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. Read our disclaimer for details. |
ClinicalTrials.gov Identifier: NCT03425539 |
Recruitment Status :
Completed
First Posted : February 7, 2018
Last Update Posted : August 9, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Fabry Disease | Drug: Lucerastat Drug: Placebo | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 182 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 |
Actual Primary Completion Date : | August 17, 2021 |
Actual Study Completion Date : | September 2, 2021 |
Arm | Intervention/treatment |
---|---|
Experimental: Lucerastat |
Drug: Lucerastat
Hard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) twice daily (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. |
- Change from baseline to Month 6 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) ]
- Change from baseline to Month 6 in the 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) ]
- 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) ]
- Change from baseline to Month 6 in plasma globotriaosylceramide (Gb3). [ Time Frame: From baseline to Month 6 (duration: 6 months) ]
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Signed and dated ICF prior to any study-mandated procedure;
- Male or female adult subjects;
- FD diagnosis confirmed with local genetic test results;
- Fabry-associated neuropathic pain, as defined by the subject, in the last 3 months prior to screening;
-
Enzyme replacement therapy (ERT) status:
- Subject never treated with ERT; or
- Subject has not received ERT for at least 6 months prior to screening; or
- 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.
- A woman of childbearing potential is eligible only under certain conditions, e.g. taking contraceptive measures.
- Subjects with moderate or severe neuropathic pain during the screening period.
Exclusion Criteria:
- Pregnant, planning to be become pregnant, or lactating subject.
- Severe renal insufficiency (eGFR < 30 mL/min/1.73 m2) at screening.
- Subject on regular dialysis for the treatment of chronic kidney disease.
- Known and documented transient ischemic attack, stroke, unstable angina, or myocardial infarction within 6 months prior to screening.
- Clinically significant unstable cardiac disease (e.g. uncontrolled symptomatic arrhythmia, congestive heart failure NYHA class III or IV).
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results.
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 ClinicalTrials.gov identifier (NCT number): NCT03425539
Study Director: | Clinical Trials | Idorsia Pharmaceuticals Ltd. |
Responsible Party: | Idorsia Pharmaceuticals Ltd. |
ClinicalTrials.gov Identifier: | NCT03425539 |
Other Study ID Numbers: |
ID-069A301 |
First Posted: | February 7, 2018 Key Record Dates |
Last Update Posted: | August 9, 2022 |
Last Verified: | August 2022 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Fabry Disease Sphingolipidoses 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 Lipidoses Lipid Metabolism, Inborn Errors Lysosomal Storage Diseases Metabolic Diseases Lipid Metabolism Disorders |