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

Study Purpose:

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).

Disease:

Amyotrophic Lateral Sclerosis (ALS), Familial ALS, Sporadic ALS

Study Type:

Interventional Trial

Study Category:

Drug Trial

Study Status:

Active, currently recruiting

Phase:

Phase II

Study Chair(s)/Principal Investigator(s):

Susan VanMeter, Mallinckrodt

Clinicaltrials.gov ID (11 digit #):

NCT03068754

Neals Affiliated?

Coordinating Center Contact Information

Roman Bilyk / .(JavaScript must be enabled to view this email address) / 800-556-3314
.(JavaScript must be enabled to view this email address)

Full Study Summary:

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 195 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 ) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3 week taper.

Study Sponsor:
Mallinckrodt
Participant Duration:

Estimated Enrollment:
195
Estimated Study Start Date:
06/14/2017

Estimated Study Completion Date:
12/31/2019
Posting Last Modified Date:
08/25/2017
Date Study Added to alsconsortium.org:
08/25/2017

Eligibility Criteria

Gender:
Female, Male
Minimum Age:
18
Maximum Age:
75
Min Vital Capacity (% predicted normal):
60

Time since Symptom Onset:
<24 months
Time since Diagnosis:

Can participants use Riluzole?
Yes

Inclusion Criteria:

Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, or clinically probable ALS based on revised El Escorial criteria.
ALS symptom onset ≤ 2 years prior to the Screening Visit.
On riluzole 50 mg twice a day for ≥ 4 weeks prior to the Screening Visit.
Forced vital capacity (FVC) ≥ 60% at the Screening Visit.
Systolic blood pressure ≤ 140 mm Hg and a mean diastolic blood pressure of ≤ 90 mm Hg at the Screening and Baseline Visits.

Exclusion Criteria:

History of use of adrenocorticotropic hormone (ACTH) preparations for treatment of ALS.
Any medical condition known to have an association with motor neuron dysfunction (other than ALS) which might confound or obscure the diagnosis of ALS.
Requires nocturnal continuous positive airway pressure or bilevel positive airway pressure.
Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type at Screening Visit.
Known contraindication(s) to Acthar.
History of chronic active hepatitis including active or chronic hepatitis B, or acute or chronic hepatitis C.
History of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
Clinically significant infection requiring intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the Screening Visit.

Site Contact Information

Neuromuscular Research Center
Phoenix, Arizona 85208
United States
Loma Linda University Health System, Department of Neurology
Recruiting Loma Linda, California 92354
United States
Colorado Springs Neurological Associates
Colorado Springs, Colorado 80907
United States
GW Medical Faculty Associates
Washington, D.C., District of Columbia 20037
United States
University of South Florida
Tampa, Florida 33609
United States
Wesley Neurology Clinic
Cordova, Tennessee 38018
United States
Texas Neurology, P.A.
Dallas, Texas 75215
United States
Medical College of Wisconsin/Froedtert Hospital
Milwaukee, Wisconsin 53226
United States