Summary

for people ages 18-75 (full criteria)
at San Francisco, California and other locations
study started
estimated completion

Description

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.

Official Title

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

Details

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.

Keywords

Amyotrophic Lateral Sclerosis ALS Sclerosis Motor Neuron Disease Adrenocorticotropic Hormone Acthar

Eligibility

You can join if…

Open to people ages 18-75

  1. Is 18-75 years of age at Screening
  2. Has ALS symptom onset within 2 years prior to Screening
  3. Has forced vital capacity (FVC) no higher than 60% at screening
  4. If taking riluzole, is on a stable dose for 4 weeks before Screening

You CAN'T join if...

  1. Has tracheostomy, diaphragm pacing, or an ongoing need for assisted ventilation of any type
  2. Has used any medication within a time period not allowed per protocol
  3. Has history of Type 1 or Type 2 diabetes mellitus, or any clinically significant infection
  4. Used edaravone less than 1 week before Screening
  5. Received any stem cell replacement therapy
  6. Used steroids within a time period not allowed per protocol

Locations

  • University of California San Francisco accepting new patients
    San Francisco California 94143 United States
  • California Pacific Medical Center accepting new patients
    San Francisco California 94115 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mallinckrodt
ID
NCT03068754
Phase
Phase 2/3
Study Type
Interventional
Last Updated