IPPoMS

Treatment tested

• Idebenone

  Antioxidant and mitochondrial electron carrier

Placebo or sham

• placebo

Other measures (6)

* INCLUSION CRITERIA: 1. PP-MS as determined by the 2005 modification of McDonald s diagnostic criteria 2. Age from 18-65 years (inclusive) 3. Expanded Disability Status Scale (EDSS) measure of neurological disability from 1 (no disability, clinical signs only) to 7 (ambulatory with bilateral support) 4. Able to provide informed consent 5. Willing to participate in all aspects of trial design and follow-up 6. If able to become pregnant or to father a child, agreeing to commit to the use of a reliable/accepted method of birth control (i.e. hormonal contraception (birth control pills, injected hormones, vaginal ring), intrauterine device, barrier methods with spermicide (diaphragm with spermicide, condom with spermicide) or surgical sterilization (hysterectomy, tubal ligation, or vasectomy in a partner)) for the duration of treatment arm of the study 7. Not receiving any immunomodulatory/immunosuppressive therapies for a period of at least 3 months before enrollment in the study 8. No exposure to idebenone, coenzyme-Q(10) or other dietary supplements (such as antioxidants, mitochondrial-function promoting supplements or vitamins in excess of 3 times recommended daily doses) for a period of at least 1 month before enrollment in the study EXCLUSION CRITERIA: 1. Alternative diagnoses that can explain neurological disability and MRI findings 2. Clinically significant medical disorders that, in the judgment of the investigators, could cause CNS tissue damage or limit its repair, or might expose the patient to undue risk of harm or prevent the patient from completing the study 3. History of hypersensitivity reaction to idebenone or coenzyme-Q (10) 4. Pregnant or lactating women. All women of child-bearing potential must have a negative pregnancy test prior to the medication phase of the study. 5. Abnormal screening/baseline blood tests exceeding any of the limits defined below: i. Serum alanine transaminase or aspartate transaminase levels greater than 3 times the upper limit of normal values ii. Total white blood cell count \< 3,000/mm(3) iii. Platelet count \< 85,000/mm(3) iv. Serum creatinine level > 2.0 mg/dl or eGFR (estimated glomerular filtration rate) \<30 v. Positive pregnancy test 6. Patients who are receiving any immunosuppressive therapies (including cytostatic agents) due to the concern that these drugs may contribute to neurodegeneration or limit CNS repair

Background: * Multiple sclerosis (MS) is an inflammatory disorder of the central nervous system that progressively weakens and destroys the pathways of the nervous system. About 10 percent to 15 percent of patients develop primary-progressive MS (PP-MS), characterized by progressive accumulation of disability from the disease onset, without any marked improvements or relapses. There are currently no effective treatments for PP-MS. * Idebenone is a manmade drug that is similar to a naturally occurring compound known as coenzyme Q10, a common dietary supplement. Research data suggest that idebenone may be able to limit demyelination and death of brain cells and thereby slow or halt the progression of neurological dysfunction such as that occurring in MS. Objectives: - To evaluate the safety and effectiveness of using idebenone to treat primary progressive MS. Eligibility: - Individuals between 18 and 65 years of age who have been diagnosed with primary progressive multiple sclerosis. Design: * The study will last 3 years and will be divided into two parts: a 1-year pretreatment baseline and 2 years of treatment with either idebenone or a placebo. * Pre-treatment study: approximately 5 clinic visits over 1 year. * Visit 1: Comprehensive medical history and neurological examination, with brain scans and neurological tests. * Visit 2: Magnetic resonance imaging (MRI) scan of the spine and lymphocytapheresis (withdrawal of white blood cells for testing). * Visit 3: Lumbar puncture. * Visit 4: Skin biopsy. * Visit 5: Repeat MRI of the brain and spinal cord, as well as neurological tests; these tests will be scheduled over 2 days. * After the five pretreatment visits, patients will receive a 6-month supply of study medication (either idebenone or a placebo) to take three times a day with food * Patients will continue to have regular followup clinic visits with brain MRI scans, blood tests, and other evaluations of brain and nervous system function. Randomly selected participants will have additional MRI scans for further safety precautions.