Safety and Efficacy Study of Zamtocabtagene Autoleucel (MB-CART2019.1) in Pediatric Patients With R/R B-Cell Neoplasms

NCT06508931 | Recruiting Phase 2 FDA Drug

• 2 other identifiers: M-2021-3892023-506348-17-00
• Study Type: interventional
• Target: 31
• Locations: 4 countries
• Sites: 5

Brief Summary

This is a single-arm, multi-center, open-label Phase II study to determine the safety and efficacy of MB-CART2019.1 in pediatric and adolescent subjects (aged between 6 months and \<18 years, ≥6 kg body weight \[BW\]) with mature B-cell neoplasms and aggressive lymphomas that relapsed after or are refractory to one or more prior therapies, including subjects with primary refractory disease.

Conditions

B-Cell Neoplasm

Outcome Measures

Primary Outcomes (2)

• BORR, Best Overall Response Rate

  The primary efficacy outcome of this study is BORR, defined as the proportion of subjects with at least one partial response (PR) or complete response (CR) from MB-CART2019.1 infusion until progressive disease (PD), start of new anti-lymphoma therapy, lost to follow-up or death, whichever occurs first.

  From infusion until week 78

• Type, frequency, and severity of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESI) [Safety and toxicity of the study product]

  The primary safety outcome of this study is to evaluate the incidence, type, frequency, and severity of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESI) associated with the study product.

  From infusion until week 78

Study Arms (1)

zamtocabtagene autoleucel (MB-CART2019.1)

EXPERIMENTAL

Drug: zamtocabtagene autoleucel (MB-CART2019.1)

Interventions

zamtocabtagene autoleucel (MB-CART2019.1) DRUG

Tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy.

zamtocabtagene autoleucel (MB-CART2019.1)

Eligibility Criteria

• Age: 6 Months - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Is able to provide age-appropriate assent/consent (as applicable, according to local legislation) and/or have a guardian able to provide consent signed and dated by the parent(s) or by subject's legal guardian before conduct of any study-specific procedures.
• Has histologically confirmed mature CD19+ and/or CD20+ B-cell neoplasm such as:
• Burkitt lymphoma/Burkitt leukemia
• Diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS)
• Primary mediastinal (thymic) large B-cell lymphoma
• Burkitt-like lymphoma with 11q aberration
• Aggressive mature B-cell lymphoma
• Other rare aggressive B-cell non-Hodgkin lymphoma (NHL) after sponsor approval.
• Has r/r B-cell neoplasms after one or more prior therapies or primary refractory to first-line therapy.
• Is a pediatric/adolescent (aged between 6 months and \<18 years).
• Has a BW of ≥ 6 kg.
• Measurable disease based on the International Pediatric NHL Response Criteria (which refers to the Lugano criteria for definitions of measurability and selecting index lesions), as identified by local radiological assessment for lymphomas. Previously irradiated lesions cannot be considered measurable unless the lesion has proven radiological evidence for progression after the radiation.
• Tissue samples archival or fresh (preferred) from recent relapse or initial diagnosis (in case of primary refractory disease) must be made available for the central pathology review to confirm diagnosis (≤2 years, preferably not older than 2 months since collection).
• Has Karnofsky (aged ≥16 years) or Lansky (aged \<16 years) performance status ≥60.
• Has adequate bone marrow function as defined by the following laboratory values (as assessed by local laboratory for eligibility):

You may not qualify if:

• Is receiving active treatment for malignant disease (including participation in any additional parallel investigational drug or device studies), except for pre-enrollment therapy, including radiotherapy. Lesions that are irradiated during pre-enrollment therapy may not be considered measurable lesions. For subjects with lymphoma to be eligible, there must be at least one measurable lesion after pre-enrollment therapy.
• Had allogeneic HSCT.
• Had autologous HSCT \<120 days prior to written informed consent.
• Had major surgery within 2 weeks before leukapheresis, or has not fully recovered from an earlier surgery, or has major surgery planned during the time the subject is expected to participate in the study.
• Subjects with B-cell neoplasms in the context of post-transplant lymphoproliferative disorders-associated lymphomas.
• Has known hypersensitivity to the excipients of the MB-CART2019.1 or to any other drug product as advised for administration in the study protocol (e.g., lymphodepleting agents).
• Has active central nervous system (CNS) involvement at the time point of eligibility confirmation, as measured by the presence of lymphoma cells in cerebral spinal fluid (CSF) on cytospin preparation.
• Has history or presence of autoimmune CNS disease, such as multiple sclerosis, optic neuritis, or other immunologic or inflammatory diseases.
• Infection with human immunodeficiency virus (HIV).
• Presence of active or prior hepatitis B or C as indicated by serology. Treated infection with hepatitis B or C virus unless confirmed to be polymerase chain reaction (PCR) negative.
• Has infection with Treponema pallidum.
• Has active infection with severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2).
• Has infection with human T-lymphotropic virus 1/2 (HTLV 1/2).
• Has active severe systemic fungal, viral, or bacterial infection, requiring systemic antiviral, antifungal, or antimicrobial therapy.
• Has clinically significant seizures according to the opinion of by the investigator.

Sponsors & Collaborators

• Miltenyi Biomedicine GmbH: lead
• PPD, Part of Thermo Fisher Scientific: collaborator

Study Sites (5)

Hôpital Robert Debré

Paris, Boulevard Sérurier 48, 75019, France

ACTIVE NOT RECRUITING

Institut Gustave Roussy

Villejuif, 94805, France

RECRUITING

Universitaetsklinikum Muenster (UKM) - Klinik fuer Kinder- und Jugendmedizin - Paediatrische Haematologie und Onkologie

Münster, Albert-Schweitzer-Campus 1, Gebaeude A1, 48129, Germany

ACTIVE NOT RECRUITING

IRCCS Ospedale Pediatrico Bambino Gesù

Rome, Piazza Sant Onofrio 4, 00165, Italy

RECRUITING

Prinses Maxima Centrum

Utrecht, Heidelberglaan 25, Netherlands

ACTIVE NOT RECRUITING

MeSH Terms

Conditions

Lymphoma, B-Cell

Condition Hierarchy (Ancestors)

Lymphoma, Non-Hodgkin; Lymphoma; Neoplasms by Histologic Type; Neoplasms; Lymphoproliferative Disorders; Lymphatic Diseases; Hemic and Lymphatic Diseases; Immunoproliferative Disorders; Immune System Diseases

Study Officials

• Birgit Burkhardt, Prof. Dr.

  Uniklinik Münster, Klinik für Kinder- und Jugendmedizin Pädiatrische Hämatologie/Onkologie

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Andrey Shirokov

CONTACT

+49 151 1179 4851; andrey.shirokov@miltenyi.com

Gregor Zadoyan, Dr.

CONTACT

+49 151 4025 8225; gregor.zadoyan@miltenyi.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A multi-center, international, open-label study

Study Record Dates

• First Submitted: July 2, 2024
• First Posted: July 18, 2024
• Study Start: August 4, 2025
• Primary Completion (Estimated): December 31, 2029
• Study Completion (Estimated): December 31, 2029
• Last Updated: May 5, 2026

Record last verified: 2025-09

Data Sharing

• IPD Sharing: Will not share

Locations

FR (2); IT (1); DE (1); NL (1)