RESET-Myositis: An Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects With Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

NCT ID: NCT06154252

Last Updated: 2025-10-22

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

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

24 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-20

Study Completion Date

2028-07-31

Brief Summary

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RESET-Myositis: Open-Label Study to Evaluate the Safety and Efficacy of CABA-201 in Subjects with Active Idiopathic Inflammatory Myopathy or Juvenile Idiopathic Inflammatory Myopathy

Detailed Description

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Idiopathic inflammatory myopathies (IIMs, or myositis) are a group of rare autoimmune diseases characterized by inflammation and muscle weakness. Though the cause of IIM is not well understood, some subtypes of IIM, including dermatomyositis (DM), anti-synthetase syndrome (ASyS), immune-mediated necrotizing myopathy (IMNM), and juvenile idiopathic inflammatory myopathy (JIIM), are thought to involve B cells that cause the body to attack different tissues in the body. This study is being conducted to evaluate the safety and efficacy of an investigational cell therapy, CABA-201, that can be given to patients with DM, ASyS, IMNM, or JIIM who have active disease. A single dose of CABA-201 in combination with cyclophosphamide (CY) and fludarabine (FLU) will be evaluated.

Conditions

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Idiopathic Inflammatory Myopathy Dermatomyositis Anti-Synthetase Syndrome Immune-Mediated Necrotizing Myopathy Juvenile Dermatomyositis Juvenile Polymyositis Juvenile Idiopathic Inflammatory Myopathy (JIIM) Juvenile Myositis

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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CABA-201

DM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with DM.

ASyS Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with ASyS.

IMNM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with IMNM.

JIIM Cohort: Infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide preconditioning in subjects with JIIM.

Group Type EXPERIMENTAL

CABA-201 following preconditioning with fludarabine and cyclophosphamide

Intervention Type BIOLOGICAL

Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide

Interventions

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CABA-201 following preconditioning with fludarabine and cyclophosphamide

Single intravenous infusion of CABA-201 at a single dose level following preconditioning with fludarabine and cyclophosphamide

Intervention Type BIOLOGICAL

Other Intervention Names

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Rese-cel Resecabtagene autoleucel

Eligibility Criteria

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Inclusion Criteria

* Age ≥18 and ≤75
* A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism/American College of Rheumatology classification criteria
* Diagnosis of DM, ASyS, IMNM
* Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated creatine kinase (CK), DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography
* Presence of muscle weakness

Other protocol-defined criteria apply.


* Age ≥6 and ≤17 years at enrollment
* A clinical diagnosis of IIM, based on the 2017 The European League Against Rheumatism/American College of Rheumatology classification criteria
* Evidence of active disease, despite prior or current treatment with standard of care treatments, as defined by the presence of elevated muscle enzymes, DM rash, or active disease on muscle biopsy, magnetic resonance imaging (MRI), or electromyography

Other protocol-defined criteria apply.

Exclusion Criteria

* Contraindication to leukapheresis
* History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites
* Active infection requiring medical intervention at screening
* Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections
* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
* Significant lung or cardiac impairment
* Previous CAR T cell therapy
* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant

Other protocol-defined criteria apply.

Juvenile Cohort


* Contraindication to leukapheresis
* History of anaphylactic or severe systemic reaction to fludarabine, cyclophosphamide or any of their metabolites
* Active infection requiring medical intervention at screening
* Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, neurological, or cerebral disease, including severe and uncontrolled infections, such as sepsis and opportunistic infections.
* Concomitant medical conditions that, in the opinion of the investigator, might place the subject at unacceptable risk for participation in this study, interfere with the assessment of the effects or safety of the investigational product or with the study procedures
* Significant lung or cardiac impairment
* Previous CAR T cell therapy
* Prior solid organ (heart, liver, kidney, lung) transplant or hematopoietic cell transplant

Other protocol-defined criteria apply.
Minimum Eligible Age

6 Years

Maximum Eligible Age

75 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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University of California Irvine - Accepting Adult Patients

Orange, California, United States

Site Status RECRUITING

University of California, San Francisco Benioff Children's Hospital - Accepting Young Adult and Juvenile Patients

San Francisco, California, United States

Site Status RECRUITING

Children's Hospital Colorado - Accepting Juvenile Patients

Aurora, Colorado, United States

Site Status NOT_YET_RECRUITING

Mayo Clinic Florida - Accepting Adult Patients

Jacksonville, Florida, United States

Site Status RECRUITING

Emory University - Accepting Adult Patients

Atlanta, Georgia, United States

Site Status RECRUITING

Ann & Robert H. Lurie Children's Hospital of Chicago - Accepting Young Adult and Juvenile Patients

