Spinocerebellar Ataxia Type 3Phase 2/3STRIDESNCT05490563
STRIDES - a Clinical Research Study of an Investigational New Drug to Treat Spinocerebellar Ataxia
A Phase 2/3 study testing SLS-005 in people with Spinocerebellar Ataxia Type 3, run by Seelos Therapeutics, Inc. at 23 sites in 8 countries.
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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Spinocerebellar Ataxia Type 3
- What is being tested?
- SLS-005
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 23 participants
- Where
- 23 sitesin 8 countries
- Study dates
- 3 Jun 2022to 24 Nov 2023
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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).
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- 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.
Who cannot join
- 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.
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- 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
Trial locations and contacts
23 sites in 8 countries.
23 sites in 8 countries
United States7 sites
UCHealth Neurosciences Center - Anschutz Medical Campus
Aurora, Colorado, 80045
Harvard Medical School - Beth Israel Deaconess Medical Center (BIDMC)
Boston, Massachusetts, 02215-5395
UT Southwestern Medical Center
Dallas, Texas, 75390
UCLA
Los Angeles, California, 90095
Columbia University Medical Center
New York, New York, 10032
Swedish Neuroscience Specialists - Movement Disorders
Seattle, Washington, 98122
University of South Florida
Tampa, Florida, 33612
Brazil3 sites
Policlinica - Universidade Estadual de Campinas UNICAMP
Campinas, São Paulo, 13083
Hospital de Clinicas de Porto Alegre UFRGs
Porto Alegre, Rio Grande do Sul, 90035
University of Sao Paulo
Ribeirão Preto, São Paulo, 14049
Portugal3 sites
Centro Hospitalar e Universitrio de Coimbra
Coimbra, 3004
Hospital de Santa Maria, Centro Hospitalar Lisboa Norte, Neurologia
Lisbon, 1649-028
Hospital de Santo António, Centro Hospitalar Universitário do Porto
Porto, 4099-001
South Korea3 sites
Seoul National University Hospital
Seoul, 03080
Samsung Medical Center/Sungkyunwhan Universtiy School of Medicine
Seoul, 06351
Korea University Guro Hospital
Seoul, 08308
Spain3 sites
Hospital Clinic de Barcelona
Barcelona, 08036
Hospital Universitario Ramon y Cajal
Madrid, 28034
Hospital Universitario La Fe
Valencia, 46009
Germany2 sites
University Hospital of Leipzig
Leipzig, Saxony, 04103
Department of Neurology and Hertie Institute for Clinical Brain Research
Tübingen, 72076
About this study
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
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).
Read the 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
- Being tested
- SLS-005, Placebo
- Medical subject headings
- Machado-Joseph Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
SLS-005Drug
SLS-005
PlaceboDrug
Placebo (sodium chloride injection, 0.9%, USP)
| Study group | Role | What they receive |
|---|---|---|
| SLS-005 0.75 g/kg Dose | Experimental | 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 Receives: Drug: SLS-005 |
| SLS-005 0.50 g/kg Dose | Experimental | 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 Receives: Drug: SLS-005 |
| Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation | Placebo comparator | 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 Receives: Drug: Placebo |
| Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation | Placebo comparator | 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 Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Primary Efficacy: m-SARA
Mean change from baseline in Modified Scale for Assessment and Rating of Ataxia (m-SARA) total score at week 52
Measured: 52 weeks
Secondary outcomes (6)
Efficacy: CGI-S
Mean change from baseline in Clinical Global Impression of Severity (CGI-S) score at week 52
Measured: 4, 13, 26, 39, and 52 weeks
Efficacy: PGI-S
Mean change from baseline in Patient Global Impression of Severity (PGI-S) score at week 52
Measured: 4, 13, 26, 39, and 52 weeks
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.
Measured: 4, 13, 26, 39, and 52 weeks
Efficacy: m-SARA
Mean change from baseline in m-SARA total score at week 26.
Measured: 26 weeks
Efficacy: m-SARA
Mean change from baseline in m-SARA total score at weeks 4, 13, 26, 39, and 52
Measured: 52 weeks
Safety: Adverse Events
Incidences of Treatment-Emergent Adverse Events and Serious Adverse Events (SAEs), including clinically significant laboratory and electrocardiogram (ECG) abnormalities.
Measured: 56 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Aug 2022
- Study start3 Jun 2022
- Primary completion24 Nov 2023
- Study completion24 Nov 2023
Why it stopped: This was a business decision due to financial considerations, not based on data related to safety or therapeutic effects.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinocerebellar Ataxia Type 3Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 23 participants (actual)
Sponsor and investigators
- Lead sponsor
- Seelos Therapeutics, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05490563
- Sponsor's protocol ID
- SLS-005-302
- Acronym
- STRIDES
- Status last verified
- Apr 2024
- First submitted
- 3 Aug 2022
- Last updated
- 24 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05490563
- Acronym / sponsor ID
- STRIDES / SLS-005-302
- Conditions
- Spinocerebellar Ataxia Type 3
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This was a business decision due to financial considerations, not based on data related to safety or therapeutic effects.
- Enrolment
- 23 participants (actual)
- Lead sponsor
- Seelos Therapeutics, Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Jun 2022 (actual)
- Primary completion
- 24 Nov 2023 (actual)
- Study completion
- 24 Nov 2023 (actual)
- First posted
- 5 Aug 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| SLS-005 0.75 g/kg Dose | Experimental | Drug: SLS-005SLS-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 0.50 g/kg Dose | Experimental | Drug: SLS-005SLS-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 volume equivalent to a SLS-005 0.75 g/kg dose calculation | Placebo comparator | Drug: PlaceboPlacebo (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 volume equivalent to a SLS-005 0.50 g/kg dose calculation | Placebo comparator | Drug: PlaceboPlacebo (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 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- 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.
Key exclusion criteria (15 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| SLS-005SLS-005 | Drug | SLS-005 0.50 g/kg Dose, SLS-005 0.75 g/kg Dose |
| PlaceboPlacebo (sodium chloride injection, 0.9%, USP) | Drug | Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculation, Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculation |
Primary outcome
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
6 secondary outcomes
Efficacy: CGI-S
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: PGI-S
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: FARS-ADL
Time frame: 4, 13, 26, 39, and 52 weeks
Efficacy: m-SARA
Time frame: 26 weeks
Efficacy: m-SARA
Time frame: 52 weeks
Safety: Adverse Events
Time frame: 56 weeks
Results and safety
The study was stopped early (reason given: This was a business decision due to financial considerations, not based on data related to safety or therapeutic effects.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2024.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
23 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing SLS-005 in people with Spinocerebellar Ataxia Type 3, run by Seelos Therapeutics, Inc. at 23 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 24 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, Brazil, Portugal, South Korea, Spain and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was stopped early (reason given: This was a business decision due to financial considerations, not based on data related to safety or therapeutic effects.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Primary Efficacy: m-SARA (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Apr 2024.