RESET-MS: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Multiple Sclerosis

NCT07006805 | Not Yet Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: CAB-201-005
• Study Type: interventional
• Target: 12
• Locations: 0 countries
• Sites: N/A

Brief Summary

RESET-MS: A Phase 1/2 Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Participants with Multiple Sclerosis

Conditions

Relapsing Multiple Sclerosis (RMS); Progressive Multiple Sclerosis (PMS); Multiple Sclerosis (Relapsing Remitting); Multiple Sclerosis; Multiple Sclerosis (MS) - Relapsing-remitting; Progressive Multiple Sclerosis

Keywords

CABA-201; Rese-cel; autoimmune disease; anti-CD19 CAR-T therapy; cellular therapy; Multiple Sclerosis; Relapsing MS; RRMS; Progressive MS; PPMS; SPMS; Neurology; Resecabtagene autoleucel

Outcome Measures

Primary Outcomes (2)

• Primary (Part A: Dose Escalation) incidence and severity of adverse events

  An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal result of an investigation), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The term AE is used to include both serious and non-serious AEs.

  Up to 28 days after CABA-201 infusion

• Primary (Part B: Dose Expansion) incidence of and severity of adverse events in order to confirm the dose(s) of CABA-201

  An AE is any untoward medical occurrence in a patient or clinical study participant, temporally associated with the use of study treatment, whether or not considered related to the study treatment. An AE can therefore be any unfavorable and unintended sign (including an abnormal result of an investigation), symptom, or disease (new or exacerbated) temporally associated with the use of study treatment. The term AE is used to include both serious and non-serious AEs.

  Up to 28 days after CABA-201 infusion

Secondary Outcomes (8)

• Part A and Part B: To evaluate the incidence and severity of adverse events

  Up to 156 weeks after CABA-201 infusion

• Part A and Part B: To characterize the pharmacodynamics (PD)

  Up to 156 weeks after CABA-201 infusion

• Part A and Part B: To characterize the pharmacokinetics (PK)

  Up to 156 weeks after CABA-201 infusion

• Part A and Part B: To evaluate disease related biomarkers

  Up to 156 weeks after CABA-201 infusion

• Part A and Part B: To evaluate the effects of CABA-201 on MS disease activity as measured by Magnetic Resonance Imaging (MRI)

  Up to 156 weeks after CABA-201 infusion

Study Arms (2)

Relapsing MS Cohort

EXPERIMENTAL

Biological: CABA-201

Progressive MS Cohort

EXPERIMENTAL

Biological: CABA-201

Interventions

CABA-201 BIOLOGICAL

Single intravenous infusion of CABA-201 following preconditioning with fludarabine and cyclophosphamide

Progressive MS Cohort; Relapsing MS Cohort

Eligibility Criteria

• Age: 18 Years - 60 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Able to provide informed consent.
• Age ≥18 and ≤60 years of age.
• Diagnosis of MS per the revised 2017 McDonald criteria (Thompson et al, 2018).
• For participants with relapsing forms of MS only (RMS Cohort):
• Moderate degree of previously accumulated disability as measured by the Expanded Disability Status Scale (EDSS)
• Documentation of clinical relapse or a positive historical gadolinium (Gd)-enhancing magnetic resonance imaging (MRI) scan prior to Screening
• Prior treatment with a high-efficacy therapy or prior treatment failure of oral therapies
• For participants with progressive forms of MS only (PMS cohort):
• Moderate Disability as measured by EDSS
• Presence of abnormal function on protocol specified EDSS Functional Systems Scale
• Objective worsening of disease prior to Screening while on standard of care therapy
• Clinical stability by vital signs assessment at the time of screening

You may not qualify if:

• History of fulminant MS
• Clinically significant concomitant central nervous system pathologies which, in the Investigator's judgement, may confound the ability to interpret study results or complicate identification or evaluation of neurotoxicity, including but not limited to:
• Any history of seizure disorder, even if well-controlled on antiepileptics
• History of progressive multifocal leukoencephalopathy
• Active, inflammatory autoimmune disorder other than MS requiring immunomodulatory therapies
• a. Positive human immunodeficiency virus (HIV), hepatitis C virus (HCV) antibody, or hepatitis B surface antigen test, or evidence of active or chronic tuberculosis (TB) at Screening or other chronic viral infections as described in the protocol
• Use of the following therapies:
• Any prior or concurrent exposure to mitoxantrone, alemtuzumab, total lymphoid irradiation
• Cladribine within 1 year of Screening
• Any investigational agent within 4 weeks or 5 half-lives of Screening, whichever is longer
• Other pre-specified Disease-Modifying Therapies be discontinued by the time of pre-conditioning or earlier as described in the protocol
• Known malignancy or a history of malignancy
• Current symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, pulmonary, psychiatric, cardiac, or concomitant neurological disease, including severe (requiring medical intervention) and uncontrolled infections
• Chronic pulmonary disease
• Impaired cardiac function or clinically significant cardiac disease

Sponsors & Collaborators

• Cabaletta Bio: lead

MeSH Terms

Conditions

Multiple Sclerosis, Chronic Progressive; Multiple Sclerosis, Relapsing-Remitting; Multiple Sclerosis; Autoimmune Diseases

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Study Officials

• Medical Monitor

  Cabaletta Bio

  STUDY CHAIR

Central Study Contacts

Cabaletta Bio

CONTACT

267-759-3100; clinicaltrials@cabalettabio.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 22, 2025
• First Posted: June 5, 2025
• Study Start: June 1, 2026
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): October 1, 2029
• Last Updated: October 31, 2025

Record last verified: 2025-10

Data Sharing

• IPD Sharing: Will not share