A 24-week Open Label, Basket Study of SLS-005 (Trehalose Injection 90.5 mg/mL Intravenous Infusion) in the Treatment of Adults with Neurodegenerative Diseases.
A 24-week Open Label, Basket Study of the safety, tolerability and treatment responses of SLS-005 (Trehalose Injection 90.5 mg/mL Intravenous Infusion) in Adults with Neurodegenerative Diseases
Avance Clinical
18 participants
Dec 30, 2021
Interventional
Conditions
Summary
This is a study of safety and tolerability of a drug called SLS-005 in adults, aged 18 to 75 years with selected neurodegenerative diseases [up to 4 patients with amyotrophic lateral sclerosis (ALS), up to 10 participants with Huntington’s disease (HD), and up to 4 participants with spinocerebellar ataxia type-3 (SCA3)]. This study will also help to determine the treatment response of SLS-005 using a patient-reported outcome measure. The study comprises of a 2-week screening period, 24-week open-label treatment period of SLS-005 to be administered as a once weekly intravenous infusion, and a 2-week safety follow-up period. On the day of the infusion, the study participant is expected to remain at the study centre for up to 6-8 hours. All participants will be asked to undergo blood tests and answer questions regarding their health. It is hoped that information from this study informs researchers of how the body metabolises SLS-005 and thus how it may be used to treat neurogenerative diseases, such as ALS, HD and SCA3.
Eligibility
Inclusion Criteria20
- Men and women, 18 - 75 years (inclusive) of age at screening
- Signed informed consent
- Must meet the disease specific criteria for ALS, SCA or HD listed below:
- a) ALS:
- Diagnosis of probable, lab-supported probable, or definitive ALS
- Onset of signs and symptoms of disease within the past 2 years
- Amyotrophic Lateral Sclerosis Functional Rating Scale (ALSFRS-R) score greater than 30 at the screening visit
- Slow Vital Capacity (SVC) greater than 50% of predicted for gender, height, and age at the screening visit
- b)HD:
- Clinical diagnosis of HD with documented genetic confirmation
- Onset of signs and symptoms of disease within the past 2 years and onset between the ages of 18 and 50 years (inclusive)
- Unified Huntington's Disease Rating Scale (UHDRS)-Total Functional Capacity (TFC) score of 7 to 13 (inclusive) at the screening visit
- Body Mass Index (BMI) between 20 kg/m2 and 32 kg/m2 (inclusive)
- c)SCA3:
- Clinical diagnosis of SCA3 with documented genetic confirmation
- Modified scale for assessment and rating of ataxia (m-SARA) total score greater than 4 at the screening visit.
- m-SARA gait component score greater than 1 at the screening visit.
- Stable doses of all concomitant medications for 30 days prior to study entry and agree to remain on stable doses for the duration of the study.
- 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
Exclusion Criteria14
- 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).
- History of gene therapy, cell transplants, or brain surgery aimed at treating the medical condition of interest within 1 year of the screening visit.
- Prior treatment with SLS-005, any other intravenous trehalose formulation, or known hypersensitivity to trehalose.
- Current diagnosis and/or healthcare professional-recommended treatment (medication and/or diet) of diabetes mellitus type 1 or type 2.
- HbA1c greater than or equal to 6.5% at the screening visit
- 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
- Laboratory results at screening that indicate inadequate renal function
- Any current cardiovascular disease or abnormality on 12-lead electrocardiogram (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 #4 or #5 under Suicidal Ideation or any “yes” response under Suicidal Behavior 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.
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Interventions
SLS-005-204 is an open-label basket study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) for treatment of adults with selected neurodegenerative diseases characterized by accumulation of abnormal protein aggregates. The study plans to enroll 15 to 18 participants comprised of up to 4 participants with amyotrophic lateral sclerosis (ALS), up to 10 participants with Huntington’s disease (HD), and up to 4 participants with spinocerebellar ataxia type-3 (SCA3). The study consists of a 2-week screening period, a 24-week open-label treatment period, and a 2-week safety follow-up period. Eligible participants will receive SLS-005 administered as a once weekly intravenous infusion at a dose of 0.75 g/kg over 60 minutes for volumes less than or equal to 600 mL or 90 minutes for volumes greater than 600mL over a treatment period of 24 weeks. The total volume of the infusion will be based on participant weight and dose of 0.75 g/kg. An additional 52 weeks of open-label SLS-005 treatment is planned for consenting participants who are determined to be medically appropriate after their completion of the primary 24-week open-label treatment period. The clinical facility staff will administer each study treatment at the study centre. First patient doses will be witnessed by a Sponsor representative to ensure adherence to the administration procedure outlined in the study protocol. Investigational product dispensing and administration will also be documented as per study guidelines which will be monitored, and accountability assessed by Sponsor representatives.
Locations(2)
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ACTRN12621001755820