Fabry DiseasePhase 3MODIFYNCT03425539
Efficacy and Safety of Lucerastat Oral Monotherapy in Adult Subjects With Fabry Disease
A Phase 3 study testing Lucerastat in people with Fabry Disease, run by Idorsia Pharmaceuticals Ltd. at 49 sites in 14 countries.
Trial summary
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Fabry Disease
- What is being tested?
- Lucerastat
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 118 participants
- Where
- 49 sitesin 14 countries
- Study dates
- 21 Jun 2018to 2 Sep 2021
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Show all 10Show fewer
- 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.
Who cannot join
- 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.
Trial locations and contacts
49 sites in 14 countries.
49 sites in 14 countries
United States17 sites
Emory University School of Medicine; Department of Human Genetics
Atlanta, Georgia, 30322
University of Alabama at Birmingham - Nephrology Research Clinic
Birmingham, Alabama, 35233
Massachusetts General Hospital
Boston, Massachusetts, 02114
Rush University Medical Center
Chicago, Illinois, 60612
Research Baylor Institute of Metabolic Disease
Dallas, Texas, 75226
Duke University Medical Center
Durham, North Carolina, 27710
Lysosomal and Rare Disorders Research and Treatment Center
Fairfax, Virginia, 22030
University of Florida College of Medicine - Division of Nephrology, Hypertension & Renal Transplantation
Gainesville, Florida, 32610
Infusion Associates
Grand Rapids, Michigan, 49525
Greenwood Genetic Center
Greenville, South Carolina, 29605
University of Iowa Stead Family Children's Hospital - Division of Medical Genetics
Iowa City, Iowa, 52242
University of California Irvine
Irvine, California, 92696
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
University of Pennsylvania
Philadelphia, Pennsylvania, 19104
Children's Hospital of Pittsburgh (UPMC)
Pittsburgh, Pennsylvania, 15224
Oregon Health and Science University
Portland, Oregon, 97239
University of Utah - Division of Medical Genetics
Salt Lake City, Utah, 84113
Canada6 sites
M.A.G.I.C Clinic Ltd
Calgary, T2M 0L6
Queen Elizabeth II Health Sciences Center - Halifax Infirmary - Division of Nephrology
Halifax, B3H 3A7
London Health Sciences Centre - Victoria Hospital
London, Ontario, N6A 5W9
Research Center, Hôpital Du Sacré-Coeur de Montréal
Montreal, H4J 1C5
Vancouver Hospital & Health Sciences - Vancouver General Hospital
Vancouver, V5Z 1M9
Health Sciences Center Winnipeg
Winnipeg, R3A 1S1
Germany4 sites
Charite Campus Virchow-Klinikum - Nephrologie und Internistische Intensivmedizin
Berlin, 13353
SphinCS GmbH
Höchheim, 65239
Fachinternistische Gemeinschaftspraxis Markgräferland
Mühlheim, 79379
Medizinische Klinik und Poliklinik I der Universität - Schwerpunkt Nephrologie
Würzburg, 97080
Spain4 sites
Hospital Universitari Vall d'Hebrón
Barcelona, 08035
Hospital Universitari de Bellvitge; Hospitalet de Llobregat
Barcelona, 08907
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
Madrid, 28034
Hospital Quironsalud Zaragoza
Zaragoza, 50012
United Kingdom4 sites
University Hospital Birmingham NHS Foundation Trust - Center for Rare Diseases
Birmingham, B15 2WB
The Royal Free Hospital, Department of Haematology Royal Free London NHS Foundation Trust
London, NW3 2QG
National Hospital for Neurology and Neurosurgery
London, WC1N3BG
Salford Royal (Hope) Hospital
Salford, M6 8HD
Poland3 sites
University Hospital in Cracow - Dep. of of Allergies and Immunology
Krakow, 31-066
Cardinal Wyszynski Institute of Cardiology
Warsaw, 04-628
Department of Pediatric Nutrition and Metabolic Diseases; The Children's Memorial Health Institute
Warsaw, 04-730
Australia2 sites
Royal Melbourne Hospital - Department of Nephrology
Parkville, 3050
Royal Perth Hospital, Department of Nephrology
Perth, 6000
Belgium2 sites
University Hospital Ghent
Ghent, 9000
University Hospital Leuven
Leuven, 3000
Italy2 sites
ASST Monza, Hospital San Gerardo, Nephrology
Monza, 20900
University of Naples Federico II (Nephrology)
Naples, 80131
Austria1 site
Allgemeines Krankenhaus der Stadt Wien - Universitätsklinik für Innere Medizin III - Klinische Abteilung für Nephrologie und Dialyse
Vienna, 1090
Netherlands1 site
Hospital Academisch Medisch Centrum - Department of Internal Medicine Div. Endrocrinology and Metabolism
Amsterdam, 22660
Switzerland1 site
Universität Zürich Psychiatrische Universitätsklinik
Zurich, 8032
About this study
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
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
Read the 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.
