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Trial record 1 of 1 for:    NCT01900093
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Evaluation of the Response to Acthar Gel Therapy in Patients Who Failed Intravenous Methylprednisolone (IVMP) for MS Relapses

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ClinicalTrials.gov Identifier: NCT01900093
Recruitment Status : Recruiting
First Posted : July 16, 2013
Last Update Posted : September 6, 2017
Sponsor:
Collaborator:
Mallinckrodt
Information provided by (Responsible Party):
Aaron Miller, Icahn School of Medicine at Mount Sinai

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.

Condition or disease Intervention/treatment Phase
Multiple Sclerosis Drug: Acthar Gel Not Applicable

Detailed Description:
This is an open-label, small, proof-of-concept study examining the safety, tolerability, and extent of recovery of a two-week course of subcutaneous Acthar Gel therapy in patients with MS relapse who have failed to make a satisfactory recovery after treatment with high dose methylprednisolone. Eligible patients will be given 80 units of Acthar for 14 days. Patients will be evaluated at baseline, at 1 week of Acthar treatment, at completion of Acthar treatment, and 1 week after completion of treatment. For those who do not undergo plasmapheresis an additional evaluation will be conducted 2 weeks after completion of treatment. Monitoring will include blood pressure determination and blood sugar determination.

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Prospective Proof-Of-Concept Trial to Evaluate the Response to Acthar Gel Therapy in Patients With Multiple Sclerosis (MS) Relapses Who Have Failed To Make an Adequate Recovery After Treatment With High-Dose Intravenous Methylprednisolone
Study Start Date : July 2013
Estimated Primary Completion Date : March 2018
Estimated Study Completion Date : March 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Acthar Gel
80 units of subcutaneous Acthar Gel therapy daily
Drug: Acthar Gel
80 units of subcutaneous Acthar Gel therapy daily
Other Name: Repository corticotrophin injection




Primary Outcome Measures :
  1. Improvement in Functional System Score appropriate to the Targeted Neurological Deficit [ Time Frame: at one week ]
    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


Secondary Outcome Measures :
  1. Improvement in Expanded Disability Status Scale [ Time Frame: baseline and at 1 week ]
    Secondary outcome measures will be improvement in TND at completion of ACTHAR Gel therapy, improvement by at least 1 step on EDSS at completion of treatment and at 1 week following completion of treatment.



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Ages Eligible for Study:   18 Years to 65 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • 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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01900093


Contacts
Contact: Tarah Herrmann 212-241-4264 tarah.gustafson@mssm.edu

Locations
United States, New York
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Principal Investigator: Aaron Miller, MD         
Sponsors and Collaborators
Aaron Miller
Mallinckrodt
Investigators
Principal Investigator: 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

Responsible Party: Aaron Miller, Professor, Icahn School of Medicine at Mount Sinai
ClinicalTrials.gov Identifier: NCT01900093     History of Changes
Other Study ID Numbers: GCO 13-0600
First Posted: July 16, 2013    Key Record Dates
Last Update Posted: September 6, 2017
Last Verified: September 2017

Additional relevant MeSH terms:
Sclerosis
Multiple Sclerosis
Pathologic Processes
Demyelinating Autoimmune Diseases, CNS
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Demyelinating Diseases
Autoimmune Diseases
Immune System Diseases
Prednisolone acetate
Methylprednisolone acetate
Methylprednisolone
Methylprednisolone Hemisuccinate
Prednisolone
Prednisolone hemisuccinate
Prednisolone phosphate
Adrenocorticotropic Hormone
Anti-Inflammatory Agents
Antiemetics
Autonomic Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Gastrointestinal Agents
Glucocorticoids
Hormones
Hormones, Hormone Substitutes, and Hormone Antagonists
Neuroprotective Agents
Protective Agents
Antineoplastic Agents, Hormonal
Antineoplastic Agents