Actively Recruiting
Improving Event Free Survival by Optimizing Fludarabine Exposure During Lymphodepletion for CAR T-cell Therapy: a Randomized Multi-center Study of Children and Young Adults With B-cell Acute Lymphoblastic Leukemia
Led by Memorial Sloan Kettering Cancer Center · Updated on 2026-06-04
130
Participants Needed
3
Research Sites
N/A
Total Duration
On this page
Sponsors
M
Memorial Sloan Kettering Cancer Center
Lead Sponsor
P
Princess Maxima Center for Pediatric Oncology
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating whether pharmacokinetics-targeted fludarabine dosing is an effective lymphodepletion chemotherapy method for children and young adults with relapsed or refractory B-cell acute lymphoblastic leukemia (B-ALL) who will receive tisagenlecleucel CAR T-cell therapy. The study compares targeted fludarabine dosing with the standard dosing approach to determine which is more effective, while also assessing side effects and quality of life using questionnaires. Participants will be randomly assigned to one of two treatment groups: the standard fludarabine regimen, which involves 30 mg/m2 per dose given four times over days -6 to -3 (or -7 to -4), or the targeted fludarabine regimen, which starts with 40 mg/m2 doses on days -6 and -5 (or -7 and -6) with adjusted doses on subsequent days based on pharmacokinetic analysis to achieve a specific drug exposure target. All participants will also receive cyclophosphamide 500 mg/m2 intravenously on days -6 and -5 (or -7 and -6). Following chemotherapy, participants will receive CAR T-cell therapy following institutional guidelines. During the study, participants undergo regular assessments including evaluations of organ function, performance status, and oxygen levels before lymphodepleting chemotherapy. Researchers monitor event-free survival at 28 days and overall survival up to two years. Quality of life is measured through questionnaires. Participants will be followed and observed throughout the treatment and post-treatment periods to assess treatment effects and safety. The total participation duration spans from chemotherapy through long-term survival monitoring.
CONDITIONS
Brief Title
A Study of Fludarabine Dosing in Children and Young Adults With B-cell Acute Lymphoblastic Leukemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with B-cell acute lymphoblastic leukemia eligible to receive commercial tisagenlecleucel
- Patient's weight greater than 9 kg at time of lymphodepleting chemotherapy
- Adequate liver function: Serum bilirubin ≤ 2 mg/dL unless benign congenital hyperbilirubinemia
- Adequate liver enzymes: AST and ALT less than 5 times the upper limit of normal for age unless leukemic disease-related
- Adequate kidney function: GFR ≥ 70 ml/min/1.73m2 based on Schwartz formula
- Adequate heart function: LVEF ≥ 50% by MUGA, echocardiogram, or cardiac MRI within 6 weeks of screening
- Adequate lung function: Oxygen saturation ≥ 90% on room air
- Adequate performance status: ECOG ≤ 1 or Karnofsky > 60% if age 16 or older; Lansky ≥ 60% if under 16
- Willing to participate and provide written informed consent and assent as appropriate
You will not qualify if you...
- Known allergic reaction or intolerance to fludarabine, cyclophosphamide, tisagenlecleucel, or related drugs
- Patients with out of specification (OOS) tisagenlecleucel
- Active uncontrolled infection confirmed by clinical or laboratory evidence
- Unable to provide informed consent or comply with study treatment
- Pregnant or breastfeeding women
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 screening and enrollment visit (in-person)
Duration - Approximately 1 week
Participants receive lymphodepleting chemotherapy with fludarabine and cyclophosphamide followed by CAR T-cell therapy according to their assigned regimen.
Daily visits during lymphodepletion and CAR T-cell infusion period
Duration - Up to 2 years
Participants are monitored for event-free survival and overall survival after treatment.
Regular follow-up visits for up to 2 years
Trial Site Locations
Total: 3 locations
1
Memorial Sloan Kettering Cancer Center
New York, New York, United States, 10065
Actively Recruiting
2
Cincinnati Children's Hospital Medical Center (Data Collection Only)
Cincinnati, Ohio, United States, 45229
Actively Recruiting
3
Children's Hospital of Philadelphia (Data Collection Only)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
Research Team
K
Kevin Curran, MD
J
Jaap Jan Boelens, MD, PhD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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