Fabry DiseasePhase 3NCT03737214
A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease
A Phase 3 study testing Lucerastat in people with Fabry Disease, run by Idorsia Pharmaceuticals Ltd. at 42 sites in 13 countries.
Trial summary
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat… 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?
- No, everyone receives an active treatment
- How many people
- 107 participants
- Where
- 42 sitesin 13 countries
- Study dates
- 18 Dec 2018to Nov 2029
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
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 ICF prior to any study-mandated procedure;
- Subject completed the 6-month, double-blind treatment period in study ID 069A301
- Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests.
- Fertile male only if agreement 1) to use a condom, 2) to not father a child.
Who cannot join
- Pregnant / planning to be become pregnant or lactating subject;
- Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.
- In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
- Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
- Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;
- Subject experienced an event of stroke CTCAE grade 3 or above;
- Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization.
Trial locations and contacts
42 sites in 13 countries.
42 sites in 13 countries
United States14 sites
University of Alabama at Birmingham - Nephrology Research Clinic
Birmingham, Alabama, 35294
Massachusetts General Hospital
Boston, Massachusetts, 02114
Rush University Medical Center - Dept of Pediatrics
Chicago, Illinois, 60612
Renal Disease Research Institute LLC
Dallas, Texas, 75204
Baylore University Medical Center
Dallas, Texas, 75246
Lysosomal and Rare Disorders Research and Treatment Center, Inc.
Fairfax, Virginia, 22030
University of Florida Clinical and Translational Science Institute, UF Clinical Research Center
Gainesville, Florida, 32610
Infusion Associates
Grand Rapids, Michigan, 49525
Greenwood Genetics 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, 92697
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
University of Pennsylvania - Dept of Medicine
Philadelphia, Pennsylvania, 19104
University of Utah - Division of Medical Genetics, Clinical Genetics Research
Salt Lake City, Utah, 84108
Canada5 sites
University of Calgary - Heritage Medical Research Clinic
Calgary, T2N 4Z6
London Health Sciences CTR, Victoria Hospital
London, N6A 5W9
Research Center, Hôpital du Sacré-Coeur de Montréal
Montreal, H4J 1C5
Vancouver General Hospital - Adult Metabolic Diseases Clinic
Vancouver, V5Z 1M9
Children's Hospital Research Institute of Manitoba
Winnipeg, R3E 3P4
Germany4 sites
Universitätsmedizin Berlin - Charité Campus Mitte
Berlin, 10117
SphinCS GmbH
Höchheim, 65239
Nephrologicum Markgräflerland MVZ GmbH
Müllheim, 79379
Universitätsklinikum Würzburg
Würzburg, 97080
Spain4 sites
Vall d'Hebron University Hospital - Unit of Inherited Metabolic Disorders and Rare Diseases
Barcelona, 08035
Hospital Universitari de Bellvitge / Nephrology Dpt
Barcelona, 08907
Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
Madrid, 28034
Hospital Quironsalud Zaragoza
Zaragoza, 50012
Poland3 sites
Clinic of Immunological Diseases and Blood Coagulability Cracow University Hospital
Krakow, 31-066
Narodowy Instytut Kardiologii Stefana kardynała Wyszyńskiego - Państwowy Instytut Badawczy
Warsaw, 04-628
The Children's Memorial Health Institute, Department of Pediatric, Nutrition and Metabolic Diseases
Warsaw, 04-730
United Kingdom3 sites
Royal Free London NHS Foundation Trust Lysosomal Storage Disorder Unit; Department of Hematology
London, NW3 2QG
National Hospital for Neurology and Neurosurgery
London, WC1N 3BG
Salford Hospital
Manchester, M6 8HD
Australia2 sites
Royal Melbourne Hospital - Department of Nephrology
Parkville, 3050
Royal Perth Hospital, Department of Nephrology
Perth, 6000
Belgium2 sites
University Hospital Ghent (UZ Ghent)
Ghent, 9000
University Hospital Gasthuisberg, Leuven (UZ Leuven)
Leuven, 3000
Austria1 site
Medizinische Universität 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, 1105 AZ
About this study
Official title: A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Read the detailed description
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
- Conditions
- Fabry Disease
- Being tested
- Lucerastat
- 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
Administered in hard gelatin capsules containing 250 mg of lucerastat.
