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

Primary Purpose

Spinocerebellar Ataxia Type 3

Status
Active
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
SLS-005
Placebo
Sponsored by
Seelos Therapeutics, Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Spinocerebellar Ataxia Type 3

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • UCLA
  • UCHealth Neurosciences Center - Anschutz Medical Campus
  • University of South Florida
  • Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)
  • Columbia University Medical Center
  • UT Southwestern Medical Center
  • Swedish Neuroscience Specialists - Movement Disorders
  • The Alfred Hospital
  • Hospital de Clinicas de Porto Alegre UFRGs
  • Policlinica - Universidade Estadual de Campinas UNICAMP
  • University of Sao Paulo
  • University Hospital of Leipzig
  • Department of Neurology and Hertie Institute for Clinical Brain Research
  • Seoul National University Hospital
  • Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine
  • Korea University Guro Hospital
  • Centro Hospitalar e Universitrio de Coimbra
  • Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia
  • Hospital de Santo António, Centro Hospitalar Universitário do Porto
  • Hospital Clinic de Barcelona
  • Hospital Universitario Ramon y Cajal
  • Hospital Universitario La Fe
  • University College London

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Placebo Comparator

Placebo Comparator

Arm Label

SLS-005 0.75 g/kg Dose

SLS-005 0.50 g/kg Dose

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

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

Arm Description

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

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 (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 (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

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

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

Full Information

First Posted
August 3, 2022
Last Updated
April 4, 2023
Sponsor
Seelos Therapeutics, Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT05490563
Brief Title
STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
Acronym
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
Study Type
Interventional

2. Study Status

Record Verification Date
April 2023
Overall Recruitment Status
Active, not recruiting
Study Start Date
June 3, 2022 (Actual)
Primary Completion Date
June 3, 2024 (Anticipated)
Study Completion Date
June 3, 2024 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Seelos Therapeutics, Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Spinocerebellar Ataxia Type 3

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
245 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
SLS-005 0.75 g/kg Dose
Arm Type
Experimental
Arm Description
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
Arm Title
SLS-005 0.50 g/kg Dose
Arm Type
Experimental
Arm Description
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
Arm Title
Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation
Arm Type
Placebo Comparator
Arm Description
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
Arm Title
Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation
Arm Type
Placebo Comparator
Arm Description
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
Intervention Type
Drug
Intervention Name(s)
SLS-005
Intervention Description
SLS-005
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo (sodium chloride injection, 0.9%, USP)
Primary Outcome Measure Information:
Title
Primary Efficacy: m-SARA
Description
Mean change from baseline in Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
Efficacy: CGI-S
Description
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
Title
Efficacy: PGI-S
Description
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
Title
Efficacy: FARS-ADL
Description
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
Title
Efficacy: m-SARA
Description
Mean change from baseline in m-SARA total score at week 26.
Time Frame
26 weeks
Title
Efficacy: m-SARA
Description
Mean change from baseline in m-SARA total score at weeks 4, 13, 26, 39, and 52
Time Frame
52 weeks
Title
Safety: Adverse Events
Description
Incidences of Treatment-Emergent Adverse Events and Serious Adverse Events (SAEs), including clinically significant laboratory and electrocardiogram (ECG) abnormalities.
Time Frame
56 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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
Facility Information:
Facility Name
UCLA
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
UCHealth Neurosciences Center - Anschutz Medical Campus
City
Aurora
State/Province
Colorado
ZIP/Postal Code
80045
Country
United States
Facility Name
University of South Florida
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02215-5395
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
UT Southwestern Medical Center
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Swedish Neuroscience Specialists - Movement Disorders
City
Seattle
State/Province
Washington
ZIP/Postal Code
98122
Country
United States
Facility Name
The Alfred Hospital
City
Melbourne
State/Province
Victoria
ZIP/Postal Code
3004
Country
Australia
Facility Name
Hospital de Clinicas de Porto Alegre UFRGs
City
Porto Alegre
State/Province
RS
ZIP/Postal Code
90035
Country
Brazil
Facility Name
Policlinica - Universidade Estadual de Campinas UNICAMP
City
Campinas
State/Province
Sao Paulo
ZIP/Postal Code
13083
Country
Brazil
Facility Name
University of Sao Paulo
City
Ribeirão Preto
State/Province
Sao Paulo
ZIP/Postal Code
14049
Country
Brazil
Facility Name
University Hospital of Leipzig
City
Leipzig
State/Province
Saxonia
ZIP/Postal Code
04103
Country
Germany
Facility Name
Department of Neurology and Hertie Institute for Clinical Brain Research
City
Tuebingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Seoul National University Hospital
City
Seoul
ZIP/Postal Code
03080
Country
Korea, Republic of
Facility Name
Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine
City
Seoul
ZIP/Postal Code
06351
Country
Korea, Republic of
Facility Name
Korea University Guro Hospital
City
Seoul
ZIP/Postal Code
08308
Country
Korea, Republic of
Facility Name
Centro Hospitalar e Universitrio de Coimbra
City
Coimbra
ZIP/Postal Code
3004
Country
Portugal
Facility Name
Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia
City
Lisboa
ZIP/Postal Code
1649-028
Country
Portugal
Facility Name
Hospital de Santo António, Centro Hospitalar Universitário do Porto
City
Porto
ZIP/Postal Code
4099-001
Country
Portugal
Facility Name
Hospital Clinic de Barcelona
City
Barcelona
ZIP/Postal Code
08036
Country
Spain
Facility Name
Hospital Universitario Ramon y Cajal
City
Madrid
ZIP/Postal Code
28034
Country
Spain
Facility Name
Hospital Universitario La Fe
City
Valencia
ZIP/Postal Code
46009
Country
Spain
Facility Name
University College London
City
London
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

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

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