Health TrialsbyHealthPanel

MB-CART19.1 in Refractory SLE

ConditionSLE - Systemic Lupus Erythematosus
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18+
SponsorMiltenyi Biomedicine GmbH

About this trial

This is a phase l/ll open-label, multicentre, interventional single-arm trial of MB-CART19.1 in patients with refractory SLE systemic lupus erythematosus. In the phase I part, a maximum of n=12 patients will be treated in a maximum of 3 dose levels.

In the phase IIa part, a maximum of n=17 will be treated (n=10 patients in a 1st stage + n=7 patients in a 2nd stage). This includes the patients from the phase I part treated on the recommended dose level.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Patients at least 18 years of age.

Signed and dated informed consent before the conduct of any trial-specific procedure.

SLE fulfilling the 2019 ACR/EULAR classification criteria (refer to Appendix 8).

One BILAG A or two BILAG B despite treatment with at least two of the following treatment options: MMF, cyclophosphamide, rituximab belimumab, anifrolumab, methotrexate, azathioprine.

Disqualifiers

Active clinically significant central nervous system (CNS) dysfunction (including but not limited to uncontrolled seizure disorders, cerebrovascular ischemia or hemorrhage, dementia, paralysis).

Uncontrolled diabetes mellitus.

Therapy induced lung disease and tuberculosis.

Forced Vital Capacity (FVC) < 60 %, FEV1 < 60 %, Total Lung Capacity (TLC) < 60 % and DLCO (diffusion capacity) < 60 %.

Trial design

Design model

Sequential

Treatments tested in this trial

  • MB-CART19.1

    Biological/Vaccine

    MB-CART19.1 consists of autologous CD19 Chimeric Antigen Receptor (CAR) transduced CD4/CD8 enriched T cells

Treatment groups

29 Participants
are divided into 4 treatment groups
Group A: Dose Level 0Experimental treatment 1 intervention
Group B: Dose Level 1Experimental treatment 1 intervention
Group C: Dose Level 2Experimental treatment 1 intervention
Group D: Phase II - Recommended dose MB-CART19.1Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Phase I: Determination of the recommended dose for phase IIa out of 3 dose levels

Based on a Bayesian Optimal Interval (BOIN) design with DLT after infusion of MB-CART19.1

Time frame
Until day 28 after infusion of MB-CART19.1
2

Phase I: Safety and toxicity of MB-CART19.1

Incidence of adverse event (AE), classified according to CTCAE version 5.0, and evaluation and classification of Cytokine Release Syndrome (CRS) and Immune Effector cell-associated Neurotoxicity Syndrome (ICANS)

Time frame
24 months
3

Phase IIa: Proportion of patients in remission after infusion of MB-CART19.1

Remission is evaluated by fulfillment of DORIS remission criteria of SLE

Time frame
Month 6

Secondary outcomes

1

Phase I and Phase IIa: Treatment response to MB-CART19.1

Measured by DORIS, LLDAS, SLEDAI 2K, BILAG

Time frame
Day 28, Week 12, Month 6, Month 12, Month 24
2

Phase I and Phase IIa: Patient reported outcomes

SF-36, FACIT-F, HAQ-DI

Time frame
Day 0, Day 28, Week 12, Month 6, Month 12, Month 24
3

Phase I and Phase IIa: Cellular and humoral immunogenicity

Incidence of cellular and humoral immunogenicity

Time frame
Day 28, Week 12, Month 6, Month 12
4

Phase I and Phase IIa: B cell aplasia and immunoglobulin deficiency

Occurrence and duration of B cell aplasia, immunoglobulin levels

Time frame
Day 0, all visits after Day 7 inclusive until Month 24

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Miltenyi Biomedicine GmbH

Lead sponsor

Source ClinicalTrials.gov
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