NCT00229632

Idebenone to Treat Friedreich's Ataxia

Completed Phase 2 Last updated 18 March 2019

What this trial tests

Phase 2 trial testing Idebenone in Friedreich Ataxia in 51 participants. Completed in 17 December 2007.

Timeline

27 September 2005

Primary endpoint
17 December 2007

17 December 2007

Quick facts

Lead sponsor	National Institute of Neurological Disorders and Stroke (NINDS)
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	single group
Masking	triple
Primary purpose	treatment
Enrollment	51
Start date	27 September 2005
Primary completion	17 December 2007
Estimated completion	17 December 2007
Sites	1 location across United States

Drugs / interventions tested

• Idebenone (IDEBENONE) — full drug profile →

Conditions studied

• Friedreich Ataxia — all drugs for Friedreich Ataxia →

Sponsor

National Institute of Neurological Disorders and Stroke (NINDS)

Who can join

Adults 9 to 17, any sex, with Friedreich Ataxia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This study will determine whether a drug called idebenone is safe and effective in reducing the level of oxidants that are believed to damage the nervous system and hearts in patients with Friedreich's ataxia. Friedreich's ataxia is caused by an abnormality in the gene that makes a protein called frataxin, which is necessary for the proper functioning of energy-producing parts of cells called mitrochondria. In Friedreich's ataxia, the mitochondria become overloaded with iron, and high levels of harmful compounds called oxidants are formed. These oxidants are believed to damage the cells of the nervous system and hearts of people with Friedreich's ataxia. Idebenone is a man-made drug similar to a naturally occurring compound known as Coenzyme Q10. This study will test whether idebenone can alleviate some of the symptoms of Friedreich's ataxia and slow or halt the progression of the disease.

Patients with genetically confirmed Friedreich's ataxia who are between 9 and 18 years of age, weigh between 65 and 175 pounds and can walk 25 feet with or without an assistive device may be eligible for this study. Candidates are screened with blood tests and a review of their medical records.

Participants undergo the following tests and procedures:

* Medical interview and physical examination. Tests include blood and urine tests, an electrocardiogram, or EKG (recording of the electrical activity of the heart), echocardiogram (ultrasound test showing the pumping action of the heart, thickness of the heart walls, and any valve leakage), and a detailed neurological examination, including maneuvers such as copying a drawing and putting pegs in a board. Patients' parents are asked questions about how they feel their child's disease affects the child's quality of life.
* Magnetic resonance imaging (MRI) to examine the heart muscle and blood flow to the heart. MRI uses a magnetic field and radio waves to produce images of body tissues and organs. The patient lies on a table that is moved into the doughnut-shaped MRI scanner, wearing earplugs to muffle loud knocking and thumping sounds that occur during the scanning process. A catheter (plastic tube) is placed in a vein in the child's arm so that a chemical called gadolinium can be injected during the MRI study. Gadolinium brightens areas of the heart, improving the ability to see the heart and blood flow.
* Physical medicine and rehabilitation evaluations to test the child's physical functioning. These tests include gait evaluation, measurements of the ability to exert and maintain a constant force, assessment of visual-motor control and fine motor control, aerobic exercise endurance testing, and measurement of the ability of the child's heart and lungs to increase their effectiveness with exercise.
* Idebenone/placebo treatment. Patients are given a 6-month supply of either idebenone pills or placebo (pills that look like the study drug but have no active ingredient) to take three times a day. Patients are seen by their primary care physician after 1 and 3 months on the study medication for a brief physical examination. In addition, they have blood and urine tests once a month while on medication to check for any abnormalities.
* 6-month examination. After 6 months on the study drug, patients return to NIH to repeat all the tests listed above to determine the effects of idebenone treatment.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Neurological effects of high-dose idebenone in patients with Friedreich's ataxia: a randomised, placebo-controlled trial.
   Di Prospero NA, Baker A, Jeffries N, Fischbeck KH. · · 2007 · cited 189× · PMID 17826341 · DOI 10.1016/s1474-4422(07)70220-x
2. Mitochondrial diseases: from molecular mechanisms to therapeutic advances.
   Wen H, Deng H, Li B, Chen J, et al · · 2025 · cited 111× · PMID 39788934 · DOI 10.1038/s41392-024-02044-3
3. Altered gene expression and DNA damage in peripheral blood cells from Friedreich's ataxia patients: cellular model of pathology.
   Haugen AC, Di Prospero NA, Parker JS, Fannin RD, et al · · 2010 · cited 95× · PMID 20090835 · DOI 10.1371/journal.pgen.1000812
4. Pharmacological advances in mitochondrial therapy.
   Singh A, Faccenda D, Campanella M. · · 2021 · cited 88× · PMID 33647769 · DOI 10.1016/j.ebiom.2021.103244
5. Rating disease progression of Friedreich's ataxia by the International Cooperative Ataxia Rating Scale: analysis of a 603-patient database.
   Metz G, Coppard N, Cooper JM, Delatycki MB, et al · · 2013 · cited 36× · PMID 23365101 · DOI 10.1093/brain/aws309
6. Current Drug Repurposing Strategies for Rare Neurodegenerative Disorders.
   Shah S, Dooms MM, Amaral-Garcia S, Igoillo-Esteve M. · · 2021 · cited 32× · PMID 34992533 · DOI 10.3389/fphar.2021.768023
7. Exercise capacity and idebenone intervention in children and adolescents with Friedreich ataxia.
   Drinkard BE, Keyser RE, Paul SM, Arena R, et al · · 2010 · cited 32× · PMID 20599042 · DOI 10.1016/j.apmr.2010.04.007
8. Characterizing gait, locomotor status, and disease severity in children and adolescents with Friedreich ataxia.
   Croarkin E, Maring J, Pfalzer L, Harris-Love M, et al · · 2009 · cited 11× · PMID 19809393 · DOI 10.1097/npt.0b013e3181b5112e