Study of Venetoclax in Combination With Chemotherapy in Pediatric Patients With Refractory or Relapsed Acute Myeloid Leukemia or Acute Leukemia of Ambiguous Lineage

Part of paid clinical trials in Palo Alto, California.

Sponsor
St. Jude Children's Research Hospital
Study ID
NCT03194932
Phase
PHASE1
Status
Completed

Conditions

Eligibility Criteria

Sex
ALL
Age
2 Years - 20 Years
Healthy Volunteers
Not accepted

Interventions

  • Venetoclax — DRUG
    Venetoclax will be given as oral tablets, which are intended to be swallowed intact and may not to be crushed or otherwise altered for administration, or as an oral suspension for patients who cannot swallow tablets.
  • Cytarabine — DRUG
    Given intravenously (IV) or intrathecally (IT).
  • Idarubicin — DRUG
    Given IV.
  • Intrathecal Triple Therapy — DRUG
    Given IT.
  • Azacitidine — DRUG
    Given IV.

Study Details

The purpose of this study is to test the safety and determine the best dose of venetoclax and cytarabine when given with or without idarubicin in treating pediatric patients with acute myeloid leukemia (AML) that did not respond to treatment (refractory) or has come back after treatment (relapsed). PRIMARY OBJECTIVE: Determine a tolerable combination of venetoclax plus chemotherapy in pediatric patients with relapsed or refractory AML or acute leukemia of ambiguous lineage. The primary endpoints are the recommended phase 2 doses (RP2D) of venetoclax plus cytarabine and venetoclax plus cytarabine and idarubicin. SECONDARY OBJECTIVE: Estimate the overall response rate to the combination of venetoclax and chemotherapy in pediatric patients with relapsed or refractor AML or acute leukemia of ambiguous lineage. The secondary endpoints are the rates of complete remission (CR) and complete remission with incomplete count recovery (CRi) for patients treated at the RP2D.

Key Dates

Start date
Jul 11, 2017
Status verified
Feb 2023
Primary completion
Oct 27, 2020
Completion
Jun 22, 2022

Study Design

Enrollment
62 participants (actual)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Treatment
    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.

Primary Outcome Measure

Maximum tolerated combination (MTC) [ Time Frame: 28 days after start of therapy ]

Locations (3)

FacilityCityStateZIPSite coordinators
Lucille Packard Children's Hospital Stanford UniversityPalo AltoCalifornia94304-
UNC Lineberger Comprehensive Cancer CenterChapel HillNorth Carolina27599-
St. Jude Children's Research HospitalMemphisTennessee38105-

Find similar trials in Palo Alto, CA

By condition
Leukemia (other)
By specialty
OncologyBlood cancer
By research site
Lucille Packard Children's Hospital Stanford University· Palo Alto, CAUNC Lineberger Comprehensive Cancer Center· Chapel Hill, NCSt. Jude Children's Research Hospital· Memphis, TN

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