NCT03068754

Study of Acthar® Gel (Acthar) for Amyotrophic Lateral Sclerosis (ALS)

A Multicenter, Double Blind, Placebo Controlled Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis

• Status: Terminated
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 143 (actual)
• Sponsor: Mallinckrodt · Industry
• Sex: All
• Age: 18 Years – 75 Years
• Healthy volunteers: Not accepted

Summary

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.

Detailed description

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.

Conditions

• Amyotrophic Lateral Sclerosis

Interventions

Type	Name	Description
DRUG	Acthar	Repository corticotropin for subcutaneous injection
DRUG	Placebo	Matching placebo for subcutaneous injection

Timeline

• Start date: 2017-06-22
• Primary completion: 2019-11-25
• Completion: 2019-11-25
• First posted: 2017-03-03
• Last updated: 2020-10-14
• Results posted: 2020-09-03

Locations

69 sites across 7 countries: United States, Argentina, Canada, Chile, Colombia, Mexico, Peru

Regulatory

• FDA-regulated drug study