Summary

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

Description

Summary

This is a multicenter, multiple dose study to examine the effect of H.P. Acthar® (Acthar) on functional decline in adult subjects with amyotrophic lateral sclerosis (ALS).

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 subjects with amyotrophic lateral sclerosis (ALS). Approximately 213 subjects will be enrolled.

Following a screening period of up to 28 days, subjects 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.

Subjects who complete the 36 week double-blind treatment period are eligible to enter an Open Label Extension phase where all subjects 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. Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, or clinically probable ALS based on revised El Escorial criteria.
  2. ALS symptom onset ≤ 2 years prior to the Screening Visit.
  3. Subjects who have been on riluzole may enter the study if they have been on a stable dose of 50 mg BID for ≥ 4 weeks prior to the Screening Visit and, if possible, should remain on that dose throughout the study.
  4. Forced vital capacity (FVC) ≥ 60% at the Screening Visit.
  5. Systolic blood pressure ≤ 140 mm Hg and a mean diastolic blood pressure of ≤ 90 mm Hg at the Screening and Baseline Visits.

You CAN'T join if...

  1. History of use of adrenocorticotropic hormone (ACTH) preparations for treatment of ALS.
  2. Any medical condition known to have an association with motor neuron dysfunction(other than ALS) which might confound or obscure the diagnosis of ALS.
  3. Subject has tracheostomy, diaphragm pacing, or ongoing (used for greater than 7 consecutive days in the 4 weeks prior to the Screening Visit) need for assisted ventilation of any type.
  4. History of chronic active hepatitis including active or chronic hepatitis B, or acute or chronic hepatitis C.
  5. History of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
  6. Clinically significant infection requiring intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the Screening Visit.
  7. Subject has used edaravone in the 1 week prior to the Screening Visit or in the 2 weeks prior to the Randomization Visit.

Locations

  • University of California San Diego accepting new patients
    La Jolla California 92037 United States
  • University of California Irvine Medical Center accepting new patients
    Orange California 92868 United States
  • Loma Linda University Health System, Department of Neurology accepting new patients
    Loma Linda California 92354 United States
  • Keck School of Medicine, University of Southern California accepting new patients
    Los Angeles California 90033 United States
  • University of California Los Angeles accepting new patients
    Los Angeles California 90095 United States
  • Las Vegas Clinic accepting new patients
    Las Vegas Nevada 89145 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
Mallinckrodt
ID
NCT03068754
Phase
Phase 2
Study Type
Interventional
Last Updated
June 1, 2018