Back to results

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

Primary Purpose

Multiple Sclerosis

Status

Unknown status

Phase

Not Applicable

Locations

United States

Study Type

Interventional

Intervention

Acthar Gel

About this trial

This is an interventional treatment trial for Multiple Sclerosis

Eligibility Criteria

18 Years - 65 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

Icahn School of Medicine at Mount SinaiRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Acthar Gel

Arm Description

80 units of subcutaneous Acthar Gel therapy daily

Outcomes

Primary Outcome Measures

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

Secondary Outcome Measures

Improvement in Expanded Disability Status Scale

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.

Full Information

NCT ID

NCT01900093

First Posted

July 11, 2013

Last Updated

September 1, 2017

Sponsor

Aaron Miller

Collaborators

Mallinckrodt

1. Study Identification

Unique Protocol Identification Number

NCT01900093

Brief Title

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

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 Type

Interventional

2. Study Status

Record Verification Date

September 2017

Overall Recruitment Status

Unknown status

Study Start Date

July 2013 (undefined)

Primary Completion Date

March 2018 (Anticipated)

Study Completion Date

March 2018 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor-Investigator

Name of the Sponsor

Aaron Miller

Collaborators

Mallinckrodt

4. Oversight

Data Monitoring Committee

5. Study Description

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.

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Multiple Sclerosis

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

10 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Acthar Gel

Arm Type

Experimental

Arm Description

80 units of subcutaneous Acthar Gel therapy daily

Intervention Type

Drug

Intervention Name(s)

Acthar Gel

Other Intervention Name(s)

Repository corticotrophin injection

Intervention Description

80 units of subcutaneous Acthar Gel therapy daily

Primary Outcome Measure Information:

Title

Improvement in Functional System Score appropriate to the Targeted Neurological Deficit

Description

Time Frame

at one week

Secondary Outcome Measure Information:

Title

Improvement in Expanded Disability Status Scale

Description

Time Frame

baseline and at 1 week

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Maximum Age & Unit of Time

65 Years

Accepts Healthy Volunteers

Eligibility 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

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Tarah Herrmann

Phone

212-241-4264

tarah.gustafson@mssm.edu

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Aaron Miller, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Fred Lublin, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Stephen Krieger, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Michelle Fabian, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Ilana Katz-Sand, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Sylvia Klineova, MD

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Gretchen Mathewson, NP

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

First Name & Middle Initial & Last Name & Degree

Aliza Ben-Zacharia, NP

Organizational Affiliation

Icahn School of Medicine at Mount Sinai

Official's Role

Principal Investigator

Facility Information:

Facility Name

Icahn School of Medicine at Mount Sinai

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Aaron Miller, MD

12. IPD Sharing Statement

Learn more about this trial

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

We'll reach out to this number within 24 hrs