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

Spinocerebellar Ataxia Type 3 Clinical Trial

— STRIDES  

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT05490563
• Other study ID #: SLS-005-302
• Status: Terminated
• Phase: Phase 2/Phase 3
• Start date: June 3, 2022
• Est. completion date: November 24, 2023

Study information

• Verified date: April 2024
• Source: Seelos Therapeutics, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 23
• Est. completion date: November 24, 2023
• Est. primary completion date: November 24, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Machado-Joseph Disease
• Spinocerebellar Ataxia Type 3
• Spinocerebellar Ataxias
• Spinocerebellar Degenerations

Intervention

Drug:
• SLS-005
SLS-005
• Placebo
Placebo (sodium chloride injection, 0.9%, USP)

Locations

Country	Name	City	State
Australia	The Alfred Hospital	Melbourne	Victoria
Brazil	Policlinica - Universidade Estadual de Campinas UNICAMP	Campinas	Sao Paulo
Brazil	Hospital de Clinicas de Porto Alegre UFRGs	Porto Alegre	RS
Brazil	University of Sao Paulo	Ribeirão Preto	Sao Paulo
Germany	University Hospital of Leipzig	Leipzig	Saxonia
Germany	Department of Neurology and Hertie Institute for Clinical Brain Research	Tuebingen
Korea, Republic of	Korea University Guro Hospital	Seoul
Korea, Republic of	Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine	Seoul
Korea, Republic of	Seoul National University Hospital	Seoul
Portugal	Centro Hospitalar e Universitrio de Coimbra	Coimbra
Portugal	Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia	Lisboa
Portugal	Hospital de Santo António, Centro Hospitalar Universitário do Porto	Porto
Spain	Hospital Clinic de Barcelona	Barcelona
Spain	Hospital Universitario Ramon y Cajal	Madrid
Spain	Hospital Universitario La Fe	Valencia
United Kingdom	University College London	London
United States	UCHealth Neurosciences Center - Anschutz Medical Campus	Aurora	Colorado
United States	Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)	Boston	Massachusetts
United States	UT Southwestern Medical Center	Dallas	Texas
United States	UCLA	Los Angeles	California
United States	Columbia University Medical Center	New York	New York
United States	Swedish Neuroscience Specialists - Movement Disorders	Seattle	Washington
United States	University of South Florida	Tampa	Florida

Sponsors (1)

Lead Sponsor	Collaborator
Seelos Therapeutics, Inc.

Countries where clinical trial is conducted

United States,  Australia,  Brazil,  Germany,  Korea, Republic of,  Portugal,  Spain,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Efficacy: CGI-S	Mean change from baseline in Clinical Global Impression of Severity (CGI-S) score at week 52	4, 13, 26, 39, and 52 weeks
Secondary	Efficacy: PGI-S	Mean change from baseline in Patient Global Impression of Severity (PGI-S) score at week 52	4, 13, 26, 39, and 52 weeks
Secondary	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.	4, 13, 26, 39, and 52 weeks
Secondary	Efficacy: m-SARA	Mean change from baseline in m-SARA total score at week 26.	26 weeks
Secondary	Efficacy: m-SARA	Mean change from baseline in m-SARA total score at weeks 4, 13, 26, 39, and 52	52 weeks
Secondary	Safety: Adverse Events	Incidences of Treatment-Emergent Adverse Events and Serious Adverse Events (SAEs), including clinically significant laboratory and electrocardiogram (ECG) abnormalities.	56 weeks