Actively Recruiting
A Phase I Study of Venetoclax with Chemotherapy Including Calaspargase Pegol for Children, Adolescents, and Young Adults with High-Risk Hematologic Malignancies
Led by Andrew E. Place, MD · Updated on 2026-03-12
30
Participants Needed
5
Research Sites
104 weeks
Total Duration
On this page
Sponsors
A
Andrew E. Place, MD
Lead Sponsor
A
AbbVie
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are studying the safety and tolerability of venetoclax combined with chemotherapy in children and young adults with high-risk blood cancers, including myelodysplastic syndrome (MDS), acute myeloid leukemia (AML) derived from MDS, and acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL). This phase I trial aims to find the safest dose of venetoclax that can be given with standard chemotherapy drugs. The study includes three separate patient groups based on their specific blood cancer type and genetic risk factors. Participants receive different drug combinations depending on their disease group. In Cohorts A and B (MDS/AML patients), treatment cycles last about 28 days and include venetoclax and azacitidine daily on set days, with additional chemotherapy drugs given if leukemia cells are detected in the spinal fluid. Cohort B involves patients with genetic conditions increasing treatment toxicity risk. Cohort C includes patients with relapsed or refractory ALL or LBL, receiving a single 32-day treatment cycle with venetoclax plus several chemotherapy drugs including dexamethasone, vincristine, doxorubicin, and calaspargase pegol, with adjustments for allergies. Participants undergo screening, treatment visits, and follow-up assessments throughout the study. Researchers monitor the maximum tolerated dose, treatment-related side effects, and response to therapy. Outcome measures include remission rates, overall survival, and event-free survival up to two years. Safety is carefully tracked during and after treatment, including monitoring for toxicities related to specific drugs. The total participation time depends on the cohort and treatment cycles assigned.
CONDITIONS
Brief Title
Venetoclax Basket Trial for High Risk Hematologic Malignancies
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of MDS, AML arising from MDS, therapy-related myeloid neoplasm, or relapsed/refractory acute lymphoblastic leukemia or lymphoblastic lymphoma meeting specific disease criteria
- Age between 1 and 40 years, with some groups limited to under 18 or 21 years
- Lansky or Karnofsky performance status of 50% or higher
- Fully recovered from prior chemotherapy, immunotherapy, or radiation effects
- Adequate organ function including liver and heart function
- Female participants of childbearing potential must have a negative pregnancy test and agree to use effective contraception during and after the study
- No active graft-versus-host disease after stem cell transplant
- Specific genetic conditions allowed for certain cohorts
You will not qualify if you...
- Use of strong or moderate CYP3A inhibitors or inducers within 3 days before study entry
- Active graft-versus-host disease or ongoing treatment for GVHD
- Known active hepatitis or uncontrolled systemic infection
- Known HIV infection
- Pregnant or nursing women
- Significant other illnesses or psychiatric conditions that interfere with study participation
- Allergic reactions to study drugs (except some asparaginase allergies with alternatives allowed)
- History of asparaginase-associated pancreatitis
- Active deep venous thrombosis
- Isolated CNS or testicular relapse
- Certain genetic or disease features excluding participation
- Prior malignancy unless disease-free for at least 1 year or specific low-risk cancers treated within the past year
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 4 cycles of approximately 28 days each (Cohorts A & B) or 1 cycle of approximately 32 days (Cohort C)
Participants receive venetoclax combined with chemotherapy drugs including azacitidine, and depending on their disease and spinal fluid involvement, additional drugs such as cytarabine, methotrexate, hydrocortisone, and leucovorin. Treatment cycles vary by cohort but involve daily dosing on predetermined days per protocol. Cohorts A and B may have up to 4 cycles of about 28 days each, while Cohort C receives a single cycle of approximately 32 days.
Multiple visits per cycle as scheduled by the protocol
Duration - Up to 30 days after last dose
Participants are monitored for safety and treatment-related toxicities up to 30 days after their last dose of study treatment.
Visits as needed for safety assessments during follow-up period
Trial Site Locations
Total: 5 locations
1
University of California San Francisco-Benioff Children's Hospital
San Francisco, California, United States, 94158
Actively Recruiting
2
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
3
Children's Healthcare of Atlanta at Arthur M. Blank Hospital
Atlanta, Georgia, United States, 30329
Actively Recruiting
4
Ann & Robert H Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
5
Dana-Farber Cancer Institute
Boston, Massachusetts, United States, 02215
Actively Recruiting
Research Team
A
Andrew E Place, MD, PhD
J
Jessica A Pollard, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
3
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