HEALEY ALS Platform Trial - Regimen C CNM-Au8

Phase 2

Completed

• Conditions: Amyotrophic Lateral Sclerosis
• Interventions: Drug: Matching Placebo; Drug: CNM-Au8

• Registration Number: NCT04414345
• Lead Sponsor: Merit E. Cudkowicz, MD

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 C will evaluate the safety and efficacy of a single study drug, CNM-Au8, 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 C - CNM-Au8, the participant will complete a screening visit to assess additional Regimen C eligibility criteria. Once Regimen C eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active CNM-Au8 or matching placebo.

Regimen C will enroll by invitation, as participants may not choose to enroll in Regimen C. 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 C.

For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.

Study Timeline

• Study First Submitted: 2020-06-01
• Study First Submitted QC: 2020-06-01
• Study First Posted: 2020-06-04
• Study Start: 2020-07-30
• Primary Completion: 2022-04-13
• Study Completion: 2023-03-07
• Results First Submitted: 2023-04-06
• Status Verified: 2023-06
• Results First Submitted QC: 2023-06-30
• Last Update Submitted: 2023-06-30
• Results First Posted: 2023-07-25
• Last Update Posted: 2023-07-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 161

Inclusion Criteria

• No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).

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Exclusion Criteria

• The following exclusion criterion is in addition to the exclusion criteria specified in the Master Protocol (NCT NCT04297683).

  1. History of allergy to gold, gold salts, or colloidal gold preparations.

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Matching Placebo	Matching Placebo	Administration: Oral Dosage: 2 bottles daily
CNM-Au8	CNM-Au8	Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily

Primary Outcome Measures

Name	Time	Method
Disease Progression as Assessed by the ALSFRS-R Total Score	Baseline to 24 Weeks	Change in disease severity as measured by the ALS Functional Rating Scale-Revised (ALSFRS-R) total score using a Bayesian repeated measures model that accounts for loss to follow-up due to mortality. 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.
Mortality Event Rate	Baseline to 24 Weeks	Mortality is defined as death or death equivalent. A participant is determined to meet the criteria of death equivalent if permanent assisted ventilation (PAV) is used for more than 22 hours per day for more than seven days in a row. The rate of mortality was estimated from a Bayesian shared-parametric model that assumed exponentially distributed survival times.

Secondary Outcome Measures

Name	Time	Method
Muscle Strength	Baseline to 24 Weeks	Change in muscle strength as measured isometrically using hand-held dynamometry (HHD).
Number of Participants That Experienced Death or Death Equivalent	24 Weeks	The number of participants who died or met the criterion for a death equivalent from the date of their baseline visit to the end of the Week 24visit window (generally 175 days after baseline). The death equivalent criterion is use of permanent assisted ventilation (PAV) for more than 22hours per day for more than 7 days in a row.
Respiratory Function	Baseline to 24 Weeks	Change in respiratory function as assessed by slow vital capacity (SVC).

Trial Locations

Locations (1)

Healey Center for ALS at Mass General; 🇺🇸 Boston, Massachusetts, United States