Idebenone for Primary Progressive Multiple Sclerosis

NCT01854359 | Completed Phase 1 Results FDA Drug

• 2 other identifiers: 13008813-I-0088
• Study Type: interventional
• Target: 61
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Multiple Sclerosis; Primary Progressive Multiple Sclerosis

Keywords

Multiple Sclerosis; Extension Trial

Outcome Measures

Primary Outcomes (1)

• Area Under the Curve (AUC) of the Combinatorial Weight-Adjusted Disability Score (CombiWISE)

  CombiWISE is a composite scale derived from Expanded Disability Status Scale (EDSS) , Scripps Neurological Disability Scale (SNRS), times 25 foot walk (25FW), and non-dominant hand of 9 hole peg test (9HPT) with a minimum value of 0 (no disability) and maximum value of 100 (maximum disability). The AUC values were calculated for both study groups (Active treatment group in the 09-I-0197 trial and Placebo group in the 09-I-0197) as follows: 1. pre-treatment baseline during the 09-I-0197 trial (from Months -12, -6, and 0) 2. double-blind phase during the 09-I-0197 trial (from Months 0, 6, 12, 18, and 24) 3. extension phase during the 13-I-0088 trial (from Months 24, 30, and 36) Because the follow-up times varied from patient to patient, the AUC values were made comparable by scaling them by dividing the AUC value by the square of the actual duration (in years) of each of the phases.

  1-year pre-treatment baseline vs 2-year randomized double-blind phased vs 1-year treatment period

Secondary Outcomes (4)

• Slopes of 25 Foot Walk (25FW) Time

  3-years double-blind phase and 1-year extension phase

• Slopes of 9 Hole Peg Test (9HPT) Time

  3-years double-blind phase and 1-year extension phase

• Slopes of Expanded Disability Status Scale (EDSS) Score

  3-years double-blind phase and 1-year extension phase

• Change in Slopes of Scripps Neurological Rating Scale (SNRS) Score

  3-years double-blind phase and 1-year extension phase

Study Arms (1)

Active treatment

OTHER

Idebenone (150mg tablets) administered orally as five tablets, three times per day with food.

Drug: Idebenone

Interventions

Idebenone DRUG

Active treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• Pregnant or lactating women. All women of child-bearing potential must have a negative pregnancy test
• Patients dropping out of IPPoMS due to adverse events (AE) considered related to study medication

Sponsors & Collaborators

• National Institute of Allergy and Infectious Diseases (NIAID): lead

Study Sites (1)

National Institutes of Health Clinical Center, 9000 Rockville Pike

Bethesda, Maryland, 20892, United States

Location

Related Publications (3)

• Albrecht P, Ringelstein M, Muller AK, Keser N, Dietlein T, Lappas A, Foerster A, Hartung HP, Aktas O, Methner A. Degeneration of retinal layers in multiple sclerosis subtypes quantified by optical coherence tomography. Mult Scler. 2012 Oct;18(10):1422-9. doi: 10.1177/1352458512439237. Epub 2012 Mar 2.

  PMID: 22389411 BACKGROUND

• Andrews HE, Nichols PP, Bates D, Turnbull DM. Mitochondrial dysfunction plays a key role in progressive axonal loss in Multiple Sclerosis. Med Hypotheses. 2005;64(4):669-77. doi: 10.1016/j.mehy.2004.09.001.

  PMID: 15694681 BACKGROUND

• Artuch R, Aracil A, Mas A, Colome C, Rissech M, Monros E, Pineda M. Friedreich's ataxia: idebenone treatment in early stage patients. Neuropediatrics. 2002 Aug;33(4):190-3. doi: 10.1055/s-2002-34494.

  PMID: 12368988 BACKGROUND

Related Links

• NIH Clinical Center Detailed Web Page

MeSH Terms

Conditions

Multiple Sclerosis; Multiple Sclerosis, Chronic Progressive

Interventions

idebenone

Condition Hierarchy (Ancestors)

Demyelinating Autoimmune Diseases, CNS; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Demyelinating Diseases; Autoimmune Diseases; Immune System Diseases; Chronic Disease; Disease Attributes; Pathologic Processes; Pathological Conditions, Signs and Symptoms

Results Point of Contact

• Title: Dr. Peter Kosa (Staff Scientist)
• Organization: National Institutes of Allergy and Infectious Diseases

peter.kosa@nih.gov

(301) 731-6444

Study Officials

• Bibiana Bielekova, M.D.

  National Institute of Allergy and Infectious Diseases (NIAID)

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: NIH
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 11, 2013
• First Posted: May 15, 2013
• Study Start: March 12, 2013
• Primary Completion: October 31, 2018
• Study Completion: October 31, 2018
• Last Updated: May 19, 2021
• Results First Posted: May 19, 2021

Record last verified: 2018-10

Locations

US (1)