DMD Idebenone Trial

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This story was updated 11/9/10 with contact information for this trial and information about clinical trials in general.

Pediatric Neurologist Richard Finkel, co-director of the MDA neuromuscular disease clinic at Children's Hospital of Philadelphia, will be the lead investigator for the North American arm of a large international trial with idebenone in Duchenne muscular dystrophy (DMD).

Santhera Pharmaceuticals of Liestal, Switzerland is sponsoring the trial, which opened in 2009 in Europe and is expected to open in the United States and Canada early in 2010.

About idebenone (Catena/Sovrima)

Idebenone, which is chemically derived from the natural substance coenzyme Q10, is being developed by Santhera for the use in certain neuromuscular diseases. Santhera’s trade name for the drug in the United States is Catena. Takeda Pharmaceuticals has obtained European marketing rights to the molecule from Santhera, where it plans to use the trade name Sovrima.

Idebenone is believed to help cells produce cellular energy and is known to act as a powerful antioxidant, meaning it helps detoxify oxygen-containing byproducts of cellular metabolism known as free radicals, which can poison cells.

In July 2008, Catena received conditional market approval to treat the neuromuscular disease Friedreich's ataxia (FA) in Canada. The approval was based on the condition that Santhera provide additional information as it becomes available.

About DMD

DMD is degenerative muscle disease caused by any of a number of mutations in the X-chromosome gene that carries instructions for the muscle protein dystrophin. Without dystrophin, muscle fibers are abnormally fragile and break down under the stress of contractions.

Several experimental therapies are in development whose aim is to add dystrophin or a related protein to muscle fibers. At the same time, molecules to improve energy production, slow scar-tissue formation, combat inflammation or improve muscle repair also are being investigated and may be useful in treating this disease. (The anti-inflammatory drugs prednisone and deflazacort are routinely prescribed to treat DMD).

About this trial

This new trial of idebenone (Catena) is a phase 3 (large-scale) study that will enroll up to 240 walking and non-walking boys with DMD who are 10 to 18 years old, at up to 25 centers in Europe, the United States and Canada.

The safety, tolerability and clinical benefit of 900-milligram daily dose of Catena will be compared to a placebo (inactive, look-like substance) over the course of a year.

The main objective of this study is to demonstrate a change in respiratory function, using a measurement known as peak expiratory flow. The investigators will also look at other respiratory measurements, as well as changes in muscle strength, motor function and quality of life.

The principal investigator of the entire study is Gunnar Buyse, professor of child neurology at the University Hospitals in Leuven, Belgium. Professor Buyse was also the principal investigator of the preceding preclinical (nonhuman) and clinical (human) phase 2 program with idebenone in dystrophin-deficient muscular dystrophy.

A preceding phase 2 study conducted in Belgium included 21 DMD patients ages 8 to 16 and tested the safety, tolerability and effectiveness of a daily, 450-milligram dose of Catena compared to a placebo over a year's time. Thirteen participants received Catena, and eight received the placebo.

Results for the phase 2 study were announced in October 2007. Catena was safe and well tolerated with no apparent differences in the adverse events between the placebo group and the Catena group.
 
After the one-year treatment period, compared to participants on a placebo, trial participants who received Catena showed functional improvements in the region of the heart that is affected earliest and most severely affected in DMD. In addition, those receiving the drug improved on certain respiratory measurements, particularly peak flow, in contrast to those taking a placebo, who deteriorated over the same period.

As of October 2009, the U.S. and Canadian sites for the phase 3 idebenone trial in DMD are expected to begin recruiting participants early in 2010.

Editor's note 11/9/10: This trial is now open at Children's Hospital of Philadelphia. Contact Nancy Videon, Senior Research Coordinator, at (267) 426-7163 or videon@email.chop.edu.

About clinical trials

A clinical trial is a test, in humans, of an experimental medication or other therapy.

Clinical trials are not treatments, but experiments. Although it's possible that benefit may be derived from participating in a clinical trial, it's also possible that no benefit — or even harm — may occur. Therefore, the decision about whether to participate in a clinical trial requires careful consideration. Keep your MDA clinic doctor informed about any clinical trial participation.

Please be aware that MDA has no ability to influence who is chosen to participate in a clinical trial.

For more about clinical trials in general, see Understanding Clinical Trials, and the Quest magazine article about trial participation in neuromuscular disease, Being a Co-Adventurer.