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

NCT ID: NCT05136885

Last Updated: 2025-05-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 161 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-02-21
• Study Completion Date: 2023-08-25

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.

Conditions

Amyotrophic Lateral Sclerosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

SLS-005

SLS-005 is administered via infusion once weekly for 24 weeks.

Group Type: EXPERIMENTAL

SLS-005

Intervention Type: DRUG

Administration: Infusion Dose: 0.75 g/kg weekly

Matching Placebo

Matching placebo is administered via infusion once weekly for 24 weeks.

Group Type: PLACEBO_COMPARATOR

Matching Placebo

Intervention Type: DRUG

Administration: Infusion Dose: equivalent weight-based volume as described for trehalose

Interventions

SLS-005

Administration: Infusion Dose: 0.75 g/kg weekly

Intervention Type: DRUG

Matching Placebo

Administration: Infusion Dose: equivalent weight-based volume as described for trehalose

Intervention Type: DRUG

Eligibility Criteria

Exclusion Criteria

1. Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus

2. Master Protocol screening glucose >140 mg/dl

3. Prior treatment with IV trehalose or known hypersensitivity to trehalose

4. Current use of oral trehalose (see prohibited medication Section 5.9)

5. Inability for participant to return to site for weekly drug administration, until approved for home infusions

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Seelos Therapeutics, Inc.

INDUSTRY

Sponsor Role: collaborator

Merit E. Cudkowicz, MD

OTHER

Sponsor Role: lead

Responsible Party

Merit E. Cudkowicz, MD

Chief, Neurology Department

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Merit Cudkowicz, MD

Role: PRINCIPAL_INVESTIGATOR

Massachusetts General Hospital

Locations

Healey Center for ALS at Mass General

Boston, Massachusetts, United States

Countries

United States

References

HEALEY ALS Platform Trial; HEALEY ALS Platform Trial Study Group. Safety and efficacy of trehalose in amyotrophic lateral sclerosis (HEALEY ALS Platform Trial): an adaptive, phase 2/3, double-blind, randomised, placebo-controlled trial. Lancet Neurol. 2025 Jun;24(6):500-511. doi: 10.1016/S1474-4422(25)00173-5.

Reference Type: DERIVED

PMID: 40409314 (View on PubMed)

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

2019P003518E

Identifier Type: -

Identifier Source: org_study_id