Idebenone for Primary Progressive Multiple Sclerosis

This study is currently recruiting participants. (see Contacts and Locations)
Verified December 2013 by National Institutes of Health Clinical Center (CC)
Sponsor:
Information provided by (Responsible Party):
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
ClinicalTrials.gov Identifier:
NCT01854359
First received: May 11, 2013
Last updated: March 14, 2014
Last verified: December 2013

May 11, 2013
March 14, 2014
February 2013
October 2018   (final data collection date for primary outcome measure)
The primary outcome measure that comes out of the pre-determined analysis in the IPPoMS trial will be adopted as primary outcome measure in the IPPoMS-E trial. [ Time Frame: month 30 and 36 ] [ Designated as safety issue: Yes ]
Same as current
Complete list of historical versions of study NCT01854359 on ClinicalTrials.gov Archive Site
Neuroimaging, Clinical/functional, and biological/immunological outcomes include: Inhibition of the rates of development of brain atrophy, increased ventricular volume, SC atrophy.; Progression of neuro disability; Analysis of changes in CSF [ Time Frame: month 30 and 36 ] [ Designated as safety issue: Yes ]
Same as current
Not Provided
Not Provided
 
Idebenone for Primary Progressive Multiple Sclerosis
Open Label Extension Trial of Idebenone for Primary Progressive Multiple Sclerosis

Background:

- The Idebenone in Patients With Primary Progressive Multiple Sclerosis (IPPoMS) trial tested a new drug for multiple sclerosis. In the IPPoMS trial, participants took either idebenone or placebo. Researchers want to give idebenone to all participants for 1 year. It is still not certain whether idebenone can slow the progression of multiple sclerosis, but this study may help answer that question.

Objectives:

  • To provide idebenone to all participants on the IPPoMS trial.
  • To collect data on the safety and effectiveness of idebenone for primary progressive multiple sclerosis.

Eligibility:

- Individuals at least 18 years of age who have completed 3 years in the IPPoMS trial.

Design:

  • The first study visit for this trial will happen on the same day as the last visit for the IPPoMS trial.
  • Participants will provide a blood samples and will have a lumbar puncture. They will also receive a new supply of idebenone to take three times a day with food. They will keep a diary to report on any side effects.
  • After this first treatment visit, participants will have two follow-up visits to the NIH 6 months apart. These visits may be scheduled over multiple days. Participants will provide blood and urine samples. They will also have imaging studies of the brain and spine.
  • Participants will have phone calls with the study researchers to provide updates on their condition and any side effects.

OBJECTIVE:

A Phase I/II clinical trial is being conducted to investigate the safety, therapeutic efficacy and mechanism of action of idebenone in primary-progressive multiple sclerosis (PP-MS) patients (IPPoMS (Protocol Number 09-N-0197). Patients who have completed the 2-year treatment period of IPPoMS, may enter into this open-label extension study (IPPoMS-E) if they are found to be eligible by the Investigator and desire treatment with idebenone despite remaining blinded as to their allocation to active treatment versus placebo during the IPPoMS trial. The aim of this open-label extension study is gather additional data on safety, efficacy and effects of idebenone on CSF biomarkers in these patients over a period of 1 year. This study will provide open-label idebenone for patients with PP-MS, previously randomized to idebenone or to placebo in the blinded phase of IPPoMS.

STUDY POPULATION:

Patients who were previously enrolled in the IPPoMS (Protocol Number 09-N-0197) will be invited to participate in the trial. The same idebenone dose used in the randomized clinical trial (2250 mg/day) will be used in this study.

DESIGN:

This is a single group, open-label safety and efficacy extension trial with a one year treatment period. Patient-specific biomarkers of disease progression, CSF biomarkers of oxidative stress, longitudinal neuroimaging including quantitative measures of CNS tissue destruction and clinical data will be collected as in the randomized study.

OUTCOME MEASURES:

The measurement and collection of data will be performed as in the randomized trial. Quantitative neuroimaging measures of central nervous system (CNS: i.e. brain and spinal cord) tissue destruction and clinical and functional measures of neurological disability will be collected every 6-12 months. Additionally, biomarkers focusing on analysis of reactive oxygen species (ROS) and oxidative stress will be collected every 12 months. The primary outcome measure defined in the IPPoMS trial will be also utilized in IPPoMS-E. For patients originally randomized to placebo, patient-specific slopes of disease progression during 2 years of placebo therapy (as measured by primary and secondary outcomes) will be compared to patient-specific slopes of disease progression during 1 year of open label idebenone therapy. Combination of IPPoMS and IPPoMS-E trials will significantly expand paired no therapy vs. idebenone therapy CSF samples for biomarker studies. It will also provide (for the subgroup of subjects who were originally randomized to idebenone) longitudinal CSF samples on idebenone therapy (collected 2 years apart). This will allow calculations of intra-individual changes in CSF biomarkers on and off idebenone therapy, which may provide important insight into the mechanism of action of idebenone in PP-MS.

Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Multiple Sclerosis
  • Primary Progressive Multiple Sclerosis
Drug: Idebenone
N/A
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
80
October 2018
October 2018   (final data collection date for primary outcome measure)
  • INCLUSION CRITERIA:
  • Completion of 3 years in study IPPoMS (Protocol Number 09-N-0197)
  • Able to provide informed consent
  • Adults, at least 18 years of age
  • Willing to participate in all aspects of trial design and follow-up
  • 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)) for the duration of treatment arm of the study

EXCLUSION CRITERIA:

  • Pregnant or lactating women. All women of child-bearing potential must have a negative pregnancy test
  • Patients dropping out of IPPoMS due to AEs considered related to study medication
Both
18 Years and older
No
Contact: Joan M Ohayon, C.R.N.P. (301) 496-0064 eatonj@ninds.nih.gov
Contact: Bibiana Bielekova, M.D. (301) 402-4488 bielekovab@mail.nih.gov
United States
 
NCT01854359
130088, 13-N-0088
Not Provided
National Institutes of Health Clinical Center (CC) ( National Institute of Neurological Disorders and Stroke (NINDS) )
National Institute of Neurological Disorders and Stroke (NINDS)
Not Provided
Principal Investigator: Bibiana Bielekova, M.D. National Institute of Neurological Disorders and Stroke (NINDS)
National Institutes of Health Clinical Center (CC)
December 2013

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP