HEALEY ALS Platform Trial - Regimen C CNM-Au8

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.

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: CNM-Au8; Drug: Matching Placebo

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 Type: Interventional
• Enrollment (Actual): 161
• Phase: Phase 2; Phase 3

Contacts and Locations

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02114
      • Healey Center for ALS at Mass General

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

Inclusion Criteria:

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

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Quadruple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: CNM-Au8; Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily	Drug: CNM-Au8; Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily
Placebo Comparator: Matching Placebo; Administration: Oral Dosage: 2 bottles daily	Drug: Matching Placebo; Drug: Matching Placebo Administration: Oral Dosage: 2 bottles daily

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Disease Progression as Assessed by the ALSFRS-R Total Score	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.	Baseline to 24 Weeks
Mortality Event Rate	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.	Baseline to 24 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Respiratory Function	Change in respiratory function as assessed by slow vital capacity (SVC).	Baseline to 24 Weeks
Muscle Strength	Change in muscle strength as measured isometrically using hand-held dynamometry (HHD).	Baseline to 24 Weeks
Number of Participants That Experienced Death or Death Equivalent	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.	24 Weeks

Collaborators and Investigators

• Sponsor: Merit E. Cudkowicz, MD
• Collaborators: Clene Nanomedicine
• Investigators: Principal Investigator: Merit Cudkowicz, Massachusetts General Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 30, 2020
• Primary Completion (Actual): April 13, 2022
• Study Completion (Actual): March 7, 2023

Study Registration Dates

• First Submitted: June 1, 2020
• First Submitted That Met QC Criteria: June 1, 2020
• First Posted (Actual): June 4, 2020

Study Record Updates

• Last Update Posted (Actual): July 25, 2023
• Last Update Submitted That Met QC Criteria: June 30, 2023
• Last Verified: June 1, 2023

More Information

Terms related to this study

• Keywords: ALS; Double-Blind; Placebo-Controlled; Lou Gehrig's Disease; Regimen-Specific Appendix; CNM-Au8; Clene Nanomedicine
• Additional Relevant MeSH Terms: Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: 2019P003518C

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No