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

September 18, 2020 updated by: Mallinckrodt

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

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 Overview

Status

Terminated

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Actual)

143

Phase

  • Phase 2
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Argentina
    • Buenos Aires
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1280AEB
        • Hospital Britanico de Buenos Aires
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1015ABR
        • IADIN
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1023AAB
        • STAT Research
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1061ABD
        • DIABAID
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1192AAW
        • INEBA
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1199ABB
        • Hospital Italiano de Buenos Aires
      • Ciudad Autonoma de Buenos Aires, Buenos Aires, Argentina, C1209AAB
        • Hospital Español
    • Córdoba
      • Ciudad de Córdoba, Córdoba, Argentina, X5000BNB
        • ILAIM
    • Mendoza
      • Ciudad de Mendoza, Mendoza, Argentina, CP 5500
        • Fundación Scherbovsky
    • Santa Fe
      • Ciudad De Santa Fe, Santa Fe, Argentina, S3000ASL
        • Instituto de Neurología y Neurorrehabilitación del Litoral (INNeL )
  • Canada
    • Alberta
      • Edmonton, Alberta, Canada, T6G 1Z1
        • Edmonton Kaye Clinic
    • Quebec
      • Greenfield Park, Quebec, Canada, J4V 2J2
        • Recherche Sepmus inc
      • Montréal, Quebec, Canada, H3A 2B4
        • Montreal Neurological Institute & Hospital
      • Montréal, Quebec, Canada, H2XOA9
        • Centre de Recherche du Centre Hospitalier de l'Université de Montréal (CRCHUM)
  • Chile
      • Santiago, Chile, 7500710
        • Biomedica Research Group AV Salvador 149, oficina 1101
    • Región Metropolitana
      • Santiago, Región Metropolitana, Chile, 8380815
        • Centro de Trastornos del Movimiento (CETRAM)
      • Santiago, Región Metropolitana, Chile, 8431657
        • Clinica Dávila
  • Colombia
      • Cali, Colombia, 760036
        • Centro de Investigaciones Clínicas SAS
  • Mexico
      • Mexico City, Mexico, 54769
        • Phylasis Clinicas Research
      • Veracruz, Mexico, 91900
        • FAICIC Clinical Researc
    • Coahuila
      • Saltillo, Coahuila, Mexico, 25020
        • Clinical Research Institute Saltillo S.A. de C.V.
    • Nuevo Leon
      • Monterrey, Nuevo Leon, Mexico, 64460
        • Hospital Universitario "Dr. Jose Eleuterio Gonzalez"
    • Querétaro
      • Querétaro City, Querétaro, Mexico, 76090
        • SMIQ BRCR Global México
    • Tlalnepantla De Baz
      • San Lucas Tepetlacalco, Tlalnepantla De Baz, Mexico, 54055
        • Clinical Research Institute S.C.
    • Veracruz
      • Boca Del Río, Veracruz, Mexico, 94290
        • Centro Especializado en Investigación Clínica S.C.
  • Peru
      • Arequipa, Peru, 04001
        • Hospital Nivel IV Carlos Alberto Seguin Escobedo
      • Callao, Peru, 07016
        • Hospital Nacional IV Alberto Sabogal Sologuren
      • Lima, Peru, 15102
        • Hospital Nacional Cayetano Heredia
      • Lima, Peru, 15033
        • Hospital Almenara
      • Lima, Peru, 15074
        • Instituto Neuro Cardiovascular De Las Americas
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85028
        • Neuromuscular Research Center
      • Scottsdale, Arizona, United States, 85259
        • Mayo Clinic - Arizona
    • California
      • La Jolla, California, United States, 92037
        • University of California San Diego
      • Loma Linda, California, United States, 92354
        • Loma Linda University Health System, Department of Neurology
      • Los Angeles, California, United States, 90095
        • University of California Los Angeles
      • Los Angeles, California, United States, 90033
        • Keck School of Medicine, University of Southern California
      • Orange, California, United States, 92868
        • University of California Irvine Medical Center
      • San Francisco, California, United States, 94143
        • University of California San Francisco
      • San Francisco, California, United States, 94115
        • California Pacific Medical Center
    • Colorado
      • Colorado Springs, Colorado, United States, 80907
        • Colorado Springs Neurological Associates
    • District of Columbia
      • Washington, District of Columbia, United States, 20037
        • George Washington University
      • Washington, District of Columbia, United States, 20007
        • Georgetown University
    • Florida
      • Gainesville, Florida, United States, 32611
        • University of Florida - McKnight Brain Institute
      • Tampa, Florida, United States, 33612
        • University of South Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University
      • Augusta, Georgia, United States, 30912
        • Augusta University
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Indiana University-Neuroscience Center of Excellence/Goodman Hall
    • Kansas
      • Kansas City, Kansas, United States, 66160
        • University of Kansas Medical Center
    • Kentucky
      • Lexington, Kentucky, United States, 40536
        • University of Kentucky Chandler Medical Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • John Hopkins Outpatient Center
    • Massachusetts
      • Worcester, Massachusetts, United States, 01655
        • University of Massachusetts Medical School
    • Michigan
      • Grand Rapids, Michigan, United States, 49503
        • Mercy Health- Saint Mary's
    • Nebraska
      • Lincoln, Nebraska, United States, 68510
        • Neurology Associates
      • Omaha, Nebraska, United States, 68198
        • University of Nebraska Medical Center - Physicians Clinical Neurosciences Center
    • Nevada
      • Las Vegas, Nevada, United States, 89145
        • Las Vegas Clinic
    • New Jersey
      • Neptune, New Jersey, United States, 07753
        • Jersey Shore University Medical Center
    • New York
      • New York, New York, United States, 10032
        • Columbia Presbyterian Hospital
    • Oregon
      • Portland, Oregon, United States, 97213
        • Providence ALS Center
    • Pennsylvania
      • Hershey, Pennsylvania, United States, 17033
        • Penn State Health Milton S. Hershey Medical Center
      • Philadelphia, Pennsylvania, United States, 19140
        • Temple University School of Medicine
      • Pittsburgh, Pennsylvania, United States, 15212
        • Allegheny General Hospital
    • Tennessee
      • Cordova, Tennessee, United States, 38018
        • Wesley Neurology Clinic
    • Texas
      • Austin, Texas, United States, 78756
        • Austin Neuromuscular Center
      • Dallas, Texas, United States, 75214
        • Texas Neurology, P.A.
      • Houston, Texas, United States, 77030
        • The Methodist Hospital
    • Vermont
      • Colchester, Vermont, United States, 05401
        • University of Vermont Medical Center
    • Virginia
      • Richmond, Virginia, United States, 23298
        • VCU Medical Center
    • Washington
      • Seattle, Washington, United States, 98122
        • Swedish Neuroscience Institute
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Medical College of Wisconsin/Froedtert Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years to 75 years (ADULT, OLDER_ADULT)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: RANDOMIZED
  • Interventional Model: PARALLEL
  • Masking: DOUBLE

