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A Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis

This study is currently recruiting participants.
Verified September 2017 by Mallinckrodt
Sponsor:
ClinicalTrials.gov Identifier:
NCT03068754
First Posted: March 3, 2017
Last Update Posted: September 25, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Information provided by (Responsible Party):
Mallinckrodt
  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).

Condition Intervention Phase
Amyotrophic Lateral Sclerosis Drug: Acthar Drug: Placebo Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
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

Resource links provided by NLM:


Further study details as provided by Mallinckrodt:

Primary Outcome Measures:
  • Telephone administered Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) [ Time Frame: 36 weeks ]
    Change from baseline in telephone administered ALSFRS-R


Secondary Outcome Measures:
  • Telephone administered ALSFRS-R total score decline [ Time Frame: 36 weeks ]
    Mean slope of the telephone administered ALSFRS-R total score decline from baseline in the group treated with Acthar vs the placebo group

  • Investigator administered ALSFRS-R total score decline [ Time Frame: 36 weeks ]
    Mean slope of investigator administered ALSFRS-R total score decline from baseline in the group treated with Acthar vs the placebo group

  • Telephone administered ALSFRS-R total score and investigator administered ALSFRS-R total score [ Time Frame: 36 weeks ]
    Change from baseline in telephone administered ALSFRS-R total score and investigator administered ALSFRS-R total score over time

  • Pulmonary function test A: Mean slope of percent predicted forced vital capacity test [ Time Frame: 36 weeks ]
    Mean slope of percent predicted forced vital capacity test decline from baseline in the group treated with Acthar vs the placebo group

  • Pulmonary function test B: Mean slope of volume expired in 1 second test [ Time Frame: 36 weeks ]
    Mean slope of volume expired in 1 second test decline from baseline in the group treated with Acthar vs the placebo group

  • Columbia-Suicide Severity Rating Scale (C-SSRS) [ Time Frame: 36 weeks ]
    Summary of Columbia-Suicide Severity Rating Scale (C-SSRS)

  • Telephone administered ALSFRS-R total score [ Time Frame: 84 Weeks ]
    Change from baseline in telephone administered ALSFRS-R over time in the group treated with Acthar followed by Acthar (Acthar-Acthar) and the group treated with placebo followed by Acthar (placebo-Acthar)

  • Telephone administered ALSFRS-R total score [ Time Frame: 84 Weeks ]
    Mean slope of telephone administered ALSFRS-R total score decline from baseline in Acthar-Acthar and placebo-Acthar groups

  • Pulmonary function test A [ Time Frame: 84 Weeks ]
    Mean slope of forced vital capacity decline in Acthar-Acthar and placebo-Acthar groups

  • Pulmonary function text B [ Time Frame: 84 Weeks ]
    Mean slope of volume expired in 1 second test in Acthar-Acthar and placebo-Acthar groups

  • Survival [ Time Frame: 84 Weeks ]
    Survival in Acthar-Acthar and placebo-Acthar groups


Estimated Enrollment: 213
Actual Study Start Date: June 14, 2017
Estimated Study Completion Date: March 2020
Estimated Primary Completion Date: March 2020 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: Acthar Drug: Acthar
Repository corticotropin injection administered subcutaneously once a day
Placebo Comparator: Placebo Drug: Placebo
Placebo gel injection administered subcutaneously once a day

Detailed Description:

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.

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 75 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  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.

Exclusion Criteria:

  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.
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03068754


Contacts
Contact: Michael Zhang 800-556-3314 clinicaltrials@mallinckrodt.com

Locations
United States, Arizona
Neuromuscular Research Center Recruiting
Phoenix, Arizona, United States, 85208
United States, California
Loma Linda University Health System, Department of Neurology Recruiting
Loma Linda, California, United States, 92354
United States, Colorado
Colorado Springs Neurological Associates Recruiting
Colorado Springs, Colorado, United States, 80907
United States, District of Columbia
GW Medical Faculty Associates Recruiting
Washington, D.C., District of Columbia, United States, 20037
United States, Florida
University of South Florida Recruiting
Tampa, Florida, United States, 33609
United States, Indiana
Indiana University-Neuroscience Center of Excellence/Goodman Hall Recruiting
Indianapolis, Indiana, United States, 46202
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Maryland
John Hopkins Outpatient Center Recruiting
Baltimore, Maryland, United States, 21287
United States, Tennessee
Wesley Neurology Clinic Recruiting
Cordova, Tennessee, United States, 38018
United States, Texas
Austin Neuromuscular Center Recruiting
Austin, Texas, United States, 78756
Texas Neurology, P.A. Recruiting
Dallas, Texas, United States, 75215
United States, Wisconsin
Medical College of Wisconsin/Froedtert Hospital Recruiting
Milwaukee, Wisconsin, United States, 53226
Sponsors and Collaborators
Mallinckrodt
Investigators
Study Director: Susan VanMeter Mallinckrodt
  More Information

Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03068754     History of Changes
Other Study ID Numbers: MNK14042068
First Submitted: February 23, 2017
First Posted: March 3, 2017
Last Update Posted: September 25, 2017
Last Verified: September 2017

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Mallinckrodt:
ALS

Additional relevant MeSH terms:
Sclerosis
Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases
Adrenocorticotropic Hormone
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Physiological Effects of Drugs