HEALEY ALS Platform Trial - Regimen C CNM-Au8

NCT04414345 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 2019P003518C
• Study Type: interventional
• Target: 161
• Locations: 1 country
• Sites: 1

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.

Conditions

Amyotrophic Lateral Sclerosis

Keywords

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

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (3)

• Respiratory Function

  Baseline to 24 Weeks

• Muscle Strength

  Baseline to 24 Weeks

• Number of Participants That Experienced Death or Death Equivalent

  24 Weeks

Study Arms (2)

CNM-Au8

EXPERIMENTAL

Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily

Drug: CNM-Au8

Matching Placebo

PLACEBO COMPARATOR

Administration: Oral Dosage: 2 bottles daily

Drug: Matching Placebo

Interventions

CNM-Au8 DRUG

Drug: CNM-Au8 Administration: Oral Dosage: 30 mg or 60 mg daily

CNM-Au8

Matching Placebo DRUG

Drug: Matching Placebo Administration: Oral Dosage: 2 bottles daily

Matching Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may not qualify if:

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

Sponsors & Collaborators

• Merit E. Cudkowicz, MD: lead
• Clene Nanomedicine: collaborator

Study Sites (1)

Healey Center for ALS at Mass General

Boston, Massachusetts, 02114, United States

Location

Related Publications (1)

• Writing Committee for the HEALEY ALS Platform Trial; Berry JD, Maragakis NJ, Macklin EA, Chibnik LB, Quintana M, Saville BR, Detry MA, Vestrucci M, Marion J, McGlothlin A, Stommel EW, Chase M, Pothier L, Harkey BA, Yu H, Sherman A, Shefner J, Hall M, Kittle G, Babu S, Andrews J, D'Agostino D, Tustison E, Scirocco E, Giacomelli E, Alameda G, Locatelli E, Ho D, Quick A, Ajroud-Driss S, Katz J, Heitzman D, Appel SH, Shroff S, Felice KJ, Simmons Z, Miller T, Olney N, Weiss MD, Goutman SA, Fernandes JA Jr, Jawdat O, Owegi MA, Foster L, Vu T, Ilieva H, Newman DS, Arcila-Londono X, Jackson C, Ladha S, Heiman-Patterson T, Caress J, Swenson A, Peltier A, Lewis R, Fee D, Elliott M, Bedlack R, Kasarskis EJ, Elman L, Rosenfeld J, Walk D, McIlduff CE, Twydell P, Young E, Johnson K, Rezania K, Goyal NA, Cohen JA, Benatar M, Jones V, Glass J, Shah J, Beydoun SR, Wymer JP, Zilliox L, Nayar S, Pattee GL, Martinez-Thompson J, Rynders A, Evan J, Evan J, Hartford A, Sepassi M, Ho KS, Glanzman R, Greenberg B, Hotchkin MT, Paganoni S, Cudkowicz ME; HEALEY ALS Platform Trial Study Group. CNM-Au8 in Amyotrophic Lateral Sclerosis: The HEALEY ALS Platform Trial. JAMA. 2025 Feb 17;333(13):1138-49. doi: 10.1001/jama.2024.27643. Online ahead of print.

  PMID: 40067821 DERIVED

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Results Point of Contact

• Title: Healey Center for ALS Project Management
• Organization: Healey Center for ALS at Massachusetts General Hospital

healeyalsplatform@mgh.harvard.edu

833-425-8257 (HALT ALS)

Study Officials

• Merit Cudkowicz

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Chief, Neurology Department

Study Record Dates

• First Submitted: June 1, 2020
• First Posted: June 4, 2020
• Study Start: July 30, 2020
• Primary Completion: April 13, 2022
• Study Completion: March 7, 2023
• Last Updated: July 25, 2023
• Results First Posted: July 25, 2023

Record last verified: 2023-06

Locations

US (1)