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Wilson Disease Clinical Trials

Clinical trial opens at Yale for Wilson disease:
Single daily dosage of trientine for maintenance treatment for Wilson disease.

Wilson disease requires life-long medical treatment. A serious issue for patients with Wilson disease is adherence to treatment, or simply put, how well patients take their prescribed medications. We know all too well that when a doctor prescribes medications that this does not assure that patients take them properly. From experience we have learned that the more times a day that a patient has to take medications, the more likely that doses will be missed. The consequence of not taking medication for Wilson disease is serious liver injury, injury to the central nervous system, liver failure and death. Each year we hear of some patients who have stopped their medications even temporarily and have suffered terribly, leading to severe disability or death.

Treatment for Wilson disease with zinc requires multiple daily dosages of the zinc to be effective, and zinc must be taken apart from meals. The drugs penicillamine and trientine are chelation agents that act by removing copper from the body. These medications are also typically prescribed in multiple daily dosages, from two to four times daily. Like zinc, they are most effective given one hour before or two hours after meals. While zinc is ineffective if taken once daily, it was theoretically possible that taking either penicillamine or trientine as a single daily dosage would remove enough copper to keep patients in balance and prevent worsening disease. It came to our attention that a handful of patients were using their medications this way, and we reported on their experience in a medical publication in the American Journal of Gastroenterology in 2008. These patients all had stable disease and were well maintained by taking all of their daily medication, either penicillamine or trientine, at a single time during the day apart from their meals. This was very exciting since if taking medication this way would increase adherence it would prevent a number of people from missing their needed medicines. Furthermore those that took the medication only once a day would only have to worry about timing the dose around a single meal and not interrupt their daily routine. For these reasons, we were very excited about the possibility to test this out in other patients in a prospective manner in a very controlled setting so that we could assure patient safety. By studying this properly we will learn more about how safe and effective taking a chelating drug as a single daily dose really is. If we are successful in this study, we will be able to recommend to other patients with Wilson disease that they may take trientine as a single dosage, hopefully preventing or at least reducing non adherence as well as making treatment more convenient.

In order to determine that we are correct in our belief that taking the medication trientine in a single daily dose can effectively maintain the health and copper balance for individuals with treated and stable Wilson disease, we have opened a clinical trial at Yale and are now seeking patients interested in participating. To be eligible patients must have been treated for over one year for their Wilson disease and have stable liver disease or neurological signs and symptoms. Medication, trientine (Syprine) will be provided for patients free of charge during this one year trial. They will have to visit us for testing and examinations during the course of the study and any non-routine examinations and testing will be provided for free as part of the study. At the end of the one year treatment period, patients may return to their prior treatments for their Wilson disease or choose to continue the trientine at the same daily dosage. We will ask you for permission to be followed in the future to make sure that you are doing well and to learn about your treatment choices. Potential benefits from participating in this clinical trial include the provision of medication during this year, the ease of taking the medication only once a day and your clinical examinations testing that is overseen by Dr. Schilsky, one of the leading experts in the field of Wilson disease.

If you are interested in participating in this clinical trial please contact Stefania at 203 785-2068 or Dr. Schilsky at 203-737-1592, or e mail: Michael.Schilsky@yale.edu.

The trial is made possible by the generous support of the Yale Center for Clinical Investigation and Aton Pharma Inc. Disclosures: Dr. Schilsky is on the Medical Advisory Committee to the Wilson Disease Association and is a Medical Advisor to Aton Pharma Inc.

Protocol number: 0902004694

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