Clinical Trial of Idebenone in Primary Progressive Multiple Sclerosis (IPPoMS)

Completed · Phase 1/Phase 2 · Has a placebo group

Conditions studied: Primary Progressive Multiple Sclerosis

In brief

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.

Key facts

Study ID

NCT00950248

Run by

National Institute of Allergy and Infectious Diseases (NIAID)

People needed

85

Starts

2009-11-01

Expected to finish

2018-08-06

Last updated by the study team

2019-03-19

Who can join

Age: 18 and older, up to 65. Sex: any. Healthy volunteers: not accepted.

You may not qualify if…

• Alternative diagnoses that can explain neurological disability and MRI findings
• 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
• History of hypersensitivity reaction to idebenone or coenzyme-Q (10)
• Pregnant or lactating women. All women of child-bearing potential must have a negative pregnancy test prior to the medication phase of the study.
• 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
• 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

Where it is running

• National Institutes of Health Clinical Center, 9000 Rockville Pike — Bethesda, Maryland, United States

Full record on ClinicalTrials.gov

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