A multicenter, double-blind, randomized, placebo-controlled, parallel-group study to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Brief description of study
The purpose of the
study is to test if lucerastat, the study drug, can reduce symptoms of Fabry
disease such as pain and stomach symptoms when administered alone. The purpose
is also to see if lucerastat is safe for people with Fabry disease. Participants
may receive lucerastat or placebo. This will be decided by chance (like tossing
a coin), using a computer. Participants have a 2/3 chance of receiving the
study drug and 1/3 chances of receiving the placebo. This is a double-blind
study, which means neither you nor your study doctor will know if you're taking
lucerastat or the placebo. As the
purpose of this research study is to test if lucerastat administered alone is
effective, you should not receive enzyme replacement therapy for approximately
8 months. If you are currently receiving an enzyme replacement therapy, you
will have to stop receiving it.
Detailed description of study
Participation in the
study will last approximately 9 months for women and between 9 to 11 months for
men. This includes a screening period of 6-7 weeks to make sure the study is
suitable for you based on medical tests and electronic diary questionnaires.
During the study treatment period and post-study-treatment observation period,
four routine study visits will happen 1, 3, 5 and 6 months after starting study
treatment. Your study doctor/nurse will also call you 2 and 4 months after
starting study treatment. You will continue to record your symptoms and the
pain medication(s) you are taking every day at home in an electronic diary. You
will also record the number of capsules of study drug you are taking every day.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
-
Conditions:
Fabry disease
-
Age: Between 18 Years - 100 Years
-
Gender: All
- Have genetically-confirmed Fabry disease
- Suffer from neuropathic pain
- Agree to complete a daily electronic diary
Updated on
09 Feb 2021.
Study ID: 834541