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

NCT ID: NCT05490563

Last Updated: 2024-04-24

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-06-03
• Study Completion Date: 2023-11-24

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).

Detailed Description

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.

Conditions

Spinocerebellar Ataxia Type 3

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

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

Group Type: EXPERIMENTAL

SLS-005

Intervention Type: DRUG

SLS-005

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

Group Type: EXPERIMENTAL

SLS-005

Intervention Type: DRUG

SLS-005

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo (sodium chloride injection, 0.9%, USP)

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

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Placebo (sodium chloride injection, 0.9%, USP)

Interventions

SLS-005

SLS-005

Intervention Type: DRUG

Placebo

Placebo (sodium chloride injection, 0.9%, USP)

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Signed informed consent.

2. Men and women, 18 to 75 years (inclusive) of age.

3. Clinical diagnosis of SCA3 with documented genetic confirmation.

4. m-SARA total score ≥ 4 at the screening visit.

5. m-SARA gait component score ≥ 1 at the screening visit.

6. Body Mass Index (BMI) between 18 kg/m2 and 35 kg/m2 (inclusive).

7. Stable doses of all concomitant medications for at least 30 days prior to the screening visit.

8. Negative serum beta-human chorionic gonadotropin (ß-hCG) pregnancy result at the screening visit for female participants of childbearing potential.

9. Willingness to comply with sexual abstinence or contraception guidelines of this study.

Exclusion Criteria

1. 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.

2. A score of 4 on any 1 of the 4 items that comprise the m-SARA.

3. 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).

4. Current diagnosis and/or healthcare professional-recommended treatment (medication and/or diet) of diabetes mellitus type 1 or type 2.

5. Hemoglobin A1c (HbA1c) ≥ 6.5% at the screening visit

6. Prior treatment with SLS-005, any other IV trehalose formulation, or known hypersensitivity to trehalose.

7. Pregnant or breastfeeding.

8. History of alcohol or drug abuse within the last 2 years.

9. Chronic liver disease including Hepatitis B; Hepatitis C unless successful curative treatment is documented; human immunodeficiency virus (HIV) infection.

10. 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).

11. 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).

12. 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.

13. 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.

14. 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.

15. 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Seelos Therapeutics, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

UCLA

Los Angeles, California, United States

UCHealth Neurosciences Center - Anschutz Medical Campus

Aurora, Colorado, United States

University of South Florida

Tampa, Florida, United States

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

Boston, Massachusetts, United States

Columbia University Medical Center

New York, New York, United States

UT Southwestern Medical Center

Dallas, Texas, United States

Swedish Neuroscience Specialists - Movement Disorders

Seattle, Washington, United States

The Alfred Hospital

Melbourne, Victoria, Australia

Hospital de Clinicas de Porto Alegre UFRGs

Porto Alegre, Rio Grande do Sul, Brazil

Policlinica - Universidade Estadual de Campinas UNICAMP

Campinas, São Paulo, Brazil

University of Sao Paulo

Ribeirão Preto, São Paulo, Brazil

University Hospital of Leipzig

Leipzig, Saxony, Germany

Department of Neurology and Hertie Institute for Clinical Brain Research

Tübingen, , Germany

Centro Hospitalar e Universitrio de Coimbra

Coimbra, , Portugal

Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia

Lisbon, , Portugal

Hospital de Santo António, Centro Hospitalar Universitário do Porto

Porto, , Portugal

Seoul National University Hospital

Seoul, , South Korea

Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine

Seoul, , South Korea

Korea University Guro Hospital

Seoul, , South Korea

Hospital Clinic de Barcelona

Barcelona, , Spain

Hospital Universitario Ramon y Cajal

Madrid, , Spain

Hospital Universitario La Fe

Valencia, , Spain

University College London

London, , United Kingdom

Countries

United States; Australia; Brazil; Germany; Portugal; South Korea; Spain; United Kingdom

Other Identifiers

SLS-005-302

Identifier Type: -

Identifier Source: org_study_id