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

NCT05136885 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518E
• Study Type: interventional
• Target: 161
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

ALS; Placebo-Controlled; Double-Blind; Regimen Specific Appendix; Lou Gehrig's Disease; Trehalose; Seelos Therapeutics

Outcome Measures

Primary Outcomes (2)

• Disease Progression as Assessed by the ALSFRS-R Slope

  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

• Mortality Event Rate

  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.

  Baseline to 24 Weeks

Secondary Outcomes (3)

• Respiratory Function

  Baseline to 24 Weeks

• Muscle Strength

  Baseline to 24 Weeks

• Number of Participants That Experienced Death or Death Equivalent

  Baseline to 24 Weeks

Study Arms (2)

SLS-005

EXPERIMENTAL

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

Drug: SLS-005

Matching Placebo

PLACEBO COMPARATOR

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

Drug: Matching Placebo

Interventions

SLS-005 DRUG

Administration: Infusion Dose: 0.75 g/kg weekly

SLS-005

Matching Placebo DRUG

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

Matching Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

• 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

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Seelos Therapeutics, Inc.: collaborator

Study Sites (1)

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

Location

Related Publications (1)

• 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.

  PMID: 40409314 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Healey Center for ALS Project Management
• Organization: Healey Center for ALS at Massachusetts General Hospital

healeyalsplatform@mgh.harvard.edu

833-425-8257

Study Officials

• Merit Cudkowicz, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief, Neurology Department

Study Record Dates

• First Submitted: November 14, 2021
• First Posted: November 29, 2021
• Study Start: February 21, 2022
• Primary Completion: August 25, 2023
• Study Completion: August 25, 2023
• Last Updated: May 14, 2025
• Results First Posted: August 28, 2024

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)