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

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ClinicalTrials.gov Identifier: NCT05136885
Recruitment Status : Not yet recruiting
First Posted : November 29, 2021
Last Update Posted : November 29, 2021
Sponsor:
Collaborator:
Seelos Therapeutics, Inc.
Information provided by (Responsible Party):
Merit E. Cudkowicz, MD, Massachusetts General Hospital

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.


Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: SLS-005 Drug: Matching Placebo Phase 2 Phase 3

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 160 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose
Estimated Study Start Date : December 2021
Estimated Primary Completion Date : April 2023
Estimated Study Completion Date : October 2023


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




Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Respiratory Function [ Time Frame: 24 Weeks ]
    Change in respiratory function over time as measured by Slow Vital Capacity (SVC).

  2. Muscle Strength [ Time Frame: 24 Weeks ]
    Change in muscle strength over time as measured isometrically using hand-held dynamometry (HHD).

  3. Survival [ Time Frame: 24 Weeks ]
    Comparison of rate of occurrence between groups.



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05136885


Contacts
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Contact: Healey Center for ALS at Massachusetts General Hospital 833-425-8257 (HALT ALS) healeyalsplatform@mgh.harvard.edu

Sponsors and Collaborators
Merit E. Cudkowicz, MD
Seelos Therapeutics, Inc.
Investigators
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Principal Investigator: Merit Cudkowicz, MD Massachusetts General Hospital
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Responsible Party: Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT05136885    
Other Study ID Numbers: 2019P003518E
First Posted: November 29, 2021    Key Record Dates
Last Update Posted: November 29, 2021
Last Verified: November 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Merit E. Cudkowicz, MD, Massachusetts General Hospital:
ALS
Placebo-Controlled
Double-Blind
Regimen Specific Appendix
Lou Gehrig's Disease
Trehalose
Seelos Therapeutics
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases