Long-Term Follow-up of Patients Treated With Miltenyi Cell and Gene Therapies (LONGSAFE)

The goal of this Long-Term Follow-Up Trial is to assess long-term safety and efficacy of Miltenyi CAR T treatment.

Study Overview

• Status: Recruiting
• Conditions: Pediatric ALL; Melanoma Stage IV; Melanoma Stage III; B-cell Non Hodgkin Lymphoma; Childhood Non-Hodgkin Lymphoma; Chronic Lymphatic Leukemia; Acute Lymphatic Leukemia
• Intervention / Treatment: Biological: MB-CART19.1; Biological: MB-CART20.1; Biological: MB-CART2019.1

Detailed Description

The focus in this trial is on the occurrence of any late adverse reactions (AR), serious adverse events (SAE), serious adverse reactions (SAR) and adverse events of special interest (AESI), i.e. relapse or progression of the underlying disease, life-threatening infections, death due to any case, new and secondary malignancies, lymphocyte counts, detection of the transgene of the CAR T cells, detection of replication-competent lentivirus (RCL), developmental tracking in pediatric patients and furthermore, assessment of the primary status of disease progression and overall survival.

• Study Type: Interventional
• Enrollment (Estimated): 40
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Clinical Trial Manager; Phone Number: +4922048306820; Email: clinicaltrials.gov@miltenyi.com

Study Locations

• Germany
  • Berlin, Germany, 13353
    • Recruiting
    • Charité Universitätsmedizin Berlin
  • Cologne, Germany, 50937
    • Active, not recruiting
    • Universitätsklinikum Köln
  • Erlangen, Germany, 91054
    • Active, not recruiting
    • Uniklinikum Erlangen
  • Göttingen, Germany, 37075
    • Active, not recruiting
    • Universitätsmedizin Göttingen
  • Münster, Germany, 48149
    • Recruiting
    • Universitatsklinikum Munster
  • Tübingen, Germany, 72076
    • Active, not recruiting
    • Universitätsklinikum Tübingen
  • Würzburg, Germany, 97080
    • Active, not recruiting
    • Universitäts-Kinderklinik Würzburg

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

• Patient underwent treatment with a Miltenyi CAR T cell therapy in one of the parent trials at least 12 months prior to enrollment in long-term follow-up.
• Patient has provided informed consent prior to enrollment.

Exclusion Criteria:

• No exclusion criteria

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Other
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: Long-term follow-up of CAR T cell therapy; Assessment of long-term safety and efficacy of Miltenyi CAR T treatment.

Biological: MB-CART19.1; Assessment of long-term safety and efficacy of MB-CART19.1 treatment.; Other Names: CD19-targeting CAR T cells; Anti-CD19 CAR T cells; Biological: MB-CART20.1; Assessment of long-term safety and efficacy of MB-CART20.1 treatment.; Other Names: CD20-targeting CAR T cells; Anti-CD20 CAR T cells; Biological: MB-CART2019.1; Assessment of long-term safety and efficacy of MB-CART2019.1 treatment.; Other Names: CD20/CD19-targeting CAR T cells; Anti-CD20/CD19 CAR T cells

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Adverse Events	Percentage of patients with late-onset adverse reactions (AR), serious adverse events (SAE), serious adverse reactions (SAR) and adverse events of special interest (AESI) including relapse or progression of the underlying disease, life-threatening infections, death due to any cause, new and secondary malignancies	through study completion, up to 14 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Lymphocyte count	B and T lymphocyte count	through study completion, up to 14 years
Height	Height	through study completion, up to 14 years
Weight	Weight	through study completion, up to 14 years
Tanner staging for pediatric patients	Tanner staging for pediatric patients	through study completion, up to 14 years
Menstruation status for pediatric patients	Did the patient experience their first menstrual cycle (menarche)? If yes, enter date	through study completion, up to 14 years
RCL	Percentage of patients with detectable replication-competent lentivirus (RCL) (if results of 2 consecutive years are negative for an individual patient, further sampling will not be continued for this patient; if results of all samples in first year of primary trial were negative for an individual patient, sampling will not be continued for this patient in this long-term follow-up trial)	through study completion, up to 14 years
Relapse / Progression	Percentage of patients who relapse or progress since enrollment and rate of surviving patients	through study completion, up to 14 years
Detectable Transgene levels	Percentage of patients with detectable transgene levels (if results of 2 consecutive years are negative for an individual patient, further sampling will be stopped for this patient)	through study completion, up to 14 years

Collaborators and Investigators

• Sponsor: Miltenyi Biomedicine GmbH
• Investigators: Principal Investigator: Claudia Rössig, Prof. Dr., Universitatsklinikum Munster

Study record dates

Study Major Dates

• Study Start (Actual): May 5, 2024
• Primary Completion (Estimated): December 31, 2040
• Study Completion (Estimated): December 31, 2040

Study Registration Dates

• First Submitted: June 11, 2024
• First Submitted That Met QC Criteria: July 16, 2024
• First Posted (Actual): July 18, 2024

Study Record Updates

• Last Update Posted (Actual): November 26, 2025
• Last Update Submitted That Met QC Criteria: November 20, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Neoplasms by Site; Neoplasms; Chronic Disease; Disease Attributes; Immune System Diseases; Neoplasms by Histologic Type; Hematologic Diseases; Skin Diseases; Lymphatic Diseases; Lymphoproliferative Disorders; Immunoproliferative Disorders; Lymphoma, Non-Hodgkin; Leukemia, B-Cell; Neuroectodermal Tumors; Neoplasms, Germ Cell and Embryonal; Neoplasms, Nerve Tissue; Lymphoma; Neuroendocrine Tumors; Leukemia, Lymphoid; Leukemia; Nevi and Melanomas; Skin Neoplasms; Pathological Conditions, Signs and Symptoms; Skin and Connective Tissue Diseases; Hemic and Lymphatic Diseases; Lymphoma, B-Cell; Leukemia, Lymphocytic, Chronic, B-Cell; Precursor Cell Lymphoblastic Leukemia-Lymphoma; Melanoma
• Other Study ID Numbers: M-2022-393; 2022-501648-14-00 (Ctis)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No