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Phase II Study - Amicus Therapeutics |
Hello Canadian Fabry Patient Network
One of the objectives of the Canadian Fabry Association is to support and educate those in the Fabry community in an effort to improve the lives of Fabry patients, their families and care givers.
As such, we would like to inform you of research being done that may be of interest to the Fabry disease community. Please see the attached details about ongoing Phase 2 studies of a new investigational treatment for both males and females with Fabry disease.
If you are interested in learning more about these studies, please contact the Canadian Fabry Association and/or visit the appropriate website identified.
Sincerely,
Adrian F. (Ed) Koning
President, Canadian Fabry Association
780-489-0012
email
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ANNOUNCING A STUDY OF A NEW INVESTIGATIONAL TREATMENT FOR MALES WITH FABRY DISEASE
STUDY TITLE:
"A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients With Fabry Disease"
WHAT IS THE PURPOSE OF THE STUDY?
This study will collect information about the safety and preliminary effect of a new, orally administered investigational drug to treat certain males with Fabry disease.
WHO CAN PARTICIPATE?
This study is open to males 18 years of age and older with Fabry disease who meet certain criteria; including, having a type of genetic mutation called a missense mutation in alpha-galactosidase A. The study
investigator can tell you if your mutation meets this requirement.
WHAT IS INVOLVED?
The study will involve nine visits to one of the investigational sites over a 30-week period. Participants in the research study will be eligible to continue in an extension phase of the protocol for an additional 24 weeks if they have a positive response to treatment. The treatment centers are: Montreal, Canada; London, United Kingdom; and Paris, France.
WHY SHOULD I PARTICIPATE?
Your participation in this study will be valuable for the continued development of this new investigational drug treatment. This new drug
may benefit individuals with Fabry disease in the future.
HOW WILL TRAVEL LOGISTICS BE HANDLED?
The reasonable costs of travel necessary for participation will be covered by the sponsor of the study.
WHO IS THE SPONSOR?
The sponsor of the study is Amicus Therapeutics, a biopharmaceutical company developing a novel class of small molecule, orally-administered drugs known as pharmacological chaperones. For more information on Amicus, please visit <www.amicustherapeutics.com>www.amicustherapeutics.com.
WHO SHOULD I CONTACT FOR MORE INFORMATION?
The study is currently active. If you are interested in learning more, please contact Adrian (Ed) Koning, President of the Canadian Fabry Association at 780-489-0012 or
This email address is being protected from spam bots, you need Javascript enabled to view it
Phase2StudyMales.pdf
ANNOUNCING A STUDY OF A NEW INVESTIGATIONAL TREATMENT FOR FEMALES WITH FABRY DISEASE
STUDY TITLE:
"A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients With Fabry Disease"
WHAT IS THE PURPOSE OF THE STUDY?
This study will collect information about the safety and preliminary effect of a new, orally administered investigational drug to treat certain females with Fabry disease.
WHO CAN PARTICIPATE?
This study is open to females 18 years of age and older with Fabry disease who meet certain criteria; including, having a type of genetic mutation called a missense mutation in alpha-galactosidase A. The study
investigator can tell you if your mutation meets this requirement.
WHAT IS INVOLVED?
The study will involve seven visits to one of the investigational sites over an 18-week period. Participants in the research study will be eligible to continue in an extension phase of the protocol for an additional 36 weeks if they have a positive response to treatment. The treatment centers are located in Montreal, Canada; Atlanta, Georgia; Manchester, United Kingdom; Paris, France; Melbourne, Australia; and
Porto Alegre, Brazil.
WHY SHOULD I PARTICIPATE?
Your participation in this study will be valuable for the continued development of this new investigational drug treatment. This new drug
may benefit individuals with Fabry disease in the future.
HOW WILL TRAVEL LOGISTICS BE HANDLED?
The reasonable costs of travel necessary for participation will be covered by the sponsor of the study.
WHO IS THE SPONSOR?
The sponsor of the study is Amicus Therapeutics, a biopharmaceutical company developing a novel class of small molecule, orally-administered drugs known as pharmacological chaperones. For more information on Amicus, please visit <www.amicustherapeutics.com>www.amicustherapeutics.com.
WHO SHOULD I CONTACT FOR MORE INFORMATION?
The study is currently active. If you are interested in learning more, please contact Adrian (Ed) Koning, President of the Canadian Fabry Association at 780-489-0012 or
This email address is being protected from spam bots, you need Javascript enabled to view it
 Phase2StudyFemales.pdf
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