Open-label Study to Evaluate the Safety of Various Dosing Regimens of MuSK-CAART for MuSK Myasthenia Gravis

Cabaletta Bio

Status and phase: Completed; Phase 1

Conditions: MuSK Myasthenia Gravis

Treatments: Biological: MuSK-CAART

Study type: Interventional

Funder types: Industry

Identifiers

NCT05451212

CAB-MuSK-101

Details and patient eligibility

About

Muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG) is a rare but potentially severe disease, in which patients develop pathogenic autoantibodies that specifically target the MuSK protein in the neuromuscular junction. This phase 1 study is being conducted to evaluate the safety of various dosing regimens of an investigational cell therapy, MuSK-CAART, that can be given to patients with anti-MuSK antibody positive Myasthenia Gravis (MuSK MG), who have active disease. Various dosing regimens of MuSK-CAART alone, in combination with cyclophosphamide (CY), and in combination with CY and fludarabine (FLU) will be evaluated. Treatment with MuSK-CAART may potentially lead to complete and durable remission of disease.

Enrollment: 7 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Confirmed diagnosis of MuSK-type MG with at least 1 prior positive anti-MuSK antibody test.
• History of a negative anti-AChR (acetylcholine receptor) antibody test.
• Positive anti-MuSK antibody test at screening
• MG severity Class I to IVa on the MGFA (Myasthenia Gravis Foundation of America) Clinical Classification

Exclusion criteria

• Rituximab in the last 12 months.
• Prednisone > 0.25mg/kg/day [in Part A]
• Other autoimmune disorder requiring immunosuppressive therapies.
• Investigational treatment for MG in the past 12 weeks.
• Absolute lymphocyte count < 500/µL at screening.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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7 participants in 1 patient group

MuSK-CAART

Experimental group

Description:

Cohort A: Infusion of MuSK-CAART at various dose levels with or without pre-treatment (6 groups planned). Cohort B: Infusion of MuSK-CAART at the dose regimen selected from Part A.

Treatment:

Biological: MuSK-CAART