B-Cell NeoplasmPhase 2DALY PEDNCT06508931
Safety and Efficacy Study of Zamtocabtagene Autoleucel (MB-CART2019.1) in Pediatric Patients With R/R B-Cell Neoplasms
A Phase 2 study testing zamtocabtagene autoleucel (MB-CART2019.1) in people with B-Cell Neoplasm, run by Miltenyi Biomedicine GmbH at 5 sites in 4 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 6 months to 17 years
- Conditions the study is for
- B-Cell Neoplasm
- What is being tested?
- zamtocabtagene autoleucel (MB-CART2019.1)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 31 participants
- Where
- 5 sitesin 4 countries
- Study dates
- 4 Aug 2025to 31 Dec 2029
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
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- 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):
- Absolute neutrophil count (ANC) >1000/μL.
- Platelets ≥50000/μL.
- Hemoglobin ≥8.0 g/dL.
- Absolute lymphocyte count ≥100/μL.
- Has adequate organ function as follows:
- Renal function: estimated glomerular filtration rate (eGFR) >29 mL/min by Schwartz formula (Schwartz et al 1976).
- Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤5 × upper limit of normal (ULN) for age.
- Bilirubin <1.5 x ULN (for Gilbert's Syndrome, subject's total bilirubin <4 mg/dL).
- Adequate pulmonary function as follows:
- Resting oxygen saturation of ≥91% on room air.
- No or mild dyspnea (Grade ≤1).
- Female subjects of childbearing potential must be willing to undergo pregnancy tests before MB-CART2019.1 infusion.
- If subjects are sexually active, they must be willing to use highly effective methods of contraception.
- Female subjects must agree to use two methods of contraception;
- one of the following methods (Pearl index <1%): Hormonal contraceptives associated with inhibition of ovulation (oral, intravaginal, injected, implanted, transdermal), intrauterine devices (IUDs) or systems (e.g., hormonal and non-hormonal IUD), or vasectomized sexual partner AND one barrier method.
- Highly effective methods of contraception must be followed from inclusion until 12 months after MB-CART2019.1 infusion.
- Male subjects must agree to use a condom during intercourse from inclusion through at least 12 months after MB-CART2019.1 infusion to prevent them from fathering a child AND to prevent delivery of MB-CART2019.1 via seminal fluid to their partner. Do not use a female condom when using a male condom, since tearing can occur. In addition, male subjects must not donate sperm for the time period specified above.
- Females must agree not to breast feed or donate eggs/ova during the study and until at least 12 months after MB-CART2019.1 infusion.
- Is willing to undergo collection of non-mobilized leukapheresis.
- In the opinion of the investigator, the subject must be able to comply with all study-related procedures, medication use, and assessments.
Who cannot join
- 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).
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- 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.
- Has history of cerebral vascular accident within 12 months prior to leukapheresis.
- Has impaired cardiac function: Fractional shortening <28% or left ventricular ejection fraction <50% by echocardiography or multigated acquisition, if allowed as per local law.
- Has concomitant genetic syndromes associated with bone marrow (BM) failure status, such as Fanconi anemia, Kostmann syndrome, Shwachman syndrome, or any other known BM failure syndrome.
- Is a pregnant or breast-feeding female.
- Is sexually active and not willing to use highly effective methods of contraception as described in the inclusion criteria.
- Has history of another malignancy within the prior 3 years that required systemic therapy.
- Has other medical, psychological, or social condition that, in the opinion of the investigator, would impact subject safety or confound the study results.
- Has received vaccination with live virus within 6 weeks prior to informed consent.
- Has been previously treated with approved anti-CD19 or anti-CD20 CART cell therapies <100 days prior to informed consent/assent.
Trial locations and contacts
5 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
5 sites in 4 countries
France2 sites, 1 recruiting
- Active, not recruiting
Hôpital Robert Debré
Paris, Boulevard Sérurier 48, 75019
- Recruiting
Institut Gustave Roussy
Villejuif, 94805
Veronique Minard-Colin, +33 (0) 1-42-11-41-70, veronique.minard@gustaveroussy.fr
Germany1 site
- Active, not recruiting
Universitaetsklinikum Muenster (UKM) - Klinik fuer Kinder- und Jugendmedizin - Paediatrische Haematologie und Onkologie
Münster, Albert-Schweitzer-Campus 1, Gebaeude A1, 48129
Italy1 site, 1 recruiting
- Recruiting
IRCCS Ospedale Pediatrico Bambino Gesù
Rome, Piazza Sant Onofrio 4, 00165
Franco Locatelli, Pediatric Oncology, +39 06-6859-1 /2545/2129, franco.locatelli@opbg.net
Netherlands1 site
- Active, not recruiting
Prinses Maxima Centrum
Utrecht, Heidelberglaan 25
About this study
Official title: A Single-arm, Multi-center, Open-label Phase II Study to Determine the Safety and Efficacy of MB-CART2019.1 in Pediatric Subjects With Relapsed/Refractory Mature B-cell Neoplasms Who Have Relapsed After One or More Prior Therapies, Including Subjects With Primary Refractory Disease
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.
Read the detailed description
This is a single-arm, multi-center, open-label Phase II study designed to evaluate the efficacy of zamtocabtagene autoleucel (MB-CART2019.1) therapy infusion in pediatric subjects with relapsed/refractory (r/r) mature B-cell neoplasms. The primary study endpoint will be best overall response (BORR). Furthermore, the safety and toxicity of the study product will be assessed based on type, frequency, and severity of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESI).
