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

• ClinicalTrials.gov Identifier: NCT05136885
• Recruitment Status: Enrolling by invitation
• First Posted: November 29, 2021
• Last Update Posted: May 17, 2023
• Sponsor: Merit E. Cudkowicz, MD
• Collaborator: Seelos Therapeutics, Inc.
• Information provided by (Responsible Party): Merit E. Cudkowicz, MD, Massachusetts General Hospital

Tracking Information

• First Submitted Date: November 14, 2021
• First Posted Date: November 29, 2021
• Last Update Posted Date: May 17, 2023
• Actual Study Start Date: February 21, 2022
• Estimated Primary Completion Date: August 2023 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: November 14, 2021)

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.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: November 14, 2021)

• 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.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose
• Official Title: HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose

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.

• Study Type: Interventional
• Study Phase: Phase 2; Phase 3
• Study Design: Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
• Condition: Amyotrophic Lateral Sclerosis

Intervention

• 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

Study Arms

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

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Enrolling by invitation
• Estimated Enrollment (submitted: November 14, 2021): 160
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: September 2023
• Estimated Primary Completion Date: August 2023 (Final data collection date for primary outcome measure)

Eligibility Criteria

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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT05136885
• Other Study ID Numbers: 2019P003518E
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Merit E. Cudkowicz, MD, Massachusetts General Hospital
• Original Responsible Party: Same as current
• Current Study Sponsor: Merit E. Cudkowicz, MD
• Original Study Sponsor: Same as current
• Collaborators: Seelos Therapeutics, Inc.

Investigators

• Principal Investigator: Merit Cudkowicz, MD; Massachusetts General Hospital

• PRS Account: Massachusetts General Hospital
• Verification Date: May 2023