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

Phase 2

Completed

Brief Summary: 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: 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.

Recruitment & Eligibility

Inclusion Criteria

No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

Exclusion Criteria

Arm && Interventions

Group	Intervention	Description
SLS-005	SLS-005	SLS-005 is administered via infusion once weekly for 24 weeks.
Matching Placebo	Matching Placebo	Matching placebo is administered via infusion once weekly for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Disease Progression as Assessed by the ALSFRS-R Slope	24 Weeks	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.
Mortality Event Rate	Baseline to 24 Weeks	Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.

Secondary Outcome Measures

Name	Time	Method
Muscle Strength	Baseline to 24 Weeks	Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).
Number of Participants That Expirenced Death or Death Equivalent	Baseline to 24 Weeks	The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24 visit window (generally 175 days after baseline). The death equivalent criterion is the use of permanent assisted ventilation (PAV) for more than 22 hours per day for more than 7 days in a row.
Respiratory Function	Baseline to 24 Weeks	Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

Locations (1): Healey Center for ALS at Mass General
🇺🇸
Boston, Massachusetts, United States