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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05490563
Recruitment Status : Recruiting
First Posted : August 5, 2022
Last Update Posted : August 5, 2022
Sponsor:
Information provided by (Responsible Party):
Seelos Therapeutics, Inc.

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).

Condition or disease Intervention/treatment Phase
Spinocerebellar Ataxia Type 3 Drug: SLS-005 Drug: Placebo Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 245 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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
Actual Study Start Date : June 3, 2022
Estimated Primary Completion Date : June 3, 2024
Estimated Study Completion Date : June 3, 2024


Arm Intervention/treatment
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

Drug: SLS-005
SLS-005

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

Drug: SLS-005
SLS-005

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

Drug: Placebo
Placebo (sodium chloride injection, 0.9%, USP)

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

Drug: Placebo
Placebo (sodium chloride injection, 0.9%, USP)




Primary Outcome Measures :
  1. Primary Efficacy: m-SARA [ Time Frame: 52 weeks ]
    Mean change from baseline in Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52


Secondary Outcome Measures :
  1. Efficacy: CGI-S [ Time Frame: 4, 13, 26, 39, and 52 weeks ]
    Mean change from baseline in Clinical Global Impression of Severity (CGI-S) score at week 52

  2. Efficacy: PGI-S [ Time Frame: 4, 13, 26, 39, and 52 weeks ]
    Mean change from baseline in Patient Global Impression of Severity (PGI-S) score at week 52

  3. Efficacy: FARS-ADL [ Time Frame: 4, 13, 26, 39, and 52 weeks ]
    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. Efficacy: m-SARA [ Time Frame: 26 weeks ]
    Mean change from baseline in m-SARA total score at week 26.

  5. Efficacy: m-SARA [ Time Frame: 52 weeks ]
    Mean change from baseline in m-SARA total score at weeks 4, 13, 26, 39, and 52

  6. Safety: Adverse Events [ Time Frame: 56 weeks ]
    Incidences of Treatment-Emergent Adverse Events and Serious Adverse Events (SAEs), including clinically significant laboratory and electrocardiogram (ECG) abnormalities.



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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05490563


Contacts
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Contact: David Biondi, DO 646-2932100 SLS005Team@seelostx.com
Contact: Claudia N Moore, MS 206-945-2784 SLS005Team@seelostx.com

Locations
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United States, California
UCLA Recruiting
Los Angeles, California, United States, 90095
Contact: Aaron Fisher    310-206-8153    Adfisher@mednet.ucla.edu   
Principal Investigator: Susan Perlman, MD         
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33612
Contact: Lucretia Campbell    813-974-5633    lcampbel@usf.edu   
Contact: Paige Graham    813-974-4453    paigegraham@usf.edu   
Principal Investigator: Theresa Zesiewicz, MD         
Sponsors and Collaborators
Seelos Therapeutics, Inc.
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Responsible Party: Seelos Therapeutics, Inc.
ClinicalTrials.gov Identifier: NCT05490563    
Other Study ID Numbers: SLS-005-302
First Posted: August 5, 2022    Key Record Dates
Last Update Posted: August 5, 2022
Last Verified: August 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Ataxia
Cerebellar Ataxia
Spinocerebellar Ataxias
Spinocerebellar Degenerations
Machado-Joseph Disease
Dyskinesias
Neurologic Manifestations
Nervous System Diseases
Cerebellar Diseases
Brain Diseases
Central Nervous System Diseases
Spinal Cord Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Genetic Diseases, Inborn