HEALEY ALS Platform Trial - Regimen C CNM-Au8

• ClinicalTrials.gov Identifier: NCT04414345
• Recruitment Status: Active, not recruiting
• First Posted: June 4, 2020
• Last Update Posted: June 30, 2022
• Sponsor: Merit E. Cudkowicz, MD
• Collaborator: Clene Nanomedicine
• Information provided by (Responsible Party): Merit E. Cudkowicz, MD, Massachusetts General Hospital

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 C will evaluate the safety and efficacy of a single study drug, CNM-Au8, in participants with ALS.

Condition or disease	Intervention/treatment	Phase
Amyotrophic Lateral Sclerosis	Drug: CNM-Au8; 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 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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 161 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 C CNM-Au8
• Actual Study Start Date: July 30, 2020
• Actual Primary Completion Date: April 13, 2022
• Estimated Study Completion Date: April 2023

Arms and Interventions

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

Outcome Measures

Primary Outcome Measures :

1. Disease Progression [ Time Frame: 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 :

1. Combined Assessment of Function and Survival [ Time Frame: 24 Weeks ]
   Combined analysis of change in ALS Functional Rating Scale-Revised (ALSFRS-R) total score and survival free of permanent assisted ventilation. For the 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.
2. Respiratory Function [ Time Frame: 24 Weeks ]
   Change in respiratory function over time as measured by Slow Vital Capacity (SVC).
3. Survival [ Time Frame: 24 Weeks ]
   Occurrence of death or initiation of permanent assisted ventilation.

Eligibility Criteria

• Ages Eligible for Study: 18 Years and older (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

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.

Contacts and Locations

Locations

United States, Massachusetts

Healey Center for ALS at Mass General

Boston, Massachusetts, United States, 02114

Sponsors and Collaborators

Merit E. Cudkowicz, MD

Clene Nanomedicine

Investigators

• Principal Investigator: Merit Cudkowicz; Massachusetts General Hospital

More Information

• Responsible Party: Merit E. Cudkowicz, MD, Chief, Neurology Department, Massachusetts General Hospital
• ClinicalTrials.gov Identifier: NCT04414345
• Other Study ID Numbers: 2019P003518C
• First Posted: June 4, 2020
• Last Update Posted: June 30, 2022
• Last Verified: June 2022

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Merit E. Cudkowicz, MD, Massachusetts General Hospital:

ALS; Placebo-Controlled; Double-Blind; Regimen-Specific Appendix; Lou Gehrig's Disease; CNM-Au8; Clene Nanomedicine

Additional relevant MeSH terms:

Motor Neuron Disease; Amyotrophic Lateral Sclerosis; Neurodegenerative Diseases; Nervous System Diseases; Neuromuscular Diseases; Spinal Cord Diseases; Central Nervous System Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Metabolic Diseases