Arimoclomol in Amyotropic Lateral Sclerosis

Phase 3

Completed

Brief Summary: A multicenter, randomized, double-blind, placebo-controlled, parallel group trial to evaluate the efficacy and safety of arimoclomol in amyotropic lateral sclerosis (ALS)

Recruitment & Eligibility

Inclusion Criteria

Subject meets revised El Escorial criteria for clinically possible, clinically probable / clinically probable ALS laboratory-supported, clinically definite ALS or clinically definite familial ALS laboratory-supported
18 months or less since first appearance of weakness (e.g. limb weakness, dysarthria, dysphagia, shortness of breath)
ALS Functional Rating Scale-Revised (ALSFRS-R) equal to or above 35 and erect (seated) Slow Vital Capacity (SVC) % predicted equal to or above 70% at Screening

Exclusion Criteria

Tracheostomy or use of non-invasive ventilation for more than 2 hours during waking hours at the time of Screening and Baseline
Pregnant or breast-feeding
Current or anticipated use of diaphragmatic pacing
Any other relevant medically significant condition which could present risk to the subject or interfere with the assessment of safety or has an increased risk of causing death during the trial

Arm && Interventions

Group	Intervention	Description
Arimoclomol	Arimoclomol	248 mg arimoclomol base (equivalent to 400 mg arimoclomol citrate) 3 times daily
Placebo	Placebo	248 mg matching placebo 3 times daily

Primary Outcome Measures

Name	Time	Method
Combined Assessment of Function and Survival (CAFS)	Over 76 Weeks	Combined Assessment of Function and Survival (CAFS) is a composite endpoint that includes 1) the change from baseline in revised ALS functional rating scale (ALSFRS-R) and 2) the survival endpoint (time to permanent assisted ventilation \[PAV\], tracheostomy or death). On the ALSFRS-R, 12 functions are rated on 5-point ordinal rating scales (from 0 to 4) with a total score range (minimum and maximum score) of 0-48 (sum of all 12 items). The higher the score the better functioning. For the survival endpoint, the longer time to PAV, tracheostomy, or death the better outcome. A patient's CAFS score represents a patient's rank in the study based on comparing the patient's outcome for both the change in ALSFRS-R and the time to event (PAV, tracheostomy, or death) to the outcome for all other patients in the study in a pairwise fashion. A higher rank score (range 0-1) is considered a better outcome. The reported values are the mean rank scores in each group for the composite endpoint.

Secondary Outcome Measures

Name	Time	Method
Time to Permanent Assisted Ventilation (PAV) / Tracheostomy / Death	Over 76 weeks	Time from baseline to one of the events (PAV / tracheostomy / death). PAV is defined as the first of 7 consecutive days on which PAV was used for \>22 hours/day
Change From Baseline to Week 76 (or End-of-trial) in the Revised ALS Functional Rating Scale (ALSFRS-R)	Week 76 (or end of trial)	The ALSFRS-R score is based on a rating scale where 12 functions are rated on 5-point ordinal rating scales (from 0 to 4) with a maximum score of 48 (sum of all 12 items). The higher the score the better functioning.
Change From Baseline to Week 76 (or End-of-trial) in Percent (%) Predicted Slow Vital Capacity (SVC)	Week 76 (or end of trial)	Slow Vital Capacity (SVC) is a measure of breathing function. SVC measures the volume that can be exhaled from a full inhalation after exhaling to a maximum as slowly as possible. The percent (%) of predicted SVC is reported.

Locations (29): St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
🇺🇸
Phoenix, Arizona, United States
HonorHealth Neurology
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Phoenix, Arizona, United States
UC Irvine Health ALS and Neuromuscular Center
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Orange, California, United States
University of Miami
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Miami, Florida, United States
University of Kansas Medical Center (KUMC) - Landon Center on Aging
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Kansas City, Kansas, United States
Hospital for Special Surgery
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New York, New York, United States
Providence Brain & Spine Institute
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Portland, Oregon, United States
University of Pensylvania, Perelman Center for Advanced Medicine - Penn Neuroscience Center
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Philadelphia, Pennsylvania, United States
University of Texas Southwestern Medical Center
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Dallas, Texas, United States
University of Virginia Health System
🇺🇸
Charlottesville, Virginia, United States
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St. Joseph's Hospital and Medical Center (SJHMC) - Barrow Neurological Institute (BNI) - The Gregory W. Fulton ALS and Neuromuscular Disease Center
🇺🇸Phoenix, Arizona, United States