HEALEY ALS Platform Trial - Regimen C CNM-Au8

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

HEALEY ALS Platform Trial - Regimen C CNM-Au8

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04414345
• Other study ID #: 2019P003518C
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: July 30, 2020
• Est. completion date: March 7, 2023

Study information

• Verified date: June 2023
• Source: Massachusetts General Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 161
• Est. completion date: March 7, 2023
• Est. primary completion date: April 13, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Amyotrophic Lateral Sclerosis
• Motor Neuron Disease

Intervention

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

Locations

Country	Name	City	State
United States	Healey Center for ALS at Mass General	Boston	Massachusetts

Sponsors (2)

Lead Sponsor	Collaborator
Merit E. Cudkowicz, MD	Clene Nanomedicine

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Respiratory Function	Change in respiratory function as assessed by slow vital capacity (SVC).	Baseline to 24 Weeks
Secondary	Muscle Strength	Change in muscle strength as measured isometrically using hand-held dynamometry (HHD).	Baseline to 24 Weeks
Secondary	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