A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Questcor Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT01906658
First received: July 15, 2013
Last updated: April 4, 2014
Last verified: April 2014
  Purpose

This 8-week randomized, open-label evaluation will examine the acute safety and tolerability of 4 different dosing regimens of Acthar to inform dose selection for future studies of Acthar in patients with Amyotrophic Lateral Sclerosis (ALS). The study will also investigate the mean rate of change in the ALSFRS-R total score as an exploratory endpoint to help design future studies.

This study will enroll up to 40 patients and include an optional 28-week open-label extension period plus a 3-week treatment taper and 1-week follow up period. After completion of Week 8, patients enrolled in a treatment group that is considered safe and tolerable at that time have the option to continue into the open-label extension period. A 3-week treatment taper and a follow-up visit are planned for all patients enrolled in the study, beginning either at Week 8 or at Week 36 if a patient continues into the optional open-label extension period.


Condition Intervention Phase
Amyotrophic Lateral Sclerosis
Drug: Repository corticotropin injection
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis

Resource links provided by NLM:


Further study details as provided by Questcor Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Proportion of patients with adverse events (AEs) that require study drug discontinuation or cannot be controlled with concomitant medication [ Time Frame: Week 8 ] [ Designated as safety issue: Yes ]

Secondary Outcome Measures:
  • Proportion of patients with clinically significant change in routine clinical laboratory testing from baseline [ Time Frame: Week 4, 8, 12, 36, and 40 ] [ Designated as safety issue: Yes ]
    Significant change in routine clinical laboratory testing from baseline as measured by complete blood count, hemoglobin A1c, chemistry (including serum glucose and lipid panel), serum cortisol, and routine urinalysis.

  • Proportion of patients with clinically significant change in vital signs from baseline [ Time Frame: Week 4, 8, 12, 36, and 40 ] [ Designated as safety issue: Yes ]
    Significant change in vital signs from baseline as measured by blood pressure, heart rate, and body temperature.

  • Proportion of patients with other adverse events (AEs) [ Time Frame: Week 4, 8, 12 ,36, and 40 ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 40
Study Start Date: July 2013
Estimated Study Completion Date: December 2014
Estimated Primary Completion Date: May 2014 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Acthar 80 U (1.0 mL) SC twice weekly
Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly
Drug: Repository corticotropin injection
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Other Names:
  • H.P. Acthar Gel
  • Acthar
  • ACTH Gel
  • ACTH
Experimental: Acthar 24 U (0.3 mL) SC daily
Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily
Drug: Repository corticotropin injection
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Other Names:
  • H.P. Acthar Gel
  • Acthar
  • ACTH Gel
  • ACTH
Experimental: Acthar 56 U (0.7 mL) SC twice weekly
Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly
Drug: Repository corticotropin injection
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Other Names:
  • H.P. Acthar Gel
  • Acthar
  • ACTH Gel
  • ACTH
Experimental: Acthar 16 U (0.2 mL) SC daily
Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily
Drug: Repository corticotropin injection
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Other Names:
  • H.P. Acthar Gel
  • Acthar
  • ACTH Gel
  • ACTH

  Eligibility

Ages Eligible for Study:   18 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Able to provide informed consent.
  • Diagnosis of clinically definite ALS, clinically probable-laboratory supported ALS, clinically probable ALS, or clinically possible ALS based on the revised El Escorial criteria.
  • Patients with ALS ≤ 3 years since symptom onset. Symptom onset is defined as date of first muscle weakness or dysarthria.
  • Upright slow vital capacity (SVC)≥ 60% of predicted.
  • If taking riluzole and/or Nuedexta®, stable regimen is required for ≥ 30 days prior to screening.
  • Medically (either independently or with caregiver assistance) able to comply with study procedures, including subcutaneous (SC) injections of study medication and adherence to concomitant medication restrictions.

