A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
DFCI Protocol ID: 17-617
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.
Children's Hospital Boston, Dana-Farber Cancer Institute
Andrew Place, MD, PhD,
Dana Farber Cancer Institute
Dana-Farber Cancer Institute:
Childrens Hospital Pediatric Clinical Translation Investigation Program CTIP, firstname.lastname@example.org
- 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 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.
- Participants with primary brain tumors or disease metastatic to the brain.
- For participants with leukemia, has overt central nervous system (CNS) disease (CNS 3
- Participants who have received any of the following within the listed time frame,
prior to the first dose of study drug
- Biologic agent (i.e., antibodies) for anti-neoplastic intent within 30 days
- CAR-T infusion or other cellular therapy within 30 days
- Anticancer therapy including blinatumomab or chemotherapy, radiation therapy,
targeted small molecule agents, investigational agents within 14 days or 5
half-lives, whichever is shorter
- Steroid therapy for anti-neoplastic intent within 5 days
- Requires ongoing hydroxyurea (hydroxyurea permitted up to first dose)
- Participants who are less than 100 days post-transplant, or greater than or equal to
100 days post-transplant with active graft versus host disease (GVHD), or are
receiving immunosuppressant therapy within 7 days prior to first dose of study drug.
- Participants who are less than 6 weeks post-131 I-metaiodobenzylguanidine (mIBG)
- Participants who have received the following within 7 days prior to the first dose of
- Strong and moderate Cytochrome P450 3A (CYP3A) inhibitors (Part 1 Dose
- Strong and moderate CYP3A inducers (Part 1 Dose Determination and Part 2 Cohort
- Participants who have not recovered from clinically significant adverse
effect(s)/toxicity(s) of the previous therapy.
- Participants who have active, uncontrolled infections.
- Participants with malabsorption syndrome or any other condition that precludes enteral