Amyotrophic Lateral Sclerosis (ALS)
Conditions
Keywords
ALS, Placebo-Controlled, Double-Blind, Regimen Specific Appendix, Lou Gehrig's Disease, NUZ-001, Neurizon
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 I will evaluate the safety and efficacy of a single study drug, NUZ-001, 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 I NUZ-001, the participant will complete a screening visit to assess additional Regimen I eligibility criteria. Once Regimen I eligibility criteria are confirmed, participants will complete a baseline assessment and be randomized in a 3:1 ratio to either active NUZ-001 or matching placebo. Regimen I will enroll by invitation, as participants may not choose to enroll in Regimen I. 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 I. For a list of enrolling sites, please see the HEALEY ALS Platform Trial Master Protocol under NCT04297683.
Interventions
DRUGNUZ-001
NUZ-001 is administered orally once daily for 36 weeks.
DRUGMatching placebo
Matching placebo is administered orally once daily for 36 weeks.
Sponsors
Merit E. Cudkowicz, MD
Neurizon Therapeutics Limited
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* No additional inclusion criteria beyond the inclusion criteria specified in the Master Protocol (NCT NCT04297683).
Exclusion criteria
* The following
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Disease Progression | Baseline to 36 Weeks | Change in disease severity over time as measured by the Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) total score and survival. 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
| Measure | Time frame | Description |
|---|---|---|
| Respiratory Function | Baseline to 36 Weeks | Change in respiratory function over time as measured by Slow Vital Capacity (SVC) |
| Survival | Baseline to 36 Weeks | Survival evaluated as time to death or permanent assisted ventilation (PAV) |
Countries
United States
Contacts
PRINCIPAL_INVESTIGATORMerit Cudkowicz, MD
Massachusetts General Hospital
Outcome results
None listed