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

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ClinicalTrials.gov Identifier: NCT05451212
Recruitment Status : Recruiting
First Posted : July 11, 2022
Last Update Posted : February 6, 2023
Sponsor:
Information provided by (Responsible Party):
Cabaletta Bio

Study Description
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Brief Summary:
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.

Condition or disease Intervention/treatment Phase
MuSK Myasthenia Gravis Biological: MuSK-CAART Phase 1

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 24 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 1, Open-label, Safety and Dose-finding Study of Autologous Muscle-specific Tyrosine Kinase Chimeric Autoantibody Receptor T Cells (MuSK-CAART) in Subjects With Anti-MuSK-antibody-positive Myasthenia Gravis
Actual Study Start Date : November 23, 2022
Estimated Primary Completion Date : October 2028
Estimated Study Completion Date : October 2028

Resource links provided by the National Library of Medicine

Drug Information available for: Musks

Arms and Interventions
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Arm Intervention/treatment
Experimental: 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.

 Biological: 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.


Outcome Measures
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Primary Outcome Measures :
  1. Adverse events [ Time Frame: 3 months ]
    Incidence of adverse events (AEs), including dose-limiting toxicities (DLTs) and AEs that are related to MuSK-CAART.


Secondary Outcome Measures :
  1. Total MuSK-CAART positive cells [ Time Frame: Baseline ]
    Total MuSK-CAART positive cells for each manufacturing run.

  2. Percent of CAAR-transduced cells [ Time Frame: Baseline ]
    Percent of total cells for infusion that are CAAR (Chimeric Autoantibody Receptor)-transduced cells.

  3. Cellular kinetics profile of MuSK-CAART [ Time Frame: Up to 36 months ]
    Cellular kinetics profile of MuSK-CAART after infusion.

  4. Change in MuSK autoantibody titer [ Time Frame: Up to 36 months ]
    Change in MuSK autoantibody titer compared to pre-infusion visit by clinically validated assay.


Other Outcome Measures:
  1. Use of Concomitant Therapies [ Time Frame: Up to 36 months ]
    Frequency and dose of concomitant therapies.

  2. Measurement of Clinical Symptoms using MG-ADL [ Time Frame: Up to 36 months ]
    Measurement of clinical symptoms using the Myasthenia Gravis Activities of Daily Living (MG-ADL) assessment.

  3. Measurement of Clinical Symptoms using QMG [ Time Frame: Up to 36 months ]
    Measurement of clinical symptoms using the Quantitative Myasthenia Gravis (QMG) assessment.

  4. Measurement of Clinical Symptoms using MGC [ Time Frame: Up to 36 months ]
    Measurement of clinical symptoms using the Myasthenia Gravis Composite (MGC) assessment.

  5. Measurement of Quality of Life (QoL) using MG-QOL-15r [ Time Frame: Up to 36 months ]
    Measurement of Quality of Life using the MG-QOL-15r (Myasthenia Gravis Qualify of Life 15-item scale, revised) questionnaire.


Eligibility Criteria
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Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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 < 1,000/µL at screening.
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05451212


Contacts
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Contact: Cabaletta Bio +1 267 759 3100 clinicaltrials@cabalettabio.com

Locations
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United States, California
UC Irvine, Department of Neurology Recruiting
Orange, California, United States, 92868
Contact: Luis De La Cruz    714-509-2117    ledelacr@uci.edu   
Principal Investigator: Ali Habib, MD         
UC Davis, Department of Neurology Recruiting
Sacramento, California, United States, 95817
Contact: Joshua Valdez    916-734-7707    joavaldez@ucdavis.edu   
Principal Investigator: David Richman, MD         
United States, Oregon
Oregon Health & Science University (OHSU) Recruiting
Portland, Oregon, United States, 97239
Contact: Katie Lewis    503-494-7394    lewiskat@ohsu.edu   
Principal Investigator: Nizar Chahin, MD         
Sponsors and Collaborators
Cabaletta Bio
Investigators
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Study Chair: Medical Director Cabaletta Bio
More Information
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Responsible Party: Cabaletta Bio
ClinicalTrials.gov Identifier: NCT05451212    
Other Study ID Numbers: CAB-MuSK-101
First Posted: July 11, 2022    Key Record Dates
Last Update Posted: February 6, 2023
Last Verified: February 2023

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Cabaletta Bio:
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
Additional relevant MeSH terms:
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Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
 Neoplasms by Site
Neoplasms
Paraneoplastic Syndromes
Autoimmune Diseases of the Nervous System
Neurodegenerative Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases