About 213 people with ALS will participate in this study. There will be locations in North and South America. During the first part, participants will be randomly assigned to a group (like by flipping a coin). Out of every 3: * 2 will get the study drug * 1 will get a look-alike with no drug in it (placebo) During the second part, everyone will get the study drug. Participation will help doctors find out if Acthar can help or slow down the symptoms of ALS better than placebo.
This is a multicenter, multiple dose study to examine the effect of Acthar on functional decline in adult participants with ALS. Approximately 213 participants will be enrolled. Following a screening period of up to 28 days, participants with ALS and symptom onset (defined as first muscle weakness or dysarthria) ≤ 2 years prior to the Screening Visit will be randomized on a 2:1 basis to receive subcutaneous (SC) Acthar 0.2 mL (16 Units \[U\]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3-week taper. Participants who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase in which all participants will receive Acthar 0.2 mL (16 U) daily.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
143
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival. ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory) A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.
Time frame: Baseline, Week 36
Number of Participants Experiencing an Adverse Event During the Treatment Period
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events.
Time frame: by the end of the treatment period (within 36 Weeks)
Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.
Time frame: by the time of database lock (within 84 weeks)
Treatment Period: Spirometry (%)
Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled \[Forced Vital Capacity (FVC)\] and exhaled \[(Forced Expiratory Volume in one second (FEV1)\].
Time frame: Baseline, Week 36
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Neuromuscular Research Center
Phoenix, Arizona, United States
Mayo Clinic - Arizona
Scottsdale, Arizona, United States
University of California San Diego
La Jolla, California, United States
Loma Linda University Health System, Department of Neurology
Loma Linda, California, United States
Keck School of Medicine, University of Southern California
Los Angeles, California, United States
University of California Los Angeles
Los Angeles, California, United States
University of California Irvine Medical Center
Orange, California, United States
California Pacific Medical Center
San Francisco, California, United States
University of California San Francisco
San Francisco, California, United States
Colorado Springs Neurological Associates
Colorado Springs, Colorado, United States
...and 59 more locations
Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R
The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.
Time frame: Baseline, Week 36
Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R
The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory). The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.
Time frame: Baseline, Week 84