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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

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ClinicalTrials.gov Identifier: NCT03068754
Recruitment Status : Recruiting
First Posted : March 3, 2017
Last Update Posted : May 1, 2018
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt

Brief 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).

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis Drug: Acthar Drug: Placebo Phase 2

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.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 213 participants
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
Actual Study Start Date : June 14, 2017
Estimated Primary Completion Date : December 2019
Estimated Study Completion Date : December 2019


Arm Intervention/treatment
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



Primary Outcome Measures :
  1. 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 :
  1. 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

  2. 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

  3. 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

  4. 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

  5. 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

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

  7. 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)

  8. 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

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

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

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



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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.

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: Anne Marie Viola, BS, CCRA 800-556-3314 clinicaltrials@mnk.com

  Show 38 Study Locations
Sponsors and Collaborators
Mallinckrodt
Investigators
Study Director: Susan VanMeter Mallinckrodt

Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03068754     History of Changes
Other Study ID Numbers: MNK14042068
First Posted: March 3, 2017    Key Record Dates
Last Update Posted: May 1, 2018
Last Verified: April 2018

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