubicin Hydrochloride
• Mitoxantrone Hydrochloride
• Etoposide
• Gilteritinib

Primary outcomes

• Minimal residual disease (MRD)-negativity rate — At day 29 after induction 1
  Will compute a binomial confidence interval for the proportion of MRD-evaluable patients who become MRD-negative (defined as MRD \< 0.1%) after induction 1.
• Incidence of death or unacceptable adverse event — From initiation to completion of each course of therapy, an average of 6 weeks
  A patient is deemed to have tolerated a course of therapy if they complete that course without death or an unacceptable adverse event. Will monitor the tolerability or each course using multi-stage binomial stopping rules. Will use the method of Jung and Kim to compute 95% confidence intervals that adjust for the multi-stage monitoring.

Countries

United States