Large-Scale Trial of Idebenone in DMD is Now Open

A large-scale, phase 3 trial of idebenone (Catena) in Duchenne muscular dystrophy (DMD) is now open at one U.S. site and several sites in Europe, under the auspices of Santhera Pharmaceuticals.

Results of a phase 2 trial of Catena, conducted in Belgium with 21 DMD-affected participants, were announced in 2007. The findings suggested the drug was safe and well tolerated at 450 milligrams per day for a year. In that small trial, those receiving Catena improved on certain cardiac and respiratory measurements compared to those receiving a placebo. (See DMD Idebenone Trial.)

About idebenone

Santhera, a Swiss pharmaceutical company, is developing idebenone under the brand name Catena, for a variety of neuromuscular disorders. The company has received approval with conditions for Catena to treat Friedreich's ataxia (FA) from Health Canada, the Canadian drug regulatory agency.

According to Santhera, Catena is a small molecule designed to facilitate the transport of electrons (subatomic particles) inside mitochondria, the energy-producing parts of cells. It is administered orally. Through preserving mitochondrial function and protecting cells from a type of damage known as oxidative stress (acting as an "antioxidant"), the drug's developer says, Catena may prevent cell damage and increase energy production in impaired nerve and muscle tissue.

About this trial

This one-year, phase 3 trial in North America and Europe is slated to include some 240 boys with DMD who are 10-18 years old, have a confirmed mutation in the dystrophin gene or substantially reduced levels of dystrophin protein in a muscle sample, and meet other study criteria.

During the first stage of the study, only prospective participants who have never used corticosteroids (glucocorticoids), such as prednisone, or who have stopped using corticosteroids at least a year prior to enrollment, will be accepted. Enrollment of participants taking corticosteroids may be allowed later in the study.

The U.S. study site, which is now open to recruitment, is at the Children's Hospital of Philadelphia (CHOP), under the direction of pediatric neurologist Richard Finkel, who co-directs the MDA clinic at that institution.

The investigators will assess the effect of 900 milligrams per day of idebenone on pulmonary function, motor function, muscle strength and quality of life, and they'll continue to evaluate the safety and tolerability of this drug.

Participants will be randomly assigned to receive either idebenone or a placebo and will be expected to make approximately eight visits to the study site over a year's time.

To participate

For details, see Phase III Study of Idebenone in Duchenne Muscular Dystrophy (DELOS). For information about the CHOP site, contact Nancy Videon, Senior Research Coordinator, at (267) 426-7163 or videon@email.chop.edu.

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