Exclusion Criteria:

  • Any medical condition known to have an association with motor neuron dysfunction which might confound or obscure the diagnosis of ALS.
  • Tracheostomy, diaphragm pacing, or ongoing need for assisted ventilation of any type (e.g., bilevel positive airway pressure) for treatment of ALS-related respiratory dysfunction (vital capacity of < 60% predicted, nocturnal desaturation, and/or nocturnal hypoventilation). Patients on assisted ventilation for other reasons require approval from the Medical Monitor. (Supplemental oxygen is acceptable).
  • Recorded diagnosis or evidence of major psychiatric disorder.
  • Clinically evident cognitive and/or behavioral impairment that in the opinion of the Investigator would impair the ability of the patient to comply with the study procedures.
  • Therapies and/or Medications:

    1. History of prior sensitivity to Acthar or other porcine protein products.
    2. Chronic systemic corticosteroid use, defined as > 20 mg of prednisone or equivalent systemic corticosteroid taken for more than 4 consecutive weeks within 6 months prior to randomization. Topical, inhaled, or intra-articular corticosteroids are allowed.
    3. Planned treatment with live or live attenuated vaccines once enrolled in the study.
  • Participation in another therapeutic (drug or device) investigational study within 30 days prior to screening.
  • Type 1 or type 2 diabetes mellitus, or patients currently taking hypoglycemic medication.
  • Contraindication per Acthar Prescribing Information, Appendix D Section 4: scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history of or the presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction.

    1. For the purposes of this study, osteoporosis is defined as a history of a lumbar spine and/or femoral neck T-score ≤ -2.5 on bone densitometry (DXA), OR osteoporosis requiring pharmacologic therapy, OR a history of non-traumatic low impact hip or vertebral fracture, OR patient reported history of osteoporosis.
    2. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening.
    3. For the purposes of this study, uncontrolled hypertension is defined as mean systolic blood pressure ≥ 140 mmHg and diastolic blood pressure ≥ 90 mmHg on ≥ 3 seated readings taken at least 5 minutes apart during the screening period.
    4. For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
  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: NCT01906658

Locations
United States, Alabama
Questcor Investigational Site
Birmingham, Alabama, United States, 35233
United States, Arizona
Questcor Investigational Site
Phoenix, Arizona, United States, 85018
United States, California
Questcor Investigational Site
San Francisco, California, United States, 94115
Questcor Investigational Site
Stanford, California, United States, 94305
United States, Florida
Questcor Investigational Site
Jacksonville, Florida, United States, 32224
Questcor Investigational Site
Miami, Florida, United States, 33136
Questcor Investigational Site
Tampa, Florida, United States, 33612
United States, Georgia
Questcor Investigational Site
Atlanta, Georgia, United States, 30322
United States, Kansas
Questcor Investigational Site
Kansas City, Kansas, United States, 66160
United States, Minnesota
Questcor Investigational Site
Rochester, Minnesota, United States, 55905
United States, Nebraska
Questcor Investigational Site
Lincoln, Nebraska, United States, 68506
United States, Pennsylvania
Questcor Investigational Site
Hershey, Pennsylvania, United States, 17033
Questcor Investigational Site
Pittsburgh, Pennsylvania, United States, 15212
United States, Tennessee
Questcor Investigational Site
Memphis, Tennessee, United States, 38104
United States, Texas
Questcor Investigational Site
Dallas, Texas, United States, 75214
Questcor Investigational Site
Houston, Texas, United States, 77030
Questcor Investigational Site
San Antonio, Texas, United States, 78229
Sponsors and Collaborators
Questcor Pharmaceuticals, Inc.
  More Information

No publications provided

Responsible Party: Questcor Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT01906658     History of Changes
Other Study ID Numbers: QSC01-ALS-01
Study First Received: July 15, 2013
Last Updated: April 4, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Questcor Pharmaceuticals, Inc.:
H.P. Acthar Gel
Acthar
amyotrophic lateral sclerosis
ALS

Additional relevant MeSH terms:
Amyotrophic Lateral Sclerosis
Sclerosis
Motor Neuron Disease
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
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014