Clinical trial NCT05136885: HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose
Official title: HEALEY ALS Platform Trial - Regimen E SLS-005 - Trehalose
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Amyotrophic Lateral Sclerosis
Keywords: ALS
Placebo-Controlled
Double-Blind
Regimen Specific Appendix
Lou Gehrig's Disease
Trehalose
Seelos Therapeutics
Lead sponsor: Massachusetts General Hospital
Sponsor class: OTHER
Collaborators: name: Seelos Therapeutics, Inc.
First posted: 2021-11-29
Enrollment: 161
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Disease Progression as Assessed by the ALSFRS-R Slope
timeFrame: 24 Weeks
measure: Mortality Event Rate
timeFrame: Baseline to 24 Weeks
Secondary outcomes:
measure: Respiratory Function
timeFrame: Baseline to 24 Weeks
measure: Muscle Strength
timeFrame: Baseline to 24 Weeks
measure: Number of Participants That Experienced Death or Death Equivalent
timeFrame: Baseline to 24 Weeks
Other outcomes:
Locations:
city: Boston
country: United States
facility: Healey Center for ALS at Mass General
state: Massachusetts
Study officials:
affiliation: Massachusetts General Hospital
name: Merit Cudkowicz, MD
role: PRINCIPAL_INVESTIGATOR
References: