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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

ConditionsIdiopathic Inflammatory MyopathyDermatomyositisAnti-Synthetase SyndromeImmune-Mediated Necrotizing MyopathyJuvenile DermatomyositisJuvenile PolymyositisJuvenile Idiopathic Inflammatory Myopathy (JIIM)Juvenile Myositis
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age6-75
SponsorCabaletta Bio

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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, or 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

Disqualifiers

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

Trial design

Design model

Single group

Treatments tested in this trial

  • CABA-201 following preconditioning with fludarabine and cyclophosphamide

    Biological/Vaccine

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

Treatment groups

74 Participants
are divided into 3 treatment groups
Group A: CABA-201 Phase 1/2Experimental treatment 1 intervention
Group B: CABA-201 Phase 2b - Sub-study 1Experimental treatment 1 intervention
Group C: CABA-201 Phase 2b - Sub-study 2Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Phase 1/2: Incidence and severity of adverse events (AEs)

Incidence and severity of AEs

Time frame
Up to 28 days after CABA-201 infusion
2

Phase 2b Sub-study 1: Proportion of DM & ASyS subjects achieving at least a moderate Total Improvement Score (TIS) without any immunomodulatory medications and no or low dose of steroids

≥40 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement

Time frame
Within 16 weeks
3

Phase 2b Sub-study 2: Proportion of subjects with IMNM achieving at least a minimal TIS without any immunomodulatory medications and no or low dose of steroids

≥20 on the TIS, a composite measure ranging from 0 to 100, derived from six Core Set Measures, with higher scores indicating greater clinical improvement

Time frame
Within 24 weeks

Secondary outcomes

1

Incidence of adverse events and laboratory abnormalities

Incidence and severity of AEs, including changes in laboratory values and vital signs

Time frame
Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
2

Pharmacodynamics (PD)

Levels of B cells in the blood

Time frame
Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
3

Pharmacokinetics (PK)

Levels of CABA-201-positive T cells in the blood

Time frame
Up to 156 weeks (Phase 1/2) and through 52 weeks (Sub-study 1 and 2)
4

Change in disease-related biomarkers of muscle inflammation

Levels of muscle enzymes (CK, LDH, AST, ALT, and aldolase) in serum

Time frame
Up to 156 weeks (Phase 1/2)

Other outcomes

Sponsors and contacts

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

Cabaletta Bio

Lead sponsor

Source ClinicalTrials.gov
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