MB-HUCART19.1 for B-cell precursor acute lymphoblastic leukemia | B-cell non-Hodgkin lymphoma

Inclusion criteria

• {"criterion_text":"- 1-45 years of age"}
• {"criterion_text":"- Additional inclusion criteria phase I part of the study: The first three patients in the phase 1 part of the study must be aged 12-45 years, thereafter patients of any age between 1-45 years can be recruited once surrogate endpoint of B-cell Aplasia is reached in ≥60% patients in previous or current dose level."}
• {"criterion_text":"- Patients with relapsed or refractory CD19+ hematological malignancies (a.o. B-NHL and B-cell precursor)"}
• {"criterion_text":"- Measurable disease (at least one measurable lesion or at least 0.1% of blast in bone marrow)"}
• {"criterion_text":"- Patients must have exhausted or are ineligible for all registered therapeutic options with curative potential."}
• {"criterion_text":"- Adequate performance score"}
• {"criterion_text":"- Patients from childbearing potential must be willing and able to use highly effective methods of birth control from first chemotherapy infusion through 12 months after administering the last study treatment"}
• {"criterion_text":"- Patients must be willing to abstain from breast feeding through 12 months after administering the last study treatment."}
• {"criterion_text":"- Patients must agree to refrain from donating blood or organs following treatment with huCAR19 T-cells."}
• {"criterion_text":"- Written informed consent per local law and regulations."}

Exclusion criteria

• {"criterion_text":"- Patients with symptomatic CNS involvement will be excluded. After resolution and control of symptoms, patients can be rescreened."}
• {"criterion_text":"- Concurrent malignancy requiring treatment of having been treated <3 months before screening except for curatively treated basal cell carcinoma of the skin"}
• {"criterion_text":"- Pregnant women"}
• {"criterion_text":"- Patients unable to participate in the study according to investigator judgement"}
• {"criterion_text":"- Patients not willing or unable to adhere to protocol guidelines or follow-up."}
• {"criterion_text":"- Treatment with allogeneic stem cell transplantation <12 weeks from screening or DLI <4 weeks from screening or active GVHD requiring systemic treatment. Cutaneous GVHD requiring only topical steroids is allowed."}
• {"criterion_text":"- Hypersensitivity to the active substance"}
• {"criterion_text":"- Active uncontrolled or life-threatening infections"}
• {"criterion_text":"- Infection with HTLV-1, HTLV-2, HIV-1, HIV-2, hepatitis B (HbsAg positive) or hepatitis C (anti-HCV positive). Chronic controlled hepatitis B or C infection with undetectable viral load or controlled HIV infection with viral load <50 IU/ml and CD4+ T-cell count >200/ml may be considered when antiviral prophylaxis or therapy can be administered."}
• {"criterion_text":"- Absolute neutrophil count <0.5x10E9/L unless caused by underlying disease"}
• {"criterion_text":"- Platelet count <25x10E9/L unless caused by underlying disease"}
• {"criterion_text":"- Bilirubin and/or transaminases ≤ 2.5 x ULN, unless caused by underlying disease."}
• {"criterion_text":"- Renal insufficiency"}
• {"criterion_text":"- Inadequate pulmonary function defined as baseline oxygen saturation <92%, if not caused by underlying disease."}
• {"criterion_text":"- Inadequate cardiac function"}

Primary endpoints

• {"endpoint_text":"- Dose at which ≤ 1 patients experience a Dose Limiting Toxicity (DLT) within 28 days after CAR T-cell infusion","definition_or_measurement_approach":"DLT assessed within 28 days after CAR T-cell infusion; the RP2D determined from incidence of DLT within 28 days."}

Secondary endpoints

• {"endpoint_text":"- For B-ALL: the MRD-negative CR rate at day 28","definition_or_measurement_approach":"MRD-negative complete response assessed at day 28 (specific MRD methods not detailed)."}
• {"endpoint_text":"- For B-NHL: the overall response rate (ORR, CR +PR according to Lugano criteria) at day 90","definition_or_measurement_approach":"ORR assessed at day 90 using Lugano criteria (CR + PR)."}
• {"endpoint_text":"- Surrogate endpoint for efficacy: B-cell aplasia (<5 B-cells/µl) at day 28 after infusion","definition_or_measurement_approach":"B-cell aplasia defined as <5 B-cells/µl measured at day 28 after infusion."}
• {"endpoint_text":"- Number of days until relapse","definition_or_measurement_approach":"Time in days from relevant baseline/timepoint until documented relapse."}
• {"endpoint_text":"- Number of days from CAR T-cell infusion until recovery of B-cells (B-cell recovery is defined as peripheral blood ≥10 CD19+ B-cells/mcL OR ≥1% CD19+ B-cells in the bone marrow. Results must be confirmed on a subsequent test ≥2 weeks apart with timing of B-cell recovery defined as the date of the initial sample","definition_or_measurement_approach":"B-cell recovery defined as peripheral blood ≥10 CD19+ B-cells/mcL OR ≥1% CD19+ B-cells in bone marrow; confirmation on a subsequent test ≥2 weeks apart; timing defined as date of initial sample."}
• {"endpoint_text":"- Event free survival (EFS) at 6 and 12 months","definition_or_measurement_approach":"EFS defined with event as relapse, refractory disease or death of any cause (whichever occurs first); assessed at 6 and 12 months."}
• {"endpoint_text":"- Overall Survival (OS) at 6 and 12 months","definition_or_measurement_approach":"Overall survival assessed at 6 and 12 months."}
• {"endpoint_text":"- Cumulative Incidence of Relapse at 6 and 12 months","definition_or_measurement_approach":"Cumulative incidence of relapse measured at 6 and 12 months."}
• {"endpoint_text":"- The percentage of products fulfilling the release criteria","definition_or_measurement_approach":"Proportion of manufactured products meeting predefined release criteria (release criteria not detailed)."}

Netherlands

Earliest CTIS Part Ii Submission Date

15-09-2025

Latest Decision Or Authorization Date

12-01-2026

Processing Time Days

119

Number Of Sites

2

Number Of Participants

18

Primary sponsor

Full Name

Prinses Maxima Centrum voor Kinderoncologie B.V.

Organisation Type

Hospital/Clinic/Other health care facility

Country Of Registered Address

Netherlands

Contract research organisations

Name

Julius Clinical International B.V.

Responsibilities

sponsorDuties code: 1

Third parties

• {"country":"Netherlands","full_name":"Julius Clinical International B.V.","duties_or_roles":"sponsorDuties code: 1","organisation_type":"Pharmaceutical company"}