Evaluation of the Response to Acthar Gel Therapy in Patients Who Failed Intravenous Methylprednisolone (IVMP) for MS Relapses

NCT01900093 | Unknown

• 1 other identifier: GCO 13-0600
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

Although ACTHAR gel is approved for the treatment of acute relapses, it is currently not widely used. Thus, we propose to conduct a small open-label proof-of-concept trial to evaluate the response to ACTHAR gel therapy in patients who have failed to make a satisfactory recovery after treatment with high dose IVMP. Documentation of the clinical course as well as any adverse events related to IVMP use will be made. The investigators propose to study the potential benefit of a 14-day course (the dose historically used since the landmark clinical trial of Rose et al1) of subcutaneous ACTHAR gel in 10 patients who have demonstrated inadequate improvement after a course of IVMP, 1000 mg daily, for 5 treatments (over a maximum of 8 days). These would be patients for whom PLEX would be considered as a treatment possibility. The primary outcome measure will be improvement in the targeted neurological deficit, as measured on the appropriate functional system score (FSS) of the EDSS.

Conditions

Multiple Sclerosis

Outcome Measures

Primary Outcomes (1)

• Improvement in Functional System Score appropriate to the Targeted Neurological Deficit

  Primary outcome measure will be the percentage of patients improving by at least 1 step on the FSS appropriate to their neurological deficit (targeted neurological deficit \[TND\]} at 1 week following completion of treatment

  at one week

Secondary Outcomes (1)

• Improvement in Expanded Disability Status Scale

  baseline and at 1 week

Study Arms (1)

Acthar Gel

EXPERIMENTAL

80 units of subcutaneous Acthar Gel therapy daily

Drug: Acthar Gel

Interventions

Acthar Gel DRUG

80 units of subcutaneous Acthar Gel therapy daily

Also known as: Repository corticotrophin injection

Acthar Gel

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Ages 18-65
• EDSS of 2.0 - 7.5 (inclusive)
• Sustained a significant MS exacerbation affecting vision, brainstem, motor, or cerebellar function AND were initially treated with a 5 day course of IVMP within 10 days of the onset of symptoms.
• Failed to make adequate improvement and must initiate Acthar Gel therapy within two weeks (+/- 72 hours) after the first day of 5 treatments with 1000 mg IVMP, as judged by their treating neurologist.
• Must be able to comply with the requirements of the protocol as determined by the investigator.
• Ability to understand the purpose and risks of the study and provide signed and date informed consent and authorization to use protected health information (PHI) in accordance with national and local subject privacy regulations.

You may not qualify if:

• Patients whose relapse consists of pure sensory or bowel/bladder symptoms
• History of ACTHAR gel use or other forms of ACTH with failure to improve or with occurrence of significant adverse experiences.
• Diagnosis of scleroderma, systemic fungal infections, ocular herpes simplex, congestive heart failure, and/or uncontrolled hypertension, and/or any clinically relevant medical disease that would put the patient at risk by participating in the study
• Persistent significant or severe infection
• Recent history of drug or alcohol abuse
• Concomitant use or prior use in the preceding 6 months of any investigational drug.
• Pregnant or nursing
• Recent surgery (up to the investigator's discretion what constitutes recent)
• History of, or the presence of, a peptic ulcer
• Known sensitivity to proteins of porcine origin
• Received a live or live attenuated vaccine in the last 30 days before baseline

Sponsors & Collaborators

• Aaron Miller: lead
• Mallinckrodt: collaborator

Study Sites (1)

Icahn School of Medicine at Mount Sinai

New York, New York, 10029, United States

RECRUITING

MeSH Terms

Conditions

Multiple Sclerosis

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Aaron Miller, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Fred Lublin, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Stephen Krieger, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Michelle Fabian, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Ilana Katz-Sand, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Sylvia Klineova, MD

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Gretchen Mathewson, NP

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

• Aliza Ben-Zacharia, NP

  Icahn School of Medicine at Mount Sinai

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Tarah Herrmann

CONTACT

212-241-4264; tarah.gustafson@mssm.edu

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: July 11, 2013
• First Posted: July 16, 2013
• Study Start: July 1, 2013
• Primary Completion: March 1, 2018
• Study Completion: March 1, 2018
• Last Updated: September 6, 2017

Record last verified: 2017-09

Locations

US (1)