STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia

NCT05490563 | Terminated Phase 2 FDA Drug

• 1 other identifier: SLS-005-302
• Study Type: interventional
• Target: 23
• Locations: 8 countries
• Sites: 23

Brief 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).

Conditions

Spinocerebellar Ataxia Type 3

Outcome Measures

Primary Outcomes (1)

• Primary Efficacy: m-SARA

  Mean change from baseline in Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52

  52 weeks

Secondary Outcomes (6)

• Efficacy: CGI-S

  4, 13, 26, 39, and 52 weeks

• Efficacy: PGI-S

  4, 13, 26, 39, and 52 weeks

• Efficacy: FARS-ADL

  4, 13, 26, 39, and 52 weeks

• Efficacy: m-SARA

  26 weeks

• Efficacy: m-SARA

  52 weeks

Study Arms (4)

SLS-005 0.75 g/kg Dose

EXPERIMENTAL

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

Drug: SLS-005

SLS-005 0.50 g/kg Dose

EXPERIMENTAL

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

Drug: SLS-005

Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation

PLACEBO COMPARATOR

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

Drug: Placebo

Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

SLS-005 DRUG

SLS-005

SLS-005 0.50 g/kg Dose; SLS-005 0.75 g/kg Dose

Placebo DRUG

Placebo (sodium chloride injection, 0.9%, USP)

Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation; Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• Any hereditary ataxia that is not genetically confirmed to be SCA type 3, or any type of ataxia that is acquired or secondary to another medical condition including but not limited to, alcoholism, head injury, multiple sclerosis, olivopontocerebellar atrophy, multiple system atrophy, or stroke.
• A score of 4 on any 1 of the 4 items that comprise the m-SARA.
• Current participation in another clinical trial or completed participation in an interventional trial less than 30 days prior to the screening visit (90 days for a biological treatment).
• Current diagnosis and/or healthcare professional-recommended treatment (medication and/or diet) of diabetes mellitus type 1 or type 2.
• Hemoglobin A1c (HbA1c) ≥ 6.5% at the screening visit
• Prior treatment with SLS-005, any other IV trehalose formulation, or known hypersensitivity to trehalose.
• Pregnant or breastfeeding.
• History of alcohol or drug abuse within the last 2 years.
• Chronic liver disease including Hepatitis B; Hepatitis C unless successful curative treatment is documented; human immunodeficiency virus (HIV) infection.
• Prior history of drug-induced liver injury (DILI) and/or laboratory results at screening that indicate inadequate liver function (e.g., alanine aminotransferase \[ALT\], aspartate aminotransferase \[AST\], gamma-glutamyl transferase \[GGT\] \> 2 times the upper limit of normal \[x ULN\] and/or total bilirubin level \> 2 x ULN).
• Laboratory results at screening that indicate inadequate renal function (e.g., estimated creatinine clearance of \< 60 mL/min calculated by the Cockcroft and Gault formula).
• Any current cardiovascular disease or abnormality on 12-lead ECG at screening that, in the investigator's opinion, is clinically significant and could be a potential safety risk to the participant.
• Any current psychiatric, neurological, or cognitive disorder that, in the investigator's opinion, may interfere with the participant's ability to provide informed consent or appropriately complete the study's safety or efficacy assessments.
• Significant suicide risk as indicated by a "yes" response to question #4 or #5 under Suicidal Ideation in the past 6 months or any "yes" response under Suicidal Behavior in the past 3 years on the Columbia Suicide Severity Rating Scale (C-SSRS) during the screening visit.
• Any other medical condition or abnormal finding during screening that, in the investigator's opinion, could confound collection or interpretation of safety or efficacy data or be a potential safety risk to the participant

Sponsors & Collaborators

• Seelos Therapeutics, Inc.: lead

Study Sites (23)

UCLA

Los Angeles, California, 90095, United States

Location

UCHealth Neurosciences Center - Anschutz Medical Campus

Aurora, Colorado, 80045, United States

Location

University of South Florida

Tampa, Florida, 33612, United States

Location

Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)

Boston, Massachusetts, 02215-5395, United States

Location

Columbia University Medical Center

New York, New York, 10032, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Swedish Neuroscience Specialists - Movement Disorders

Seattle, Washington, 98122, United States

Location

The Alfred Hospital

Melbourne, Victoria, 3004, Australia

Location

Hospital de Clinicas de Porto Alegre UFRGs

Porto Alegre, Rio Grande do Sul, 90035, Brazil

Location

Policlinica - Universidade Estadual de Campinas UNICAMP

Campinas, São Paulo, 13083, Brazil

Location

University of Sao Paulo

Ribeirão Preto, São Paulo, 14049, Brazil

Location

University Hospital of Leipzig

Leipzig, Saxony, 04103, Germany

Location

Department of Neurology and Hertie Institute for Clinical Brain Research

Tübingen, 72076, Germany

Location

Centro Hospitalar e Universitrio de Coimbra

Coimbra, 3004, Portugal

Location

Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia

Lisbon, 1649-028, Portugal

Location

Hospital de Santo António, Centro Hospitalar Universitário do Porto

Porto, 4099-001, Portugal

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine

Seoul, 06351, South Korea

Location

Korea University Guro Hospital

Seoul, 08308, South Korea

Location

Hospital Clinic de Barcelona

Barcelona, 08036, Spain

Location

Hospital Universitario Ramon y Cajal

Madrid, 28034, Spain

Location

Hospital Universitario La Fe

Valencia, 46009, Spain

Location

University College London

London, United Kingdom

Location

MeSH Terms

Conditions

Machado-Joseph Disease

Condition Hierarchy (Ancestors)

Spinocerebellar Ataxias; Cerebellar Ataxia; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinocerebellar Degenerations; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Ataxia; Dyskinesias; Neurologic Manifestations; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 3, 2022
• First Posted: August 5, 2022
• Study Start: June 3, 2022
• Primary Completion: November 24, 2023
• Study Completion: November 24, 2023
• Last Updated: April 24, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

GB (1); DE (2); AU (1); PT (3); KR (3); US (7); BR (3); ES (3)