FLUDARABINE PHOSPHATE for B-cell acute lymphoblastic leukemia

Inclusion criteria

• {"criterion_text":"- Patients with B-ALL and eligible to receive commercial tisagenlecleucel.\n- Patient’s weight >9kg at time of screening\n- Adequate organ function at time of LD is required and is defined: Hepatic: Serum bilirubin ≤ 2 mg/dL, unless benign congenital hyperbilirubinemia, Hepatic: AST and ALT <5x the upper limit of normal for age, unless thought to be leukemic disease-related, Renal: Calculated glomerular filtration rate (GFR) 70 ml/min/1.73m2. (based on Schwartz formula GFR (mL/min/1.73 m²) = (36.2×Height in cm) / Creatinine in µmol/L, Cardiac: LVEF ≥50% by multi-gated acquisition scan (MUGA), resting echocardiogram, or cardiac magnetic resonance imaging (MRI) within 6 weeks of screening, Pulmonary: Oxygen saturation as recorded by pulse oximetry of ≥90% on room air\n- Adequate performance status: Age ≥16 years: ECOG ≤1 or Karnofsky >60% at screening, Age <16 years: Lansky >60% at screening\n- Willing to participate as research subject and provide written informed consent from parents/legal representative, patient, and age-appropriate assent as appropriate before any study specific screening procedures are conducted, according to local, regional or national law and legislation."}

Exclusion criteria

• {"criterion_text":"- Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to the study drugs, or drugs chemically related to study treatment or excipients that contraindicate their participation, including fludarabine, cyclophosphamide and tisagenlecleucel.\n- Patients with tisagenlecleucel that is deemed out of specification (OOS) will be excluded from this protocol\n- Clinically significant active and uncontrolled infection confirmed by clinical evidence, imaging, or positive laboratory tests (e.g., blood cultures, PCR for DNA/RNA etc.)\n- Patient/parent/guardian unable to give informed consent or unable to comply with the treatment protocol.\n- Pregnant or lactating women\n- Other uncontrolled, symptomatic, intercurrent illness including but not limited to infection, psychiatric illness, or social situations that would limit compliance with study requirements or in the opinion of the PI would pose an unacceptable risk to the subject."}

Primary endpoints

• {"endpoint_text":"- EFS is defined as time from randomization until non-response at day 28 after CAR T cell infusion, loss of B-cell aplasia <6 months from the time of CAR T cell infusion, disease relapse, initiation of anti-leukemic therapy or death of any cause","definition_or_measurement_approach":"EFS is defined as time from randomization until non-response at day 28 after CAR T cell infusion, loss of B-cell aplasia <6 months from the time of CAR T cell infusion, disease relapse, initiation of anti-leukemic therapy or death of any cause"}

Secondary endpoints

• {"endpoint_text":"- Death from any cause from the day of randomization.","definition_or_measurement_approach":"All-cause mortality measured from day of randomization."}
• {"endpoint_text":"- Non-response at 28 days","definition_or_measurement_approach":""}
• {"endpoint_text":"- Cumulative incidence of relapse is defined as time from CAR T cell infusion until disease relapse in patients with a morphological complete response on day 28","definition_or_measurement_approach":"Time from CAR T cell infusion to disease relapse in patients with morphological complete response at day 28."}
• {"endpoint_text":"- Cumulative incidence of initiation of anti-leukemic therapy during the first two years after CAR T cell infusion","definition_or_measurement_approach":"Time to initiation of any anti-leukemic therapy within 2 years after CAR T infusion; cumulative incidence estimated over first two years."}
• {"endpoint_text":"- Type, incidence, severity, seriousness and relationship of CRS, ICANS, IEC-HS, cytopenia day 28 – day 180 (grade 3-4), and infectious adverse events (grade ≥3) during the first 180 days after CAR T cell infusion","definition_or_measurement_approach":"Adverse events graded by severity and relationship; specific AESIs (CRS, ICANS, IEC-HS) and specified cytopenias/infections monitored from day 28 to day 180 post-infusion."}
• {"endpoint_text":"- Duration of B-cell aplasia and CAR T cell numbers from the day of CAR T cell infusion until 1 year","definition_or_measurement_approach":"Measured duration of B-cell aplasia and serial CAR T cell counts from infusion up to 1 year."}
• {"endpoint_text":"- Duration of B-cell aplasia in the bone marrow from the day of CAR T cell infusion until 6 months after CAR T cell infusion","definition_or_measurement_approach":"Measured duration of bone marrow B-cell aplasia from infusion up to 6 months."}
• {"endpoint_text":"- Definition of accuracy of fludarabine therapeutic drug monitoring (percentage of patients within the target of 18 mg*h/L, range 17.5-18.5 mg*h/L)","definition_or_measurement_approach":"Percentage of patients achieving target fludarabine AUC of 18 mg*h/L (acceptable range 17.5-18.5 mg*h/L) by TDM."}
• {"endpoint_text":"- Patient-reported outcome measures","definition_or_measurement_approach":""}

Netherlands

Earliest CTIS Part Ii Submission Date

26-03-2026

Latest Decision Or Authorization Date

16-04-2026

Processing Time Days

21

Number Of Sites

1

Number Of Participants

10

Germany

Earliest CTIS Part Ii Submission Date

22-01-2026

Latest Decision Or Authorization Date

16-04-2026

Processing Time Days

84

Number Of Sites

1

Number Of Participants

10

Spain

Earliest CTIS Part Ii Submission Date

09-04-2026

Latest Decision Or Authorization Date

27-04-2026

Processing Time Days

18

Number Of Sites

1

Number Of Participants

15

Primary sponsor

Full Name

Prinses Maxima Centrum voor Kinderoncologie B.V.

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands