MB-CART2019.1 in Refractory Multiple Sclerosis
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-55
SponsorMiltenyi Biomedicine GmbH
About this trial
The goal of this trial is to assess the feasibility, safety and preliminary efficacy of MB-CART2019.1 in patients with active refractory primary and secondary progressive MS.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Have read, understood and signed/dated the informed consent form.
Age ≥18 years at the time of screening.
Diagnosis of multiple sclerosis fulfilling the 2017 McDonald criteria.
Progressive or worsening MS according to 2014 Lublin MS phenotypic criteria
Disqualifiers
For relapsing and progressive MS forms: the disability status according to the EDSS scale is larger than 7.0 or the age is larger than 55 years.
History of a malignancy unless disease free for ≥5 years with the exception of basal or squamous cell skin cancer
Known history of and/or active infection with hepatitis B (hepatitis B surface antigen positive)
Known history of infection with hepatitis C virus unless treated and confirmed to be polymerase chain reaction (PCR) negative
Trial design
Design model
Single group
Treatments tested in this trial
MB-CART2019.1
Biological/VaccineCAR T cell therapy
Treatment groups
26 Participants
are divided into 1 treatment groupGroup A: MB-CART2019.1 in MSExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Primary endpoints Phase I
* Recommended dose for phase IIa out of 2 dose levels, based on a Bayesian Optimal Interval (BOIN) design, using a target dose limited toxicity (DLT) rate of 30%, with DLT until day 28 after infusion of MB-CART2019.1. * Safety and tolerability of MB-CART2019.1 per incidence of adverse event (AE), classified according to CTCAE version 5.0. * Evaluation and classification of Cytokine Release Syndrome (CRS) until day 28 after infusion of MB-CART2019.1. * Evaluation and classification of Immune Effector cell-associated Neurotoxicity Syndrome (ICANS) until day 28 after infusion of MB-CART2019.1.
Time frame
day 282
Primary endpoints Phase IIa
* NEDA-3 ("no evident disease activity") at 48 weeks (fulfilling all of the following): * no relapse activity: relapse activity is defined as any new neurologic symptom consistent with MS/not explained otherwise, accompanied by new neurologic signs present for at least 24 hrs. * no disease progression in the absence of relapses using a composite endpoint of no 24-week confirmed progression: * of ≥1 point/ ≥0.5 point on the EDSS if baseline EDSS ≤5.5/ \>5.5 points ; * of ≥20% on the timed 25-foot walk test * of ≥20%on the 9-hole peg test (NHPT). * no MRI activity: MRI activity is defined as: * new and/or enlarging T2-hyperintense lesions as detected by MRI (defined as the sum of the individual number of new and/or enlarging T2 lesions at all scheduled at 48 weeks compared to baseline) or * new T1-contrast enhancing (CE) lesions as detected by MRI at 12, 24 or 48 weeks compared to baseline.
Time frame
48 weeksSecondary outcomes
1
Secondary endpoints Phase I only (primary in phase IIa)
NEDA-3 ("no evident disease activity") at 48 weeks (fulfilling all of the following): 1. no relapse activity: relapse activity is defined as any new neurologic symptom consistent with MS/not explained otherwise, accompanied by new neurologic signs present for at least 24 hrs 2. no disease progression in the absence of relapses using a composite endpoint of * no 24-week confirmed progression of ≥1 point/ ≥0.5 point on the EDSS if baseline EDSS ≤5.5/ \>5.5 points ; * no 24-week confirmed progression of ≥20% on the timed 25-foot walk test and * no 24-week confirmed progression of ≥20%on the 9-hole peg test (NHPT) 3. no MRI activity: MRI activity is defined as * new and/or enlarging T2-hyperintense lesions as detected by MRI (defined as the sum of the individual number of new and/or enlarging T2 lesions at all scheduled at 48 weeks compared to baseline) or * new T1-contrast enhancing (CE) lesions as detected by MRI at 12, 24 or 48 weeks compared to baseline.
Time frame
48 weeksSponsors and contacts
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Source ClinicalTrials.gov