Sites Open Soon in Multinational Trial of Idebenone in DMD
Idebenone, an antioxidant and possible cellular energy booster, will be tested in some 240 boys with DMD



The idebenone trial will look for any changes in respiratory function, motor function or quality of life.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.

Watch the MDA clinical trials site and ClinicalTrials.gov for information about enrollment, as it becomes available.

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Ofelia,
I can't wait until it is approved for Duchenne! Joshua is taking idebenone right now and we started seeing great results within 2 weeks. He's been on it now for almost three months. Even though we are taking idebenone and not "Catena", they both have the same ingredients et al.
My problem with the Catena which is already available in Canada, where I'm from, is what they charge for it. When we were going to get it through prescription (which we had), they were going to charge us $20,000.00 for a year's worth... and we have to buy in year supply each time. We worked it out to see how much each pill would cost since he would only need one per day, and came out with about $55/pill. I hope if more is approved the price will come down. Not everyone can afford it!

Naomi
We have been using Idebenone now for almost 9 months and swear by it. It really does make a day and night difference. Less falls, more stability. More energy, less fatigue.
Wow, so expensive!!! I think they take advantage that there are no other drugs approved for FA or DMD and set the price based on that. It is scary to hear these numbers and think what will happen in the future when/if 2-3 drugs will be approved. Not to mention exon skipping, if idebenone is 20K... The price will be out of control!

Joshua's mom said:
Ofelia,
I can't wait until it is approved for Duchenne! Joshua is taking idebenone right now and we started seeing great results within 2 weeks. He's been on it now for almost three months. Even though we are taking idebenone and not "Catena", they both have the same ingredients et al.
My problem with the Catena which is already available in Canada, where I'm from, is what they charge for it. When we were going to get it through prescription (which we had), they were going to charge us $20,000.00 for a year's worth... and we have to buy in year supply each time. We worked it out to see how much each pill would cost since he would only need one per day, and came out with about $55/pill. I hope if more is approved the price will come down. Not everyone can afford it!

Naomi
What brand are you using? Does it come in pills and/or liquid? What dose are you using?

Thanks.

Kulwant Pannu said:
We have been using Idebenone now for almost 9 months and swear by it. It really does make a day and night difference. Less falls, more stability. More energy, less fatigue.
We order from a website called smartpowders.com It comes in a powder form. I recently met a parent who has a child with a different form of MD and she takes the powder and makes pills out of it. We mix it into a teaspoon of honey and ginger. Neel just turned 3 he's only taking 1/8 of a teaspoon right now.

Ofelia Marin said:
What brand are you using? Does it come in pills and/or liquid? What dose are you using?

Thanks.

Kulwant Pannu said:
We have been using Idebenone now for almost 9 months and swear by it. It really does make a day and night difference. Less falls, more stability. More energy, less fatigue.
Thanks!

Kulwant Pannu said:
We order from a website called smartpowders.com It comes in a powder form. I recently met a parent who has a child with a different form of MD and she takes the powder and makes pills out of it. We mix it into a teaspoon of honey and ginger. Neel just turned 3 he's only taking 1/8 of a teaspoon right now.

Ofelia Marin said:
What brand are you using? Does it come in pills and/or liquid? What dose are you using?

Thanks.

Kulwant Pannu said:
We have been using Idebenone now for almost 9 months and swear by it. It really does make a day and night difference. Less falls, more stability. More energy, less fatigue.
I buy it from Smart nutrition. It comes in pills they can swallow. We get 150mg pills. Joshua is almost 7 and weighs 64lbs of no fat! Another one I know is 12 and weighs 110 so they take 3/day. I hope you can get what you need here.

http://www.smartnutrition.info/idebenone.html

Naomi
Can we mix Idebenone and CoQ10? I realize they work pretty much the same, but I see online at Smart Nutrition that they have a mix of Idebenone and CoQ10. Kelvin's CoQ10 levels are good since starting Qunol Liquid CoQ10, so I don't want to stop that and would like to possibly add the powdered Idebenone. Thanks, Michelle

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