A 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
Status: Terminated
Location: See all (23) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2/Phase 3
SUMMARY
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).
Eligibility
Participation Requirements
Sex: All
Minimum Age: 18
Maximum Age: 75
Healthy Volunteers: f
View:
• Signed informed consent.
• Men and women, 18 to 75 years (inclusive) of age.
• Clinical diagnosis of SCA3 with documented genetic confirmation.
• m-SARA total score ≥ 4 at the screening visit.
• m-SARA gait component score ≥ 1 at the screening visit.
• Body Mass Index (BMI) between 18 kg/m2 and 35 kg/m2 (inclusive).
• Stable doses of all concomitant medications for at least 30 days prior to the screening visit.
• Negative serum beta-human chorionic gonadotropin (ß-hCG) pregnancy result at the screening visit for female participants of childbearing potential.
• Willingness to comply with sexual abstinence or contraception guidelines of this study.
Locations
United States
California
UCLA
Los Angeles
Colorado
UCHealth Neurosciences Center - Anschutz Medical Campus
Aurora
Florida
University of South Florida
Tampa
Massachusetts
Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)
Boston
New York
Columbia University Medical Center
New York
Texas
UT Southwestern Medical Center
Dallas
Washington
Swedish Neuroscience Specialists - Movement Disorders
Seattle
Other Locations
Australia
The Alfred Hospital
Melbourne
Brazil
Policlinica - Universidade Estadual de Campinas UNICAMP
Campinas
Hospital de Clinicas de Porto Alegre UFRGs
Porto Alegre
University of Sao Paulo
Ribeirão Preto
Germany
University Hospital of Leipzig
Leipzig
Department of Neurology and Hertie Institute for Clinical Brain Research
Tübingen
Portugal
Centro Hospitalar e Universitrio de Coimbra
Coimbra
Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia
Lisbon
Hospital de Santo António, Centro Hospitalar Universitário do Porto
Porto
Republic of Korea
Korea University Guro Hospital
Seoul
Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine
Seoul
Seoul National University Hospital
Seoul
Spain
Hospital Clinic de Barcelona
Barcelona
Hospital Universitario Ramon y Cajal
Madrid
Hospital Universitario La Fe
Valencia
United Kingdom
University College London
London
Time Frame
Start Date:2022-06-03
Completion Date:2023-11-24
Participants
Target number of participants:23
Treatments
Experimental: SLS-005 0.75 g/kg Dose
SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion). SLS-005 will be administered as a weight-based dose of 0.75 g/kg by IV infusion once a week.~For 52 weeks
Experimental: SLS-005 0.50 g/kg Dose
SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion). SLS-005 will be administered as a weight-based dose of 0.50 g/kg by IV infusion once a week.~For 52 weeks
Placebo_comparator: Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation
Placebo (sodium chloride injection, 0.9, USP) will be administered by IV infusion once a week as a weight-based volume equivalent to a SLS-005 0.75 g/kg dose.~For 52 weeks
Placebo_comparator: Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation
Placebo (sodium chloride injection, 0.9, USP) will be administered by IV infusion once a week as a weight-based volume equivalent to a SLS-005 0.50 g/kg dose.~For 52 weeks