Clinical trial NCT05490563: STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
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
Overall status: TERMINATED
Phase: PHASE2
PHASE3
Conditions: Spinocerebellar Ataxia Type 3
Lead sponsor: Seelos Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2022-08-05
Enrollment: 23
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Primary Efficacy: m-SARA
timeFrame: 52 weeks
Secondary outcomes:
measure: Efficacy: CGI-S
timeFrame: 4, 13, 26, 39, and 52 weeks
measure: Efficacy: PGI-S
timeFrame: 4, 13, 26, 39, and 52 weeks
measure: Efficacy: FARS-ADL
timeFrame: 4, 13, 26, 39, and 52 weeks
measure: Efficacy: m-SARA
timeFrame: 26 weeks
measure: Efficacy: m-SARA
timeFrame: 52 weeks
measure: Safety: Adverse Events
timeFrame: 56 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: UCLA
state: California
city: Aurora
country: United States
facility: UCHealth Neurosciences Center - Anschutz Medical Campus
state: Colorado
city: Tampa
country: United States
facility: University of South Florida
state: Florida
city: Boston
country: United States
facility: Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)
state: Massachusetts
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Seattle
country: United States
facility: Swedish Neuroscience Specialists - Movement Disorders
state: Washington
city: Melbourne
country: Australia
facility: The Alfred Hospital
state: Victoria
city: Porto Alegre
country: Brazil
facility: Hospital de Clinicas de Porto Alegre UFRGs
state: RS
city: Campinas
country: Brazil
facility: Policlinica - Universidade Estadual de Campinas UNICAMP
state: Sao Paulo
city: Ribeirão Preto
country: Brazil
facility: University of Sao Paulo
state: Sao Paulo
city: Leipzig
country: Germany
facility: University Hospital of Leipzig
state: Saxonia
city: Tuebingen
country: Germany
facility: Department of Neurology and Hertie Institute for Clinical Brain Research
city: Seoul
country: Korea, Republic of
facility: Seoul National University Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine
city: Seoul
country: Korea, Republic of
facility: Korea University Guro Hospital
city: Coimbra
country: Portugal
facility: Centro Hospitalar e Universitrio de Coimbra
city: Lisboa
country: Portugal
facility: Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia
city: Porto
country: Portugal
facility: Hospital de Santo António, Centro Hospitalar Universitário do Porto
city: Barcelona
country: Spain
facility: Hospital Clinic de Barcelona
city: Madrid
country: Spain
facility: Hospital Universitario Ramon y Cajal
city: Valencia
country: Spain
facility: Hospital Universitario La Fe
city: London
country: United Kingdom
facility: University College London
Study officials:
References: