Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease



Status:Recruiting
Conditions:Hematology, Metabolic
Therapuetic Areas:Hematology, Pharmacology / Toxicology
Healthy:No
Age Range:18 - Any
Updated:3/15/2019
Start Date:June 21, 2018
End Date:January 7, 2020
Contact:Clinical Trial Disclosure Desk
Email:clinical-trials-disclosure@idorsia.com
Phone:+41 58 844 0000

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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

This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult
subjects with Fabry disease.

The primary objective of this prospective, multicenter, double-blind, randomized,
placebo-controlled, parallel group, Phase 3 study is to determine the effect of oral
lucerastat monotherapy on neuropathic pain in subjects with Fabry disease (FD) through daily
collection of patient-reported outcomes with an electronic diary.

Inclusion Criteria:

1. Signed and dated ICF prior to any study-mandated procedure;

2. Male or female adult subjects;

3. FD diagnosis confirmed with local genetic test results;

4. Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months
prior to screening;

5. Enzyme replacement therapy (ERT) status:

1. Subject never treated with ERT; or

2. Subject has not received ERT for at least 6 months prior to screening; or

3. Subject treated with ERT since at least 12 months at the time of the screening
visit, and agreeing to stop ERT for approximately 8 months.

6. A woman of childbearing potential is eligible only under certain conditions, e.g.
taking contraceptive measures.

7. Subjects with moderate or severe neuropathic pain during the screening period.

Exclusion Criteria:

1. Pregnant, planning to be become pregnant, or lactating subject.

2. Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.

3. Subject on regular dialysis for the treatment of chronic kidney disease.

4. Known and documented transient ischemic attack, stroke, unstable angina, or myocardial
infarction within 6 months prior to screening.

5. Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic
arrhythmia, congestive heart failure NYHA class III or IV).

6. Any known factor or disease that might interfere with treatment compliance, study
conduct or interpretation of the results.
We found this trial at
12
sites
4401 Penn Avenue
Pittsburgh, Pennsylvania 15224
412-692-5325
Children's Hospital of Pittsburgh of UPMC UPMC is one of the leading nonprofit health systems...
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3959 Pender Drive
Fairfax, Virginia 22030
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Gainesville, FL
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Iowa City, IA
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Irvine, California 92697
949-824-5011
University of California, Irvine Since 1965, the University of California, Irvine has combined the strengths...
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515 Delaware Street Southeast
Minneapolis, Minnesota 55455
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