The study is an investigator-run, study following participants for 2 years with twice-daily remibrutinib. MRI is the main endpoint. Safety, tolerability, and efficacy are secondary endpoints. Approximately 20 participants with relapsing or progressive forms of MS will be recruited.
The study is an investigator-run, open-label Phase 2 study with approximately 24 total months of observation, involving approximately 20 participants with relapsing or progressive forms of MS. Participants will be recruited from the patient populations followed at CCF. All participants will take 100 mg remibrutinib twice daily. They will receive 4 MRIs, blood tests, EKGs, physical exams, and clinical functioning exams periodically to assess safety, tolerability, and efficacy of the study drug. All study activities will be performed at the Cleveland Clinic Mellen Center.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
100 mg remibrutinib, twice daily
Cleveland Clinic
Cleveland, Ohio, United States
Quantitative MRI volumetric measurements
Rate of change in regional brain volumes using 7T MRI over 24 months
Time frame: Baseline to Month 24
Slowly expanding lesions (SELs)
Prevalence of SELs identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Paramagnetic rim lesions (PRLs) -- count
Change in count of PRLs identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Paramagnetic rim lesions (PRLs) -- size
Change in size of PRLs identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Paramagnetic rim lesions (PRLs) -- quantitative susceptibility measures
Change in quantitative susceptibility measures of PRLs identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Myelin Water Fraction
Rate of change in myelin water fraction assessed using 7T MRI over 24 months
Time frame: Baseline to Month 24
Functional MRI (Default mode network)
Functional MRI changes (default mode network) assessed using 7T MRI over 24 months
Time frame: Baseline to Month 24
Microstructural tissue integrity
Rate of change in microstructural tissue integrity assessed using 7T MRI over 24 months
Time frame: Baseline to Month 24
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Leptomeningeal enhancement
Prevalence of leptomeningeal enhancement identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Neuromelanin
Prevalence of regional neuromelanin changes identified using 7T MRI over 24 months
Time frame: Baseline to Month 24
Frequency of Treatment Emergent Adverse Events (TEAEs)
Time frame: Baseline to Month 24
Frequency of Serious Adverse Events (SAEs)
Time frame: Baseline to Month 24
Frequency of study medication discontinuation
Time frame: Baseline to Month 24
Gadolinium Enhancing Lesions
Change in number of Gadolinium enhancing lesions over 24 months
Time frame: Baseline to Month 24
Quantitative Lesion Burden
Change in the volume of brain lesions identified over 24 months
Time frame: Baseline to Month 24
Annualized Relapse Rate (ARR)
Time frame: Baseline to Month 24
Expanded Disability Status Scale (EDSS)
A scale from 0 to 10. An increase in EDSS score is indicative of an increase in disability
Time frame: Baseline to Month 24
Serum Neurofilament Light Chain (NfL)
Time frame: Baseline to Month 24