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

Primary Purpose

Amyotrophic Lateral Sclerosis

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Repository corticotropin injection
Sponsored by
Mallinckrodt
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Amyotrophic Lateral Sclerosis focused on measuring H.P. Acthar Gel, Acthar, amyotrophic lateral sclerosis, ALS

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site
  • Questcor Investigational Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Experimental

Arm Label

Acthar 80 U (1.0 mL) SC twice weekly

Acthar 24 U (0.3 mL) SC daily

Acthar 56 U (0.7 mL) SC twice weekly

Acthar 16 U (0.2 mL) SC daily

Arm Description

Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly

Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily

Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly

Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily

Outcomes

Primary Outcome Measures

Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication

Secondary Outcome Measures

Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
Proportion of Subjects With Treatment Emergent Suicidality

Full Information

First Posted
July 15, 2013
Last Updated
November 8, 2016
Sponsor
Mallinckrodt
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1. Study Identification

Unique Protocol Identification Number
NCT01906658
Brief Title
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
Official Title
A Study to Explore the Safety and Tolerability of Acthar in Patients With Amyotrophic Lateral Sclerosis
Study Type
Interventional

2. Study Status

Record Verification Date
November 2016
Overall Recruitment Status
Completed
Study Start Date
July 2013 (undefined)
Primary Completion Date
November 2014 (Actual)
Study Completion Date
December 2014 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Mallinckrodt

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Amyotrophic Lateral Sclerosis
Keywords
H.P. Acthar Gel, Acthar, amyotrophic lateral sclerosis, ALS

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
43 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Acthar 80 U (1.0 mL) SC twice weekly
Arm Type
Experimental
Arm Description
Acthar (Repository Corticotropin Injection) 80 U (1.0 mL) SC twice weekly
Arm Title
Acthar 24 U (0.3 mL) SC daily
Arm Type
Experimental
Arm Description
Acthar (Repository Corticotropin Injection) 24 U (0.3 mL) SC daily
Arm Title
Acthar 56 U (0.7 mL) SC twice weekly
Arm Type
Experimental
Arm Description
Acthar (Repository Corticotropin Injection) 56 U (0.7 mL) SC twice weekly
Arm Title
Acthar 16 U (0.2 mL) SC daily
Arm Type
Experimental
Arm Description
Acthar (Repository Corticotropin Injection) 16 U (0.2 mL) SC daily
Intervention Type
Drug
Intervention Name(s)
Repository corticotropin injection
Other Intervention Name(s)
H.P. Acthar Gel, Acthar, ACTH Gel, ACTH
Intervention Description
Acthar given SC twice weekly (80 U or 56 U) or daily (24 U or 16 U) for 8 weeks
Primary Outcome Measure Information:
Title
Proportion of Subjects With Adverse Events (AEs) That Required Study Drug Discontinuation or Could Not be Controlled With Concomitant Medication
Time Frame
Baseline to Week 8
Secondary Outcome Measure Information:
Title
Proportion of Subjects With Adverse Events That Required Study Drug Discontinuation
Time Frame
Baseline to Week 8
Title
Proportion of Subjects With Adverse Events That Could Not be Controlled by Concomitant Medication
Time Frame
Baseline to Week 8
Title
Proportion of Subjects With Treatment Emergent Suicidality
Time Frame
Baseline to Week 36

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility 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: History of prior sensitivity to Acthar or other porcine protein products. 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. 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. 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. For the purposes of this study, history of peptic ulcer is defined as ≤ 6 months prior to screening. 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. For the purposes of this study, congestive heart failure is defined as New York Heart Association Functional Class III-IV.
Facility Information:
Facility Name
Questcor Investigational Site
City
Birmingham
State/Province
Alabama
ZIP/Postal Code
35233
Country
United States
Facility Name
Questcor Investigational Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85018
Country
United States
Facility Name
Questcor Investigational Site
City
San Francisco
State/Province
California
ZIP/Postal Code
94115
Country
United States
Facility Name
Questcor Investigational Site
City
Stanford
State/Province
California
ZIP/Postal Code
94305
Country
United States
Facility Name
Questcor Investigational Site
City
Jacksonville
State/Province
Florida
ZIP/Postal Code
32224
Country
United States
Facility Name
Questcor Investigational Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Questcor Investigational Site
City
Tampa
State/Province
Florida
ZIP/Postal Code
33612
Country
United States
Facility Name
Questcor Investigational Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Questcor Investigational Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Questcor Investigational Site
City
Rochester
State/Province
Minnesota
ZIP/Postal Code
55905
Country
United States
Facility Name
Questcor Investigational Site
City
Lincoln
State/Province
Nebraska
ZIP/Postal Code
68506
Country
United States
Facility Name
Questcor Investigational Site
City
Hershey
State/Province
Pennsylvania
ZIP/Postal Code
17033
Country
United States
Facility Name
Questcor Investigational Site
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15212
Country
United States
Facility Name
Questcor Investigational Site
City
Memphis
State/Province
Tennessee
ZIP/Postal Code
38104
Country
United States
Facility Name
Questcor Investigational Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75214
Country
United States
Facility Name
Questcor Investigational Site
City
Houston
State/Province
Texas
ZIP/Postal Code
77030
Country
United States
Facility Name
Questcor Investigational Site
City
San Antonio
State/Province
Texas
ZIP/Postal Code
78229
Country
United States

12. IPD Sharing Statement

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

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