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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 not yet open for participant recruitment. (see Contacts and Locations)
Verified February 2017 by Mallinckrodt
Sponsor:
Information provided by (Responsible Party):
Mallinckrodt
ClinicalTrials.gov Identifier:
NCT03068754
First received: February 23, 2017
Last updated: March 1, 2017
Last verified: February 2017
  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: 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

  • Pulmonary function test C: Mean slope of negative inspiratory force test [ Time Frame: 36 weeks ]
    Mean slope of negative inspiratory force 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)


Estimated Enrollment: 195
Anticipated Study Start Date: May 2017
Estimated Study Completion Date: December 2019
Estimated Primary Completion Date: November 2019 (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 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 [U]) daily (QD) or SC matching placebo 0.2 mL QD for 36 weeks, followed by a 3 week taper.

  Eligibility

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. On riluzole 50 mg twice a day for ≥ 4 weeks prior to the Screening Visit.
  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. Requires nocturnal continuous positive airway pressure or bilevel positive airway pressure.
  4. Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type at Screening Visit.
  5. Known contraindication(s) to Acthar.
  6. History of chronic active hepatitis including active or chronic hepatitis B, or acute or chronic hepatitis C.
  7. History of tuberculosis (TB) infection, any signs/symptoms of TB, or any close contact with an individual with an active TB infection.
  8. Clinically significant infection requiring intravenous administration of antibiotics and hospitalization in the 4 weeks prior to the Screening Visit.
  Contacts and Locations
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, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT03068754

Contacts
Contact: Roman Bilyk 908-238-6755 roman.bilyk@mallinckrodt.com

Sponsors and Collaborators
Mallinckrodt
  More Information

Responsible Party: Mallinckrodt
ClinicalTrials.gov Identifier: NCT03068754     History of Changes
Other Study ID Numbers: MNK14042068
Study First Received: February 23, 2017
Last Updated: March 1, 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

ClinicalTrials.gov processed this record on April 25, 2017