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

NCT ID: NCT05451212

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

7 participants

Study Classification

INTERVENTIONAL

Study Start Date

2022-11-23

Study Completion Date

2025-07-24

Brief Summary

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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.

Detailed Description

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Conditions

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MuSK Myasthenia Gravis

Keywords

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CAAR-T (Chimeric Autoantibody Receptor T Cells) Therapy CAR-T (Chimeric Antigen Receptor T Cells) Therapy Cell Therapy Autoimmune Disease Autoimmunity Immunotherapy, Adoptive Immune System Diseases Myasthenia Gravis (MG) Muscle-specific tyrosine kinase (MuSK) Muscle Weakness Neuromuscular Diseases Musculoskeletal Diseases

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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MuSK-CAART

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.

Group Type EXPERIMENTAL

MuSK-CAART

Intervention Type BIOLOGICAL

Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU.

Interventions

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MuSK-CAART

Intravenous infusion of MuSK-CAART at different doses. Subjects may also receive MuSK-CAART following pre-treatment with CY, or CY plus FLU.

Intervention Type BIOLOGICAL

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Cabaletta Bio

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Medical Director

Role: STUDY_CHAIR

Cabaletta Bio

Locations

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UC Irvine, Department of Neurology

Orange, California, United States

Site Status

UC Davis, Department of Neurology

Sacramento, California, United States

Site Status

University of Kansas Medical Center

Kansas City, Kansas, United States

Site Status

Oregon Health & Science University (OHSU)

Portland, Oregon, United States

Site Status

Houston Methodist Hospital

Houston, Texas, United States

Site Status

Countries

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United States

Other Identifiers

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CAB-MuSK-101

Identifier Type: -

Identifier Source: org_study_id