MB-CART2019.1 is designed to effectively target malignant B cells in patients suffering from haematological B-cell malignancies. Zamtocabtagene autoleucel (MB-CART2019.1) is a tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy. Pediatric patients who are suitable for this study will be treated with MB-CART2019.1.
Zamtocabtagene autoleucel (MB-CART2019.1) administration consists of a single infusion with fresh formulation of 2.5 × 10^6 CAR-transduced autologous T cells/kg bodyweight. IMP is only to be administered after a lymphodepleting chemotherapy with fludarabine and cyclophosphamide. For MB-CART2019.1 production, pediatric patients will undergo a leukapheresis. The enrollment period will last approximately 36 months and the duration of the study for each subject will be 85 weeks (including screening period and a 78 weeks safety follow-up).
- Conditions
- B-Cell Neoplasm
- Being tested
- zamtocabtagene autoleucel (MB-CART2019.1)
- Medical subject headings
- Lymphoma, B-Cell
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
zamtocabtagene autoleucel (MB-CART2019.1)Drug
Tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy.
| Study group | Role | What they receive |
|---|---|---|
| zamtocabtagene autoleucel (MB-CART2019.1) | Experimental | Not described Receives: Drug: zamtocabtagene autoleucel (MB-CART2019.1) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 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.
Measured: From infusion until week 78
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Jul 2024
- Study start4 Aug 2025
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for B-Cell NeoplasmStudies of the same condition, open and under way.
- More trials run by Miltenyi Biomedicine GmbHThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- A multi-center, international, open-label study
- Masking
- None (open label)
- Enrolment
- 31 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Miltenyi Biomedicine GmbH
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- PPD, Part of Thermo Fisher Scientific
- Responsible party
- Sponsor
- Investigators
- Birgit Burkhardt, Prof. Dr., Principal investigator, Uniklinik Münster, Klinik für Kinder- und Jugendmedizin Pädiatrische Hämatologie/Onkologie
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06508931
- Sponsor's protocol ID
- M-2021-389
- Other IDs
- 2023-506348-17-00, 2023
- Acronym
- DALY PED
- Status last verified
- Sep 2025
- First submitted
- 2 Jul 2024
- Last updated
- 5 May 2026
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Single-arm, Multi-center, Open-label Phase II Study to Determine the Safety and Efficacy of MB-CART2019.1 in Pediatric Subjects With Relapsed/Refractory Mature B-cell Neoplasms Who Have Relapsed After One or More Prior Therapies, Including Subjects With Primary Refractory Disease
- ClinicalTrials.gov ID
- NCT06508931
- Acronym / sponsor ID
- DALY PED / M-2021-389
- Conditions
- B-Cell Neoplasm
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 31 participants (estimated)
- Lead sponsor
- Miltenyi Biomedicine GmbHIndustry (pharma, biotech, devices)
- Collaborators
- PPD, Part of Thermo Fisher Scientific
- Study start
- 4 Aug 2025 (actual)
- Primary completion
- 31 Dec 2029 (estimated)
- Study completion
- 31 Dec 2029 (estimated)
- First posted
- 18 Jul 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
This is a single-arm, multi-center, open-label Phase II study designed to evaluate the efficacy of zamtocabtagene autoleucel (MB-CART2019.1) therapy infusion in pediatric subjects with relapsed/refractory (r/r) mature B-cell neoplasms. The primary study endpoint will be best overall response (BORR). Furthermore, the safety and toxicity of the study product will be assessed based on type, frequency, and severity of adverse events (AEs), serious adverse events (SAEs), and adverse events of special interest (AESI).
MB-CART2019.1 is designed to effectively target malignant B cells in patients suffering from haematological B-cell malignancies. Zamtocabtagene autoleucel (MB-CART2019.1) is a tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy. Pediatric patients who are suitable for this study will be treated with MB-CART2019.1.
Zamtocabtagene autoleucel (MB-CART2019.1) administration consists of a single infusion with fresh formulation of 2.5 × 10^6 CAR-transduced autologous T cells/kg bodyweight. IMP is only to be administered after a lymphodepleting chemotherapy with fludarabine and cyclophosphamide. For MB-CART2019.1 production, pediatric patients will undergo a leukapheresis. The enrollment period will last approximately 36 months and the duration of the study for each subject will be 85 weeks (including screening period and a 78 weeks safety follow-up).
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- A multi-center, international, open-label study
| Arm | Type | Receives |
|---|---|---|
| zamtocabtagene autoleucel (MB-CART2019.1) | Experimental | Drug: zamtocabtagene autoleucel (MB-CART2019.1) |
Who may qualify
- Ages
- 6 months to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (35 listed)
- 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:
- 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.
Key exclusion criteria (24 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| zamtocabtagene autoleucel (MB-CART2019.1)Tandem CD20-CD19-directed non-cryopreserved CAR-T cell therapy. | Drug | zamtocabtagene autoleucel (MB-CART2019.1) |
Primary outcome
BORR, Best Overall Response Rate
Time frame: From infusion until week 78
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.
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]
Time frame: From infusion until week 78
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.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 May 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Birgit Burkhardt, Prof. Dr. principal investigator
Uniklinik Münster, Klinik für Kinder- und Jugendmedizin Pädiatrische Hämatologie/Onkologie
5 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing zamtocabtagene autoleucel (MB-CART2019.1) in people with B-Cell Neoplasm, run by Miltenyi Biomedicine GmbH at 5 sites in 4 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 35 inclusion and 24 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 5 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in France, Germany, Italy, Netherlands. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Andrey Shirokov (+49 151 1179 4851, andrey.shirokov@miltenyi.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
BORR, Best Overall Response Rate (time frame: From infusion until week 78). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 May 2026.