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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Seelos Therapeutics Inc.

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Nervous System Diseases [C10]

Trial duration

30 Jan 2023 → 12 Feb 2024

Decision date (initial)

2022-12-19

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Seelos Therapeutics, Inc.

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Pharmacodynamic, Others, Dose response, Efficacy

Determine the efficacy of SLS-005 0.75 g/kg for the treatment of spinocerebellar ataxia type-3 (SCA3) in adults

Secondary objectives 1

1. Determine the safety and tolerability of SLS-005 for the treatment of spinocerebellar ataxia type-3 (SCA3) in adults

Conditions and MedDRA coding

Spinocerebellar Ataxia type-3 (SCA3)

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 9

1. 1. Signed informed consent.
2. 2. Men and women, 18 to 75 years (inclusive) of age.
3. 3. Clinical diagnosis of SCA3 with documented genetic confirmation.
4. 4. m-SARA total score ≥ 4 at the screening visit.
5. 5. m-SARA gait component score ≥ 1 at the screening visit.
6. 6. Body Mass Index (BMI) between 18 kg/m2 and 35 kg/m2 (inclusive).
7. 7. Stable doses of all concomitant medications for at least 30 days prior to the screening visit.
8. 8. Negative serum beta-human chorionic gonadotropin (ß-hCG) pregnancy result at the screening visit for female participants of childbearing potential.
9. 9. Willingness to comply with sexual abstinence or contraception guidelines of this study.

Exclusion criteria 15

1. 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. 2. A score of 4 on any 1 of the 4 items that comprise the m-SARA.
3. 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. 4. Current diagnosis and/or healthcare professional-recommended treatment (medication and/or diet) of diabetes mellitus type 1 or type 2.
5. 5. Hemoglobin A1c (HbA1c) ≥ 6.5% at the screening visit.
6. 6. Prior treatment with SLS-005, any other IV trehalose formulation, or known hypersensitivity to trehalose.
7. 7. Pregnant or breastfeeding.
8. 8. History of alcohol or drug abuse within the last 2 years.
9. 9. Chronic liver disease including Hepatitis B; Hepatitis C unless successful curative treatment is documented; HIV infection.
10. 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. 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. 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. 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. 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. 15. Any other medical condition or abnormal finding at 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.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Change from baseline in m-SARA total score at Week 52 in participants with SCA3 treated with SLS-005 0.75 g/kg compared to placebo

Secondary endpoints 10

1. Key Secondary Endpoints In participants treated with SLS-005 0.75 g/kg: • Change from baseline in Clinical Global Impression of Severity (CGI-S) at Week 52
2. • Change from baseline in Patient Global Impression of Severity (PGI-S) at Week 52
3. • Change from baseline in Friedreich’s Ataxia Rating Scale – Activities of Daily Living (FARS-ADL) score at Week 52
4. • Change from baseline in m-SARA total score at Week 26
5. Secondary Endpoints In participants with treated with SLS-005 0.75 g/kg: • Changes from baseline in m-SARA total score at Weeks 4, 13, and 39
6. • Changes from baseline in CGI-S at Weeks 4, 13, 26, and 39
7. • Changes from baseline in PGI-S at Weeks 4, 13, 26, and 39
8. • Changes from baseline in FARS-ADL score at Weeks 4, 13, 26, and 39
9. • Changes from baseline in scores for each item of the m-SARA at Weeks 4, 13, 26, 39, and 52
10. In all participants: • Incidences of treatment-emergent adverse events (TEAEs) and SAEs, including clinically significant laboratory and ECG abnormalities

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Seelos Therapeutics Inc.

Sponsor organisation

Seelos Therapeutics Inc.

Address

300 Park Avenue

City

New York

Postcode

10022-7402

Country

United States

Scientific contact point

Organisation

Seelos Therapeutics Inc.

Contact name

Tim Whitaker

Public contact point

Organisation

Seelos Therapeutics Inc.

Contact name

Tim Whitaker

Third parties 6

Locations

3 EU/EEA countries · 8 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Oversight and notifications

Regulatory notifications under CTR Articles 38, 52, 53, 54 and 77

Temporary halts 1 · Art. 38 CTR

Application history

3 submissions — initial application plus substantial / non-substantial modifications