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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT03425539
Recruitment Status : Recruiting
First Posted : February 7, 2018
Last Update Posted : September 24, 2020
Information provided by (Responsible Party):
Idorsia Pharmaceuticals Ltd.

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

Condition or disease Intervention/treatment Phase
Fabry Disease Drug: Lucerastat Drug: Placebo Phase 3

Detailed Description:
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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 108 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : June 21, 2018
Estimated Primary Completion Date : May 15, 2021
Estimated Study Completion Date : June 15, 2021

Resource links provided by the National Library of Medicine

Arm Intervention/treatment
Experimental: Lucerastat Drug: Lucerastat
Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) b.i.d.; dose adjusted for renal function.

Placebo Comparator: Placebo Drug: Placebo
Placebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function.

Primary Outcome Measures :
  1. Response to study treatment on neuropathic pain defined as a reduction from baseline to Month 6 of at least 30% in the "modified" Brief Pain Inventory-Short Form 3 (BPI-SF3) score of "neuropathic pain at its worst in the last 24 hours" [ Time Frame: From baseline to Month 6 (duration: 6 months) ]

Secondary Outcome Measures :
  1. Change from baseline to Month 6 in the average daily 11-point Numerical Rating Scale (NRS-11) score of "abdominal pain at its worst in the last 24 hours" in subjects with GI symptoms at baseline. [ Time Frame: From baseline to Month 6 (duration: 6 months) ]
  2. Change from baseline to Month 6 in the number of days with at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 in subjects with GI symptoms at baseline. [ Time Frame: From baseline to Month 6 (duration: 6 months) ]
  3. Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3). [ Time Frame: From baseline to Month 6 (duration: 6 months) ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03425539

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Contact: Clinical Trial Disclosure Desk +18566613721

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Sponsors and Collaborators
Idorsia Pharmaceuticals Ltd.
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Study Director: Clinical Trials Idorsia Pharmaceuticals Ltd.
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Responsible Party: Idorsia Pharmaceuticals Ltd. Identifier: NCT03425539    
Other Study ID Numbers: ID-069A301
First Posted: February 7, 2018    Key Record Dates
Last Update Posted: September 24, 2020
Last Verified: September 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Fabry Disease
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Cerebral Small Vessel Diseases
Cerebrovascular Disorders
Vascular Diseases
Cardiovascular Diseases
Genetic Diseases, X-Linked
Genetic Diseases, Inborn
Metabolism, Inborn Errors
Lipid Metabolism, Inborn Errors
Lysosomal Storage Diseases
Metabolic Diseases
Lipid Metabolism Disorders