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

Aaron Miller

Status

Unknown

Conditions

Multiple Sclerosis

Treatments

Drug: Acthar Gel

Study type

Interventional

Funder types

Other

Industry

Identifiers

NCT01900093

GCO 13-0600

Details and patient eligibility

About

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.

Full 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.

Enrollment

10 estimated patients

Sex

All

Ages

18 to 65 years old

Volunteers

No 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