- Conditions
- Fabry Disease
- Being tested
- Lucerastat, Placebo
- Medical subject headings
- Fabry Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
LucerastatDrug
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.
PlaceboDrug
Placebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function.
| Study group | Role | What they receive |
|---|---|---|
| Lucerastat | Experimental | Not described Receives: Drug: Lucerastat |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Neuropathic Pain Monthly Score: Change From Baseline to Month 6
Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
Measured: From baseline to Month 6 (duration: 6 months)
Secondary outcomes (3)
Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6
Measured: From baseline to Month 6 (duration: 6 months)
Abdominal Pain Monthly Score: Change From Baseline to Month 6
Abdominal pain on the 11-point Numerical Rating Scale (NRS-11): subjects rated their abdominal pain intensity ("abdominal pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no pain) to 10 (worst imaginable pain).
Measured: From baseline to Month 6 (duration: 6 months)
Number of Days With Diarrhea: Change From Baseline to Month 6
A subject was considered to have diarrhea on a specific day if at least one stool of a Bristol Stool Scale (BSS) consistency Type 6 or 7 was reported. The number of days with diarrhea at baseline and Month 6 was the number of days with diarrhea over the 4 weeks prior to the randomization visit or the Month 6 visit, respectively, adjusted for the number of days with data available.
Measured: From baseline to Month 6 (duration: 6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Feb 2018
- Study start21 Jun 2018
- Primary completion17 Aug 2021
- Study completion2 Sep 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Fabry DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 118 participants (actual)
Sponsor and investigators
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trials, Study director, Idorsia Pharmaceuticals Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03425539
- Sponsor's protocol ID
- ID-069A301
- Acronym
- MODIFY
- Status last verified
- Aug 2024
- First submitted
- 16 Jan 2018
- Last updated
- 9 Aug 2024
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03425539
- Acronym / sponsor ID
- MODIFY / ID-069A301
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 118 participants (actual)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- 21 Jun 2018 (actual)
- Primary completion
- 17 Aug 2021 (actual)
- Study completion
- 2 Sep 2021 (actual)
- First posted
- 7 Feb 2018 (actual)
- Results
- Posted 9 Aug 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study aimed to determine the efficacy and safety of lucerastat oral monotherapy in adult subjects with Fabry disease.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Lucerastat | Experimental | Drug: Lucerastat |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (10 listed)
- 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:
Key exclusion criteria (6 listed)
- 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).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| LucerastatHard gelatin capsules containing 250 mg of lucerastat and inactive excipients; 1000 mg (4 capsules) twice daily (b.i.d.); dose adjusted for renal function. | Drug | Lucerastat |
| PlaceboPlacebo capsules are identical in appearance to the lucerastat capsules, and contain inactive excipients; 4 capsules b.i.d.; dose adjusted for renal function. | Drug | Placebo |
Primary outcome
Neuropathic Pain Monthly Score: Change From Baseline to Month 6
Time frame: From baseline to Month 6 (duration: 6 months)
Neuropathic pain on the modified BPI-SF3: subjects rated their neuropathic pain intensity ("neuropathic pain at its worst in the last 24 hours") on an 11-point scale, from 0 (no neuropathic pain) to 10 (worst imaginable neuropathic pain).
3 secondary outcomes
Plasma Globotriaosylceramide (Gb3; in ng/ml): Change From Baseline to Month 6
Time frame: From baseline to Month 6 (duration: 6 months)
Abdominal Pain Monthly Score: Change From Baseline to Month 6
Time frame: From baseline to Month 6 (duration: 6 months)
Number of Days With Diarrhea: Change From Baseline to Month 6
Time frame: From baseline to Month 6 (duration: 6 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Aug 2024.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Fabry Disease
RecruitingPhase 3
A Study to Learn How Well Lucerastat Works and How Safe it is in Untreated Adult Male Participants With Fabry Disease
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RecruitingObservational study
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
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A Study of Patients With Fabry Disease (US Specific)
Fabry Disease
Active, not recruiting
Evaluation of the Effect of a Low-FODMAP Diet on Gastrointestinal Symptoms and Inflammatory Markers in Fabry Patients
Fabry Disease
RecruitingPhase 3
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trials study director
Idorsia Pharmaceuticals Ltd.
49 sites in 14 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Lucerastat in people with Fabry Disease, run by Idorsia Pharmaceuticals Ltd. at 49 sites in 14 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 10 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 9 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 49 sites in United States, Canada, Germany, Spain, United Kingdom and 9 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 9 Aug 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Neuropathic Pain Monthly Score: Change From Baseline to Month 6 (time frame: From baseline to Month 6 (duration: 6 months)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Aug 2024.