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

Merit E. Cudkowicz, MD

Status and phase: Completed; Phase 3; Phase 2

Conditions: Amyotrophic Lateral Sclerosis

Treatments: Drug: SLS-005; Drug: Matching Placebo

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT05136885

2019P003518E

Details and patient eligibility

About

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 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.

Enrollment: 161 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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161 participants in 2 patient groups, including a placebo group

SLS-005

Experimental group

Description:

SLS-005 is administered via infusion once weekly for 24 weeks.

Treatment:

Drug: SLS-005

Matching Placebo

Placebo Comparator group

Description:

Matching placebo is administered via infusion once weekly for 24 weeks.

Treatment:

Drug: Matching Placebo