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

Overview

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.

Full Title of Study: “HEALEY ALS Platform Trial – Regimen E SLS-005 – Trehalose”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Randomized
    • Intervention Model: Parallel Assignment
    • Primary Purpose: Treatment
    • Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
  • Study Primary Completion Date: April 2023

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.

Interventions

  • Drug: SLS-005
    • Administration: Infusion Dose: 0.75 g/kg weekly
  • Drug: Matching Placebo
    • Administration: Infusion Dose: equivalent weight-based volume as described for trehalose

Arms, Groups and Cohorts

  • Experimental: SLS-005
    • SLS-005 is administered via infusion once weekly for 24 weeks.
  • Placebo Comparator: Matching Placebo
    • Matching placebo is administered via infusion once weekly for 24 weeks.

Clinical Trial Outcome Measures

Primary Measures

  • Disease Progression
    • Time Frame: 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.

Secondary Measures

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

Participating in This Clinical Trial

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

Gender Eligibility: All

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Merit E. Cudkowicz, MD
  • Collaborator
    • Seelos Therapeutics, Inc.
  • Provider of Information About this Clinical Study
    • Sponsor-Investigator: Merit E. Cudkowicz, MD, Chief, Neurology Department – Massachusetts General Hospital
  • Overall Official(s)
    • Merit Cudkowicz, MD, Principal Investigator, Massachusetts General Hospital

Clinical trials entries are delivered from the US National Institutes of Health and are not reviewed separately by this site. Please see the identifier information above for retrieving further details from the government database.

At TrialBulletin.com, we keep tabs on over 200,000 clinical trials in the US and abroad, using medical data supplied directly by the US National Institutes of Health. Please see the About and Contact page for details.