What side effects are associated with this type of intervention?

Trehalose, used as a treatment for Spinocerebellar Ataxias, has shown potential benefits such as improved gait behavior and motor coordination, as well as reduced astrocyte gliosis in animal models. The context does not provide specific side effects for Trehalose or similar treatments. Generally, protein stabilizers and anti-aggregation agents can have side effects, but further studies are needed to confirm the safety profile of Trehalose in humans.

What prior FDA approvals exist?

There is no specific mention of prior FDA approvals for the treatment of Spinocerebellar Ataxias (SCAs) in the provided context. However, SLS-005 (Trehalose Injection) is being studied as a potential treatment due to its properties as a protein stabilizer and anti-aggregation agent. Trehalose has shown promise in preclinical studies for improving motor coordination and reducing gliosis in SCA17 mice, suggesting its potential as a non-toxic treatment option for SCAs.

What other types of research exist?

One promising alternative to SLS-005 for Spinocerebellar Ataxias patients is taliglucerase alfa, a plant cell-based recombinant enzyme therapy. Although primarily developed for Gaucher disease, its unique mechanism and advanced clinical development stage suggest potential applicability in other neurodegenerative conditions. Additionally, therapies involving adeno-associated viral vectors, which have been evaluated in large animal models for various lysosomal storage diseases, may offer novel treatment avenues for SCA patients.

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Investigational Drug for Spinocerebellar Ataxia (STRIDES Trial)

Phase 2 & 3

Waitlist Available

Research Sponsored by Seelos Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Clinical diagnosis of SCA3 with documented genetic confirmation.

Men and women, 18 to 75 years (inclusive) of age.

Must not have

Current diagnosis and/or healthcare professional-recommended treatment (medication and/or diet) of diabetes mellitus type 1 or type 2.

Prior treatment with SLS-005, any other IV trehalose formulation, or known hypersensitivity to trehalose.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 4, 13, 26, 39, and 52 weeks

Summary

This trial tests a sugar solution injection called trehalose to help adults with a condition affecting coordination and balance. The treatment aims to protect nerve cells and improve symptoms. Trehalose, also known as SLS-005, is being investigated in various regimens for its potential neuroprotective effects.

Who is the study for?

Adults aged 18-75 with a clinical diagnosis of Spinocerebellar Ataxia type 3 (SCA3), confirmed genetically, and an m-SARA score ≥4 can join. Participants must have stable medication use for the past month, a BMI between 18-35 kg/m2, and agree to contraception guidelines. Exclusions include diabetes treatment, chronic liver disease or infections like HIV/Hepatitis B/C, recent drug/alcohol abuse, other medical conditions that could affect results or pose risks.

What is being tested?

The STRIDES study is testing SLS-005 (trehalose injection) against a placebo in adults with spinocerebellar ataxia. This Phase 2b/3 trial randomly assigns participants to either the investigational drug or placebo group in equal numbers without them knowing which one they're getting to measure its safety and effectiveness.

What are the potential side effects?

While specific side effects are not listed here, potential side effects may include reactions related to intravenous infusions such as discomfort at the injection site, allergic reactions, and systemic responses depending on how the body tolerates trehalose.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been genetically confirmed to have SCA3.

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I am between 18 and 75 years old.

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I have been genetically confirmed to have SCA3.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have been diagnosed with or am being treated for type 1 or type 2 diabetes.

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I have never been treated with SLS-005 or similar drugs, nor am I allergic to trehalose.

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I have chronic liver disease or HIV, or I had Hepatitis B or C but it was successfully treated.

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I have had liver injury from medication or my liver tests are not normal.

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My kidney function is not adequate, with a creatinine clearance of less than 60 mL/min.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 4, 13, 26, 39, and 52 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~4, 13, 26, 39, and 52 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 4, 13, 26, 39, and 52 weeks for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Primary Efficacy: m-SARA

Secondary study objectives

Efficacy: CGI-S

Efficacy: FARS-ADL

Efficacy: PGI-S

+2 more

Side effects data

From 2023 Phase 2 & 3 trial • 161 Patients • NCT05136885

27%

Fall

18%

Muscular weakness

16%

Fatigue

15%

Neuromyopathy

13%

Constipation

11%

Diarrhoea

10%

COVID-19

Headache

Dysphagia

Cough

Post lumbar puncture syndrome

Musculoskeletal pain

Urinary tract infection

Infusion site bruising

Abdominal discomfort

Dyspnoea

Oedema peripheral

Infusion related reaction

Dysarthria

Salivary hypersecretion

Amyotrophic lateral sclerosis

Acute respiratory failure

Pneumonia

Respiratory failure

Pneumonia aspiration

Urticaria

Abdominal pain upper

Complication associated with device

Hypertension

Syncope

Pulmonary embolism

Cerebrovascular accident

Hip fracture

Breast cancer

100%

80%

60%

40%

20%

Study treatment Arm

Matching Placebo

SLS-005

Trial Design

4Treatment groups

Experimental Treatment

Placebo Group

Group I: SLS-005 0.75 g/kg DoseExperimental Treatment1 Intervention

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

Group II: SLS-005 0.50 g/kg DoseExperimental Treatment1 Intervention

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

Group III: Placebo volume equivalent to a SLS-005 0.75 g/kg dose calculationPlacebo Group1 Intervention

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

Group IV: Placebo volume equivalent to a SLS-005 0.50 g/kg dose calculationPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

SLS-005

2022

Completed Phase 3

~170

0 / 8Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

SLS-005 (Trehalose Injection) functions as a protein stabilizer and anti-aggregation agent. Trehalose helps to prevent the misfolding and aggregation of proteins, which are common pathological features in Spinocerebellar Ataxias (SCAs). By stabilizing proteins and reducing their aggregation, trehalose can potentially mitigate the neurodegenerative processes that lead to the symptoms of SCAs, such as gait ataxia and motor coordination issues. This mechanism is crucial for SCA patients as it targets the underlying cause of neuronal damage, offering a potential therapeutic approach to slow disease progression and improve quality of life.

Chaperone therapy for Krabbe disease: potential for late-onset GALC mutations.Trehalose attenuates the gait ataxia and gliosis of spinocerebellar ataxia type 17 mice.Chemical chaperone therapy: clinical effect in murine G(M1)-gangliosidosis.