Phase 2b/3 double blind, randomized, placebo-controlled trial to assess safety and efficacy of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for the treatment of adults with spinocerebellar ataxia).
This is a randomized, double-blind, placebo-controlled trial to assess the safety and efficacy of SLS-005 for the treatment of adults with SCA. The study consists of a 2-week screening period, a 52-week treatment period, and a 2-week safety follow-up period. Eligible participants between the ages of 18-75 years, will be randomized to treatment with SLS-005 or equivalent placebo (sodium chloride injection, 0.9%, USP). The study plans to enroll up to 245 participants with SCA3. Biomarkers associated with neuro-axonal injury, pharmacokinetics, Modified Scale for Assessment and Rating of Ataxia (m-SARA), Clinical Global Impression of Severity (CGI-S), Patient Global Impression of Severity (PGI-S), and Friedreich's Ataxia Rating Scale - Activities of Daily Living (FARS-ADL), will be assessed at screening and/or baseline and at scheduled times throughout the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
23
UCLA
Los Angeles, California, United States
Primary Efficacy: m-SARA
Mean change from baseline in Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52
Time frame: 52 weeks
Efficacy: CGI-S
Mean change from baseline in Clinical Global Impression of Severity (CGI-S) score at week 52
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: PGI-S
Mean change from baseline in Patient Global Impression of Severity (PGI-S) score at week 52
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: FARS-ADL
Mean change from baseline in Friedreich's Ataxia Rating Scale (FARS) for the assessment of performance in basic activities that are typically required daily for independent living.
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: m-SARA
Mean change from baseline in m-SARA total score at week 26.
Time frame: 26 weeks
Efficacy: m-SARA
Mean change from baseline in m-SARA total score at weeks 4, 13, 26, 39, and 52
Time frame: 52 weeks
Safety: Adverse Events
Incidences of Treatment-Emergent Adverse Events and Serious Adverse Events (SAEs), including clinically significant laboratory and electrocardiogram (ECG) abnormalities.
Time frame: 56 weeks
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