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

Sponsor

Merit E. Cudkowicz, MD (Other)

Overall Status

Enrolling by invitation

CT.gov ID

NCT05136885

Collaborator

Seelos Therapeutics, Inc. (Industry)

160

Enrollment

Location

Arms

19.3

Anticipated Duration (Months)

8.3

Patients Per Site Per Month

Study Details

Study Description

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.

Study Design

Study Type:

Interventional

Anticipated 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

Actual Study Start Date :

Feb 21, 2022

Anticipated Primary Completion Date :

Apr 1, 2023

Anticipated Study Completion Date :

Oct 1, 2023

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

Disease Progression [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

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

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts 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).

Current diagnosis or healthcare professional-recommended treatment (medication, exercise or diet) of diabetes mellitus
Master Protocol screening glucose >140 mg/dl
Prior treatment with IV trehalose or known hypersensitivity to trehalose
Current use of oral trehalose (see prohibited medication Section 5.9)
Inability for participant to return to site for weekly drug administration, until approved for home infusions