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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
143
Dexamethasone and/or vincristine and/or pegasparaginase OR cytarabine and/or etoposide and/or pegasparaginase; tyrosine kinase inhibitor; cytarabine OR azacitidine OR decitabine; rituximab and/or dexamethasone and/or vincristine; cyclophosphamide and/or topotecan
Oral tablet for participants; Tablet for oral suspension (participants who cannot swallow a tablet)
Univ California, San Francisco /ID# 163460
San Francisco, California, United States
Children's Hospital Colorado /ID# 161551
Aurora, Colorado, United States
Children's Healthcare of Atlan /ID# 161552
Atlanta, Georgia, United States
Dana-Farber Cancer Institute /ID# 163440
Boston, Massachusetts, United States
Memorial Sloan Kettering Cancer Center-Koch Center /ID# 163444
New York, New York, United States
Number of Participants Experiencing Adverse Events
An adverse event (AE) is defined as any untoward medical occurrence in a patient or clinical investigation participant administered a pharmaceutical product which does not necessarily have a causal relationship with this treatment. The investigator assesses the relationship of each event to the use of study.
Time frame: Up to 9 months
Number of Participants With Dose Limiting Toxicities (DLT) of Venetoclax Monotherapy
A DLT is any Grade 3 or higher non-hematologic adverse event (AE) with exceptions outlined in the protocol.
Time frame: First 21 days venetoclax monotherapy
Recommended Phase 2 dose (RPTD) of Venetoclax
Venetoclax RPTD is the dose determined based on adverse event reporting and dose-limiting toxicity information from all participants.
Time frame: First 21 days venetoclax monotherapy
Cmax of Venetoclax
Maximum plasma concentration (Cmax) of venetoclax.
Time frame: Up to approximately 2 weeks
Tmax of venetoclax
Time to maximum plasma concentration (Tmax) of venetoclax.
Time frame: Up to approximately 2 weeks
AUC0-24 Post-Dose of Venetoclax
Area under the plasma concentration-time curve from 0 to 24 hours (AUC24) post-dose of venetoclax.
Time frame: Up to approximately 2 weeks
Objective Response Rate (ORR)
ORR is defined as the proportion of participants who achieved a response according to established criteria described in detail in the study protocol.
Time frame: Up to 9 months
Partial Response (PR) Rate
PR is defined according to established criteria for each tumor type and is described in detail within the study protocol.
Time frame: Up to 9 months
Complete Response (CR) Rate
CR is defined according to established criteria for each tumor type and is described in detail within the study protocol.
Time frame: Up to 9 months
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Cincinnati Children's Hospital /ID# 161550
Cincinnati, Ohio, United States
Children's Hospital of Philadelphia /ID# 163445
Philadelphia, Pennsylvania, United States
St Jude Children's Research Hospital /ID# 163447
Memphis, Tennessee, United States
Primary Children's /ID# 164399
Salt Lake City, Utah, United States
Seattle Children's Hospital /ID# 163459
Seattle, Washington, United States
...and 21 more locations