RESET-MS: A Study to Evaluate the Safety and Efficacy of CABA-201 in Participants With Multiple Sclerosis
- Conditions
- Relapsing Multiple Sclerosis (RMS)Progressive Multiple Sclerosis (PMS)Multiple Sclerosis - Relapsing RemittingMultiple SclerosisMultiple Sclerosis (Relapsing Remitting)Multiple Sclerosis (MS) - Relapsing-remittingProgressive Multiple Sclerosis
- Interventions
- Registration Number
- NCT07006805
- Lead Sponsor
- Cabaletta Bio
- 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
- Detailed Description
This is a Phase 1/2, open-label study designed to evaluate the safety, tolerability, and efficacy of different doses of CABA-201 in adult participants with MS to determine an appropriate dose for future studies. Any participant who receives CABA-201 will be followed after infusion for 156 weeks. Two cohorts of participants will be studied based upon their MS diagnosis.
* Relapsing MS Cohort (RMS Cohort): Participants with active relapsing MS, including relapsing remitting MS (RRMS) and relapsing secondary progressive MS (SPMS) that is treatment-resistant
* Progressive MS Cohort (PMS Cohort): Participants with worsening progressive MS, including primary progressive MS (PPMS) or non-relapsing SPMS that is treatment-resistant
The study will consist of 2 parts: Part A (dose escalation) and Part B (dose expansion).
Recruitment & Eligibility
- Status
- NOT_YET_RECRUITING
- Sex
- All
- Target Recruitment
- 12
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Relapsing MS Cohort CABA-201 - Progressive MS Cohort CABA-201 -
- Primary Outcome Measures
Name Time Method Primary (Part A: Dose Escalation) incidence and severity of adverse events Up to 28 days after CABA-201 infusion 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.
Primary (Part B: Dose Expansion) incidence of and severity of adverse events in order to confirm the dose(s) of CABA-201 Up to 28 days after CABA-201 infusion 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.
- Secondary Outcome Measures
Name Time Method Part A and Part B: To evaluate the incidence and severity of adverse events Up to 156 weeks after CABA-201 infusion 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.
Part A and Part B: To characterize the pharmacodynamics (PD) Up to 156 weeks after CABA-201 infusion Levels of B cells in the blood
Part A and Part B: To characterize the pharmacokinetics (PK) Up to 156 weeks after CABA-201 infusion Levels of CABA-201-positive T cells in the blood
Part A and Part B: To evaluate disease related biomarkers Up to 156 weeks after CABA-201 infusion Levels of MS biomarkers in the blood and CSF
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 Incidence of accumulated MS-related lesions
Part A and Part B: The effects of CABA-201 on MS disease activity as measured by EDSS Up to 156 weeks after CABA-201 infusion The EDSS is a scale for assessing neurologic impairment in MS. Values are from 0 points (normal neurological examination) up to 10 points (death). Higher scores represent increased disability.
Part A and Part B: To evaluate the effect of CABA-201 on use of subsequent MS-related therapy Up to 156 weeks after CABA-201 infusion Proportion of participants who require no subsequent MS-related immunomodulatory therapy
Part A and Part B: To evaluate the effect of CABA-201 on patient reported and health outcomes as measured by SF-36 v2 Up to 156 weeks after CABA-201 infusion Change in SF-36 v2