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
EXPERIMENTAL: Arm A: Treatment Period Acthar

Participants receive one 0.2 mL subcutaneous (SC) injection (shot under the skin) of the study drug (Acthar), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of tapering off the drug, ending their participation by Week 39.
Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection
PLACEBO_COMPARATOR: Arm B: Treatment Period Placebo

Participants receive one 0.2 mL SC injection that looks like Acthar, but has no drug in it (Matching Placebo), daily for up to 36 weeks.

Those who do not continue into the extension period will have 3 weeks of simulated tapering, ending their participation by Week 39.
Drug: Placebo
Matching placebo for subcutaneous injection
Other Names:
  • Reference Product
  • Matching Placebo
EXPERIMENTAL: Arm C: Extension Period Acthar-Acthar
Participants who receive Acthar during the treatment period and continue into the extension period do not go through the treatment-period tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection
EXPERIMENTAL: Arm D: Extension Period Placebo-Acthar
Participants who receive Placebo during the treatment period and continue into the extension period do not go through the treatment-period simulated tapering, but receive one 0.2 mL SC injection of Acthar, daily for up to 48 weeks, followed by 3 weeks of tapering off the drug, ending their participation within about 21 months.
Drug: Acthar
Repository corticotropin for subcutaneous injection
Other Names:
  • Trade name: Acthar® Gel
  • Generic name: repository corticotropin injection
Drug: Placebo
Matching placebo for subcutaneous injection
Other Names:
  • Reference Product
  • Matching Placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment Period: Scores on a Scale for Telephone-administered Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R)
Time Frame: Baseline, Week 36

The ALSFRS-R is a validated questionnaire-based scale used extensively as a primary outcome measure in ALS clinical trials and is considered a predictor of survival.

ALSFRS-R is a 12-item scale that measures 4 domains relevant for ALS (gross motor, fine motor, bulbar and respiratory)

A trained, independent rater calls each participant (or the caregiver) to administer the questionnaire. The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.
Baseline, Week 36
Number of Participants Experiencing an Adverse Event During the Treatment Period
Time Frame: by the end of the treatment period (within 36 Weeks)
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study Clinically significant changes in safety measures are recorded as adverse events.
by the end of the treatment period (within 36 Weeks)
Number of Participants Experiencing an Adverse Event by the End of the Trial in the OLE Period
Time Frame: by the time of database lock (within 84 weeks)
Serious adverse events, non-serious treatment-emergent adverse events, and all-cause mortality are collected until the participant no longer participates in the study (estimated about 1 year for participants leaving after the treatment period, and two years for participants who participate also in the open label extension). Clinically significant changes in safety measures are recorded as adverse events.
by the time of database lock (within 84 weeks)

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Treatment Period: Spirometry (%)
Time Frame: Baseline, Week 36
Spirometry (meaning the measuring of breath) is the most common of the lung function tests. It measures how much air can be inhaled [Forced Vital Capacity (FVC)] and exhaled [(Forced Expiratory Volume in one second (FEV1)].
Baseline, Week 36
Treatment Period: Scores on a Scale for Investigator-administered ALSFRS-R
Time Frame: Baseline, Week 36

The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible (summed) score of 48. Higher scores represent better function.
Baseline, Week 36
Extension Period: Scores on a Scale for Investigator-administered ALSFRS-R
Time Frame: Baseline, Week 84

The ALSFRS-R is a 12-item scale evaluating 4 domains relevant to ALS (gross motor, fine motor, bulbar and respiratory).

The trained investigator (or designee) administers the ALSFRS-R questionnaire in person with the participant (or caregiver). The 12 functions are rated on a scale from 0 to 4, with a highest possible score of 48. Higher scores represent better function.
Baseline, Week 84

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Mallinckrodt

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (ACTUAL)

June 22, 2017

Primary Completion (ACTUAL)

November 25, 2019

Study Completion (ACTUAL)

November 25, 2019

Study Registration Dates

First Submitted

February 23, 2017

First Submitted That Met QC Criteria

March 1, 2017

First Posted (ACTUAL)

March 3, 2017

Study Record Updates

Last Update Posted (ACTUAL)

October 14, 2020

Last Update Submitted That Met QC Criteria

September 18, 2020

Last Verified

September 1, 2020

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • MNK14042068

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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