Safety and Efficacy of Cellcept and Avonex as Combination Treatment in Multiple Sclerosis

A Randomized, Open-label, Parallel-Group Multicenter Study to Determine the Safety/Efficacy of Mycophenolate Mofetil in Mono & Combination Therapy With Interferon Beta 1a in Patients With Relapsing Remitting Multiple Sclerosis

The primary objective of this safety/mechanistic study is to determine the safety and tolerability of oral Cellcept when compared with weekly intramuscular Avonex in relapsing multiple sclerosis. Safety will be assessed by virtue of changes in size and number of lesions on MRI scans.

Study Overview

• Status: Completed
• Conditions: Multiple Sclerosis
• Intervention / Treatment: Drug: Mycophenolate Mofetil (CellCept)

Detailed Description

Sixty patients (20 patients at each recruiting center) with RR MS who satisfy both inclusion and exclusion criteria will be treated with CellCept® or Avonex® for the first 6 months of the study. Those patients will have a fifty-fifty chance of receiving either Avonex or Cellcept. Baseline data will be collected before treatment begins including MRIs, chest x-ray, EKG, and standard labwork, along with a blood test for HIV and Hepatitis B. Once enrolled, study visits include periodic MRI scans, a neurological exam by the examining neurologist every three months, frequent bloodwork, questionnaires, and eye-testing at month zero, six, and twelve months. Eye testing takes about one hour and requires dilation of pupils. All assessments are standard of care for ophthalmology with the exception of optical coherence tomography (OCT)-- a non-invasive procedural device that records graphical and numerical measurements of the optic nerve and macula.

All patients will begin active combination therapy on both CellCept® and Avonex® during the second 6 months of the study. During this second phase, MRI and clinical examinations will be performed.

• Study Type: Interventional
• Enrollment (Actual): 43
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • Michigan
    • Farmington Hills, Michigan, United States, 48334
      • Michigan Institute for Neurological Disorders (M.I.N.D.)
  • New York
    • Buffalo, New York, United States, 14203
      • Buffalo Neuroimaging Analysis Center (BNAC)
  • Texas
    • Dallas, Texas, United States, 75390
      • University of Texas Southwestern Medical Center at Dallas

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 55 years (Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Patient diagnosed with clinically definite MS according to McDonald criteria #1-#4
• Age 18-55
• Have a RR disease course
• Have EDSS scores less than or equal to 5.0
• Have a disease duration of one day to 20 years
• Have at least one medically documented clinical relapse within the 12 months prior to randomization (for eligibility, a pre-study relapse will be defined as neurologic symptoms and signs documented by review of the history with the subject or in the medical record, of sufficient severity and duration to be determined by the investigator as consistent with an acute MS relapse; the relapse does not need to have been treated to qualify) and/or have progression of ≥1.0 points in EDSS in the previous year
• Have ≥1 Gd-enhancing brain lesion on a monthly run-in baseline MRI and ≥2 T2 brain lesions consistent with MS on the screening scan
• Signed informed consent
• None of the exclusion criteria

Exclusion Criteria:

• Previous treatment 3 months prior to study entry with standard disease-modifying therapy (interferon-beta and glatiramer acetate, IVIG and plasmaphoresis).
• Previous treatment 12 months prior to study entry with immunosuppressant agents, e.g., mitoxantrone, cyclophosphamide, cladribine, fludarabine, cyclosporine or total body irradiation or any other concomitant immunomodulatory therapies (e.g., azathioprine, methotrexate,, CellCept®, natalizumab, and other immunomodulators/monoclonal agents).
• Patients who received steroid treatment 30 days prior to the MRI scan date
• Women who are pregnant, lactating or of childbearing age who do not consent to approved contraceptive use during the study.
• Abnormal blood tests, performed during the screening visit (see adverse events section)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Double

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Active Comparator: Cellcept and Avonex	Drug: Mycophenolate Mofetil (CellCept)

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
The primary objective of this safety/mechanistic study is to determine the safety and tolerability of oral Cellcept when compared with weekly intramuscular Avonex in relapsing multiple sclerosis. Safety will be assessed by virtue of changes in MRI	1 year

Secondary Outcome Measures

Outcome Measure	Time Frame
Secondary Objectives:	one year
Changes in exacerbation frequency, incidence of exacerbations in the treated groups, changes in level of sustained disability	one year
, changes in quality of life measures, assessment of fatigue	one year

Collaborators and Investigators

• Sponsor: University of Texas Southwestern Medical Center
• Collaborators: Aspreva Pharmaceuticals
• Investigators: Principal Investigator: Elliot M Frohman, MD/PhD, University of Texas, Southwestern Medical Center at Dallas

Study record dates

Study Major Dates

• Study Start: May 1, 2006
• Primary Completion (Actual): June 1, 2009
• Study Completion (Actual): June 1, 2009

Study Registration Dates

• First Submitted: May 9, 2006
• First Submitted That Met QC Criteria: May 9, 2006
• First Posted (Estimate): May 11, 2006

Study Record Updates

• Last Update Posted (Estimate): June 19, 2013
• Last Update Submitted That Met QC Criteria: June 17, 2013
• Last Verified: June 1, 2013

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Nervous System Diseases; Immune System Diseases; Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Demyelinating Diseases; Autoimmune Diseases; Multiple Sclerosis; Sclerosis; Molecular Mechanisms of Pharmacological Action; Anti-Infective Agents; Enzyme Inhibitors; Antineoplastic Agents; Anti-Bacterial Agents; Antibiotics, Antineoplastic; Antitubercular Agents; Antibiotics, Antitubercular; Mycophenolic Acid
• Other Study ID Numbers: IIT 355349; IRB #012006-028