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:

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.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:

Acthar, Placebo




Phase 2/Phase 3

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

Clinical Team Leader, Mallinckrodt

Clinicaltrials.gov ID:


Neals Affiliated?


Coordinating Center Contact Information

Full Study Summary:

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 Sponsor:


Estimated Enrollment:


Estimated Study Start Date:

06 / 22 / 2017

Estimated Study Completion Date:

11 / 25 / 2019

Posting Last Modified Date:

10 / 14 / 2020

Date Study Added to neals.org:

03 / 03 / 2017

Minimum Age:

18 Years

Maximum Age:

75 Years

Can participants use Riluzole?


Inclusion Criteria:

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

Exclusion Criteria:

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

Neuromuscular Research Center

Phoenix, Arizona 85028
United States

Mayo Clinic - Arizona

Scottsdale, Arizona 85259
United States

University of California San Diego

La Jolla, California 92037
United States

Loma Linda University Health System, Department of Neurology

Loma Linda, California 92354
United States

Keck School of Medicine, University of Southern California

Los Angeles, California 90033
United States

University of California Los Angeles

Los Angeles, California 90095
United States

University of California Irvine Medical Center

Orange, California 92868
United States

California Pacific Medical Center

San Francisco, California 94115
United States

University of California San Francisco

San Francisco, California 94143
United States

Colorado Springs Neurological Associates

Colorado Springs, Colorado 80907
United States

Georgetown University

Washington, District of Columbia 20007
United States

George Washington University

Washington, District of Columbia 20037
United States

University of Florida - McKnight Brain Institute

Gainesville, Florida 32611
United States

University of South Florida

Tampa, Florida 33612
United States

Emory University

Atlanta, Georgia 30322
United States

Augusta University

Augusta, Georgia 30912
United States

Indiana University-Neuroscience Center of Excellence/Goodman Hall

Indianapolis, Indiana 46202
United States

University of Kansas Medical Center

Kansas City, Kansas 66160
United States

University of Kentucky Chandler Medical Center

Lexington, Kentucky 40536
United States

John Hopkins Outpatient Center

Baltimore, Maryland 21287
United States

University of Massachusetts Medical School

Worcester, Massachusetts 01655
United States

Mercy Health- Saint Mary's

Grand Rapids, Michigan 49503
United States

Neurology Associates

Lincoln, Nebraska 68510
United States

University of Nebraska Medical Center - Physicians Clinical Neurosciences Center

Omaha, Nebraska 68198
United States

Las Vegas Clinic

Las Vegas, Nevada 89145
United States

Jersey Shore University Medical Center

Neptune, New Jersey 07753
United States

Columbia Presbyterian Hospital

New York, New York 10032
United States

Providence ALS Center

Portland, Oregon 97213
United States

Allegheny General Hospital

Pittsburgh, Pennsylvania 15212
United States

Temple University School of Medicine

Philadelphia, Pennsylvania 19140
United States

Penn State Health Milton S. Hershey Medical Center

Hershey, Pennsylvania 17033
United States

Wesley Neurology Clinic

Cordova, Tennessee 38018
United States

Austin Neuromuscular Center

Austin, Texas 78756
United States

Texas Neurology, P.A.

Dallas, Texas 75214
United States

The Methodist Hospital

Houston, Texas 77030
United States

University of Vermont Medical Center

Colchester, Vermont 05401
United States

VCU Medical Center

Richmond, Virginia 23298
United States

Swedish Neuroscience Institute

Seattle, Washington 98122
United States

Medical College of Wisconsin/Froedtert Hospital

Milwaukee, Wisconsin 53226
United States


Ciudad Autonoma de Buenos Aires, Buenos Aires C1015ABR

STAT Research

Ciudad Autonoma de Buenos Aires, Buenos Aires C1023AAB


Ciudad Autonoma de Buenos Aires, Buenos Aires C1061ABD


Ciudad Autonoma de Buenos Aires, Buenos Aires C1192AAW

Hospital Italiano de Buenos Aires

Ciudad Autonoma de Buenos Aires, Buenos Aires C1199ABB

Hospital Español

Ciudad Autonoma de Buenos Aires, Buenos Aires C1209AAB

Hospital Británico de Buenos Aires

Ciudad Autonoma de Buenos Aires, Buenos Aires C1280AEB


Ciudad de Córdoba, Córdoba X5000BNB

Fundación Scherbovsky

Ciudad de Mendoza, Mendoza CP 5500

Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )

Ciudad De Santa Fe, Santa Fe S3000ASL

Edmonton Kaye Clinic

Edmonton, Alberta T6G 1Z1

Recherche Sepmus inc

Greenfield Park, Quebec J4V 2J2

Centre de recherché du Centre Hospitalier de l'Universite de Montreal (CRCHUM)

Montréal, Quebec H2XOA9

Montreal Neurological Institute & Hospital

Montréal, Quebec H3A 2B4

Biomedica Research Group AV Salvador 149, oficina 1101

Santiago 7500710

Clinica Dávila

Santiago, Región Metropolitana 8431657

Centro de Trastornos del Movimiento (CETRAM)

Santiago, Región Metropolitana 8380815

Centro de Investigaciones Clínicas SAS

Cali 760036

Clinical Research Institute Saltillo S.A. de C.V.

Saltillo, Coahuila 25020

Hospital Universitario "Dr. José Eleuterio González"

Monterrey, Nuevo Leon 64460

SMIQ BRCR Global México

Querétaro City, Querétaro 76090

Clinical Research Institute S.C.

San Lucas Tepetlacalco, Tlalnepantla De Baz 54055

Centro Especializado en Investigación Clínica S.C.

Boca Del Río, Veracruz 94290

Phylasis Clinicas Research

Mexico City 54769

FAICIC Clinical Researc

Veracruz 91900

Hospital Nivel IV Carlos Alberto Seguin Escobedo

Arequipa 04001

Hospital Nacional IV Alberto Sabogal Sologuren

Callao 07016

Hospital Almenara

Lima 15033

Instituto Neuro Cardiovascular de las Américas

Lima 15074

Hospital Nacional Cayetano Heredia

Lima 15102