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.

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

Interested in the study

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