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

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: SLS-005; Drug: Matching Placebo

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.

• Study Type: Interventional
• Enrollment (Actual): 161
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Healey Center for ALS at Mass General

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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).

  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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Merit E. Cudkowicz, MD
• Collaborators: Seelos Therapeutics, Inc.
• Investigators: Principal Investigator: Merit Cudkowicz, MD, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): February 21, 2022
• Primary Completion (Actual): August 25, 2023
• Study Completion (Actual): August 25, 2023

Study Registration Dates

• First Submitted: November 14, 2021
• First Submitted That Met QC Criteria: November 14, 2021
• First Posted (Actual): November 29, 2021

Study Record Updates

• Last Update Posted (Actual): October 4, 2023
• Last Update Submitted That Met QC Criteria: October 2, 2023
• Last Verified: October 1, 2023

More Information

Terms related to this study

• Keywords: ALS; Double-Blind; Placebo-Controlled; Lou Gehrig's Disease; Trehalose; Seelos Therapeutics; Regimen Specific Appendix
• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 2019P003518E

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No