Pilot Study of Acthar® Gel in Chronic Inflammatory Demyelinating Neuropathy

The purpose of this pilot study is to assess the safety and efficacy of Acthar® Gel in Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) patients.

Study Overview

• Status: Withdrawn
• Conditions: Chronic Inflammatory Demyelinating Polyneuropathy
• Intervention / Treatment: Drug: H.P. Acthar® Gel

• Study Type: Interventional
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Arizona
    • Phoenix, Arizona, United States, 85018
      • Phoenix Neurological Associates
  • California
    • Los Angeles, California, United States, 90033
      • University of Southern California
  • Kansas
    • Kansas City, Kansas, United States, 66160
      • University of Kansas Medical Center
  • Tennessee
    • Memphis, Tennessee, United States, 38163
      • Wesley Neurology Group
  • Vermont
    • Burlington, Vermont, United States, 05405
      • University of Vermont

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• CIDP diagnosed according to the European Federation of Neurological Societies/Peripheral Nerve Society (EFNS/PNS) criteria 2010
• Age > 18 years
• Able to give written informed consent
• Patient's signs and symptoms should not be better explained by another disease process
• Patients can be on prednisone as long as there has been no dose change for 4 weeks from baseline
• Patients can be on following drugs as long as there has been no change for 60 days from baseline visit including azathioprine, cyclosporine, cyclophosphamide, mycophenolate mofetil, intravenous immunoglobulin (IVIg) or other immunosuppressive drugs
• INCAT score greater than or equal to 2

Exclusion Criteria:

• Presence of any other causes of polyneuropathy or multifocal motor neuropathy, which in the opinion of the investigator is the major contributor to the numbness and weakness.
• Other neurologic or orthopedic condition causing weakness
• Treatment with plasma exchange (PLEX) within the last 30 days from baseline
• Participation in another trial within the last 30 days from baseline or two ½ life of the drug being studied.
• Latent tuberculosis or active infection
• Contraindication per Acthar® Gel prescribing information scleroderma, osteoporosis, systemic fungal infections, ocular herpes simplex, recent surgery, history or presence of peptic ulcer, congestive heart failure, uncontrolled hypertension, primary adrenocortical insufficiency, or adrenal cortical hyperfunction.
• History of prior sensitivity to Acthar® Gel or other porcine products
• Previous or present Infection with hepatitis C and hepatitis B
• Pregnancy or nursing mothers.
• Female subjects of childbearing potential either not using, or not willing to use, a medically reliable method of contraception for the entire duration of the study, or not sexually abstinent for the entire duration of the study, or not surgically sterile.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Acthar® Gel; For the first two weeks participants will receive 1 mL of study drug subcutaneously every other day. After that, participants will received 1 mL of study drug twice a week for up to 6 months.	Drug: H.P. Acthar® Gel; Other Names: repository corticotropin injection; Acthar® Gel

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Related Adverse Events	Count of adverse events experienced by study participants.	28 Weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Efficacy of study drug	Measured using the Inflammatory Neuropathy Cause and Treatment (INCAT) score. The INCAT scale has upper and lower extremity components (maximum of 5 points for upper (arm disability) and maximum of 5 points for lower (leg disability) that add up to a maximum of 10-points. A score of 0 indicates no problems. A score of 10 indicates person is severely incapacitated.	Change from Baseline to 28 Weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Maximum Grip Strength	Measured by the amount of force a person uses to squeeze around a dynamometer.	28 weeks
Manual Muscle Testing (MMT)	Measure based on the Medical Research Council (MRC) sum score (strength score combined for 12 specified muscle groups) on MMT.	28 Weeks
Rasch-built Overall Disability Scale (R-ODS)	The R-ODS is a questionnaire that rates a person's ability to perform 24 daily activities such as eating, using the toilet, taking a shower, dressing, walking, dancing, running, or standing. Scores range from 0-48 with 0 meaning they cannot perform any of the tasks and 48 meaning they can perform all of the tasks without any difficulty.	28 Weeks

Collaborators and Investigators

• Sponsor: Mamatha Pasnoor, MD
• Collaborators: Mallinckrodt
• Investigators: Principal Investigator: Mamatha Pasnoor, MD, University of Kansas Medical Center

Study record dates

Study Major Dates

• Study Start: August 1, 2015
• Primary Completion (Anticipated): July 1, 2018
• Study Completion (Anticipated): July 1, 2018

Study Registration Dates

• First Submitted: August 30, 2015
• First Submitted That Met QC Criteria: October 12, 2015
• First Posted (Estimate): October 14, 2015

Study Record Updates

• Last Update Posted (Estimate): July 18, 2016
• Last Update Submitted That Met QC Criteria: July 15, 2016
• Last Verified: July 1, 2016

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Nervous System Diseases; Immune System Diseases; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Neuromuscular Diseases; Peripheral Nervous System Diseases; Polyradiculoneuropathy; Polyneuropathies; Polyradiculoneuropathy, Chronic Inflammatory Demyelinating; Physiological Effects of Drugs; Hormones; Hormones, Hormone Substitutes, and Hormone Antagonists; Adrenocorticotropic Hormone
• Other Study ID Numbers: STUDY00002770