Chicago, Illinois, United States

Site Status NOT_YET_RECRUITING

Northwestern Memorial Hospital - Accepting Adult Patients

Chicago, Illinois, United States

Site Status RECRUITING

The University of Chicago Medical Center - Accepting Adult and Juvenile Patients

Chicago, Illinois, United States

Site Status RECRUITING

University of Kansas Medical Center - Accepting Adult Patients

Kansas City, Kansas, United States

Site Status RECRUITING

National Institutes of Health - Accepting Adult and Juvenile Patients

Bethesda, Maryland, United States

Site Status NOT_YET_RECRUITING

University of Michigan - Accepting Young Adult and Juvenile Patients

Ann Arbor, Michigan, United States

Site Status RECRUITING

Mayo Clinic - Accepting Adult Patients

Rochester, Minnesota, United States

Site Status RECRUITING

Hospital for Special Surgery - Accepting Adult and Juvenile Patients

New York, New York, United States

Site Status RECRUITING

Memorial Sloan Kettering Cancer Center - Accepting Adult and Juvenile Patients

New York, New York, United States

Site Status RECRUITING

Children's Hospital at Montefiore - Accepting Juvenile Patients

The Bronx, New York, United States

Site Status NOT_YET_RECRUITING

Oregon Health & Science University - Accepting Adult Patients

Portland, Oregon, United States

Site Status RECRUITING

Children's Hospital of Philadelphia - Accepting Young Adult and Juvenile Patients

Philadelphia, Pennsylvania, United States

Site Status RECRUITING

Vanderbilt University Medical Center - Accepting Adult Patients

Nashville, Tennessee, United States

Site Status RECRUITING

Houston Methodist Hospital - Accepting Adult Patients

Houston, Texas, United States

Site Status RECRUITING

University of Texas MD Anderson Cancer Center - Accepting Adult Patients

Houston, Texas, United States

Site Status RECRUITING

Seattle Children's Research Institute - Accepting Juvenile Patients

Seattle, Washington, United States

Site Status NOT_YET_RECRUITING

Kings College Hospital NHS Foundation Trust - Accepting Adult Patients

London, , United Kingdom

Site Status RECRUITING

University College London Hospitals NHS Foundation Trust - Accepting Adult Patients

London, , United Kingdom

Site Status RECRUITING

Manchester Royal Infirmary - Accepting Adult Patients

Manchester, , United Kingdom

Site Status RECRUITING

Salford Royal Hospital - Accepting Adult Patients

Salford, , United Kingdom

Site Status RECRUITING

Countries

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United States United Kingdom

Central Contacts

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Cabaletta Bio

Role: CONTACT

267-759-3100 ext. 4444

Facility Contacts

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Alpha Clinic

Role: primary

949-824-3990

Zilan Zheng

Role: primary

415-353-1301

Dr. Katharine Moore, MD

Role: primary

720-777-7371

Wesley Dillinger

Role: primary

904-953-4511

Trinh Van

Role: primary

404-712-3940

John Varghese

Role: backup

Tyler Sorensen

Role: primary

312-277-6828

Matthew Selle

Role: primary

312-695-0990

Dr. Iazsmin Ventura, MD - Adult patient contact

Role: primary

Dr. Cuoghi Edens, MD, FAAP - Juvenile patient contact

Role: backup

Ali Russo, B.S., CCRP

Role: primary

913-945-9942

Julie Thompson

Role: primary

301-480-3191

Anela Mukherjee

Role: primary

734-232-9896

Abigail Whalen

Role: backup

734-615-4466

Bridget Neja

Role: primary

507-266-9150

Janvi Bhatia

Role: primary

212-774-2123

Atifah Osrat

Role: primary

646-608-2091

Emily Gillies

Role: primary

718-696-2402

Katie Lewis

Role: primary

503-908-9724

Nur Ali

Role: primary

615-875-5106

Amelia A. Nounes, MSN, RN, CCRP

Role: primary

346-356-3639

Christina Amos Briggs, BSN, RN

Role: primary

713-563-5076

Dr. Susan Shenoi, MD, MS

Role: primary

206-987-2193

Rheumatology Research Team

Role: primary

0207 848 0214 ext. 0628

Dr. Pedro Machado, FRCP, PhD

Role: primary

020 3456 7890

Eleni Tholouli, MD, PhD, MRCP(UK), FRCPath

Role: primary

01612765052

Michelle Beswick

Role: primary

07759 136787

Other Identifiers

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CAB-201-002

Identifier Type: -

Identifier Source: org_study_id

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