HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose

Study Overview
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Brief Summary
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The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. Regimen E will evaluate the safety and efficacy of a single study drug, SLS-005 (Trehalose injection, 90.5 mg/mL for intravenous infusion) in participants with ALS.

Detailed Description
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The HEALEY ALS Platform Trial is a perpetual multi-center, multi-regimen clinical trial evaluating the safety and efficacy of investigational products for the treatment of ALS. This trial is designed as a perpetual platform trial. This means that there is a single Master Protocol dictating the conduct of the trial. The HEALEY ALS Platform Trial Master Protocol is registered as NCT04297683. Once a participant enrolls into the Master Protocol and meets all eligibility criteria, the participant will be eligible to be randomized into any currently enrolling regimen. All participants will have an equal chance of being randomized to any currently enrolling regimen. If a participant is randomized to Regimen E SLS-005 - Trehalose, the participant will complete a screening visit to assess additional Regimen E eligibility criteria. Once Regimen E eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active SLS-005 or matching placebo. Regimen E will enroll by invitation, as participants may not choose to enroll in Regimen E. Participants must first enroll into the Master Protocol and be eligible to participate in the Master Protocol before being able to be randomly assigned to Regimen E. For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

Official Title
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HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose

Conditions
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Amyotrophic Lateral Sclerosis

Intervention / Treatment
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* Drug: SLS-005
* Drug: Matching Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683). Exclusion Criteria: The following exclusion criteria are in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683). Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus Master Protocol screening glucose >140 mg/dl Prior treatment with IV trehalose or known hypersensitivity to trehalose Current use of oral trehalose (see prohibited medication Section 5.9) Inability for participant to return to site for weekly drug administration, until approved for home infusions

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: SLS-005<br>SLS-005 is administered via infusion once weekly for 24 weeks. | Drug: SLS-005<br>* Administration: Infusion Dose: 0.75 g/kg weekly<br>|
| Placebo Comparator: Matching Placebo<br>Matching placebo is administered via infusion once weekly for 24 weeks. | Drug: Matching Placebo<br>* Administration: Infusion Dose: equivalent weight-based volume as described for trehalose<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Disease Progression | Change in disease severity over time as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R). Each type of function is scored from 4 (normal) to 0 (no ability), with a maximum total score of 48 and a minimum total score of 0. Patients with higher scores have more physical function. | 24 Weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Respiratory Function | Change in respiratory function over time as measured by Slow Vital Capacity (SVC). | 24 Weeks | 
| Muscle Strength | Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD). | 24 Weeks | 
| Survival | Comparison of rate of occurrence between groups. | 24 Weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, Trehalose, Seelos Therapeutics