A Study of the Safety and Pharmacokinetics of Venetoclax in Pediatric and Young Adult Patients With Relapsed or Refractory Malignancies
Trial Status: Active
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.
- 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.
- 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 within 30 days
- 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 (Exceptions: Ph+ALL participants on Tyrosine Kinase Inhibitor (TKI) at Screening may enroll and remain on TKI therapy to control disease).
- 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) therapy.
- 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 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 administration.
UCSF Medical Center-Mount Zion
Contact: UCSF Clinical Trials
Children's Hospital Colorado
Children's Healthcare of Atlanta - Egleston
Boston Children's Hospital
Dana-Farber Cancer Institute
Memorial Sloan Kettering Cancer Center
Saint Jude Children's Research Hospital
Contact: Sara Michele Federico
Salt Lake City
Primary Children's Hospital
Seattle Children's Hospital
Trial Phase Phase I
Trial Type Treatment
- Primary ID M13-833
- Secondary IDs NCI-2017-02027, 2017-000439-14
- Clinicaltrials.gov ID NCT03236857