A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies

  • End date
    Jun 2, 2023
  • participants needed
  • sponsor
Updated on 4 October 2022
bone marrow procedure
primary cancer
solid tumour
solid neoplasm


An open-label, global, multi-center study to evaluate the safety and pharmacokinetics of venetoclax monotherapy, to determine the dose limiting toxicity (DLT) and the recommended Phase 2 dose (RPTD), and to assess the preliminary efficacy of venetoclax in pediatric and young adult participants with relapsed or refractory malignancies.

Condition Malignancies, Acute Lymphoblastic Leukemia (ALL), Acute Myeloid Leukemia (AML), Non-Hodgkin's Lymphoma, Neuroblastoma
Treatment Chemotherapy, venetoclax
Clinical Study IdentifierNCT03236857
Last Modified on4 October 2022


Yes No Not Sure

Inclusion Criteria

Participants must have relapsed or refractory cancer
Participants must have adequate hepatic and kidney function
Participants less than or equal to 16 years of age must have performance status of Lansky greater than or equal to 50% and participants greater than 16 years of age must have performance status of Karnofsky greater than or equal to 50%
Participants with solid tumors (with the exception of neuroblastoma) must have adequate bone marrow function in Part 1
For the fifth cohort during Part 2 Cohort Expansion, participants with solid tumors must have evidence of BCL-2 expression (except participants with TCF3-HLF ALL)

Exclusion Criteria

Participants with primary brain tumors or disease metastatic to the brain
Participants who have central nervous system (CNS) disease with cranial involvement that requires radiation
Participants who have received any of the following within the listed time frame, prior to the first dose of study drug
Inotuzumab ozogamicin or gemtuzumab ozogamicin within 30 days
Biologic agent (i.e., antibodies) for anti-neoplastic intent within 30 days or 5 half-lives whichever is shorter
CAR-T infusion or other cellular therapy within 30 days
Anticancer therapy including chemotherapy, radiation therapy, targeted small molecule agents, investigational agents within 14 days or 5 half-lives, whichever is shorter (Exceptions: Ph+ALL participants on Tyrosine Kinase Inhibitor (TKI) at Screening may enroll and remain on TKI therapy to control disease and TCF3-HLF ALL participants are allowed to have received chemotherapy within 14 days or 5 half-lives, whichever is shorter)
Steroid therapy for anti-neoplastic intent within 5 days (with the exception of TCF3-HLF ALL participants)
Requires ongoing hydroxyurea (hydroxyurea permitted up to first dose)
Participants who are less than 100 days post-transplant, or greater than or equal to
days post-transplant with active graft versus host disease (GVHD), or are
Participants who are less than 6 weeks post-131 I-metaiodobenzylguanidine (mIBG) therapy
receiving immunosuppressant therapy within 7 days prior to first dose of study
Participants who have received the following within 7 days prior to the first dose of study drug
Strong and moderate Cytochrome P450 3A (CYP3A) inhibitors (Part 1 Dose Determination)
Strong and moderate CYP3A inducers (Part 1 Dose Determination and Part 2 Cohort Expansion)
Participants who have active, uncontrolled infections
Participants with malabsorption syndrome or any other condition that precludes enteral administration
Participants with recent positive test for SARS-CoV-2 (COVID-19) and no follow up test with negative result cannot be enrolled. Participants with contact to persons with COVID-19 and participants with signs and symptoms for COVID-19 infection must be tested before enrolling
Participants who have not recovered from clinically significant adverse
effect(s)/toxicity(s) of the previous therapy (Exception: Chemotherapy induced
side effects that are expected to return to baseline in TCF3-HLF ALL
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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