| Study group | Role | What they receive |
|---|---|---|
| Lucerastat | Experimental | Dose will be based on subject's eGFR. Receives: Drug: Lucerastat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (AEs)
Measured: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Nov 2018
- Study start18 Dec 2018
- Primary completionAug 2029 (estimated)
- Study completionNov 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Not applicable
- Intervention model
- Single group
- Model description
- Multi-center, open-label, uncontrolled, single-arm, extension study
- Masking
- None (open label)
- Enrolment
- 107 participants (estimated)
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
- NCT03737214
- Sponsor's protocol ID
- ID-069A302
- Other IDs
- 2024-513884-20-00, 2018-002210-12, 2018
- Status last verified
- Aug 2026
- First submitted
- 6 Nov 2018
- Last updated
- 31 Aug 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
- Expanded access
- Available
Publications
Wanner C, Kimonis V, Politei J, Warnock DG, Uceyler N, Frey A, Cornelisse P, Hughes D. Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study. Mol Genet Metab Rep. 2022 Mar 26;31:100862. doi: 10.1016/j.ymgmr.2022.100862. eCollection 2022 Jun.
PubMed 35782623
Study at a glance
From the registry record, last updated by the sponsor on 31 Aug 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Open-label, Uncontrolled, Single-arm, Extension Study to Determine the Long-term Safety and Tolerability of Oral Lucerastat in Adult Subjects With Fabry Disease
- ClinicalTrials.gov ID
- NCT03737214
- Acronym / sponsor ID
- ID-069A302
- Conditions
- Fabry Disease
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 107 participants (estimated)
- Lead sponsor
- Idorsia Pharmaceuticals Ltd.Industry (pharma, biotech, devices)
- Study start
- 18 Dec 2018 (actual)
- Primary completion
- Aug 2029 (estimated)
- Study completion
- Nov 2029 (estimated)
- First posted
- 9 Nov 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
A study to determine the long-term safety and tolerability of oral lucerastat in adult subjects with Fabry disease. This study includes a sub-study evaluating kidney Gb3 inclusions (and other histologic lesions) in male participants with classic Fabry disease who have been treated for at least 2 years with lucerastat monotherapy in study ID-069A302.
Detailed description
Study ID-069A302 will continue at each site until lucerastat is commercially available in the respective country, or until all subjects have (prematurely) discontinued the trial, or until the sponsor terminates the study, whichever is earliest.
Note that, in Europe (local protocol amendment), the maximum individual study participation is up to Month 96.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- Multi-center, open-label, uncontrolled, single-arm, extension study
| Arm | Type | Receives |
|---|---|---|
| Lucerastat | Experimental | Drug: LucerastatDose will be based on subject's eGFR. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Signed ICF prior to any study-mandated procedure;
- Subject completed the 6-month, double-blind treatment period in study ID 069A301
- Woman of childbearing potential only if agreement 1) to follow a specified contraception scheme, 2) to undertake monthly urine pregnancy tests.
- Fertile male only if agreement 1) to use a condom, 2) to not father a child.
Key exclusion criteria (8 listed)
- Pregnant / planning to be become pregnant or lactating subject;
- Subject considered to be at high risk of developing clinical signs of organ involvement within the time period of the study, as per investigator judgment;
- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.
- In addition, the subject must not be enrolled in study ID-069A302 if at any time during study ID-069A301, one of the following criteria was met:
- Subject's eGFR per the Chronic Kidney Disease Epidemiology Collaboration creatinine equation < 15 mL/min/1.73 m2;
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 |
|---|---|---|
| LucerastatAdministered in hard gelatin capsules containing 250 mg of lucerastat. | Drug | Lucerastat |
Primary outcome
Treatment-emergent adverse events (AEs)
Time frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Aug 2026.
- Derived from this study: PubMed 35782623Wanner C, Kimonis V, Politei J, Warnock DG, Uceyler N, Frey A, Cornelisse P, Hughes D. Understanding and modifying Fabry disease: Rationale and design of a pivotal Phase 3 study and results from a patient-reported outcome validation study. Mol Genet Metab Rep. 2022 Mar 26;31:100862. doi: 10.1016/j.ymgmr.2022.100862. eCollection 2022 Jun.
- Individual participant dataSharing planned: No
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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.
42 sites in 13 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 42 sites in 13 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 4 inclusion and 8 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: under way, not looking for participants, as of the sponsor's last update on 31 Aug 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 42 sites in United States, Canada, Germany, Spain, Poland and 8 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. 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?
This study is still under way. Results are not available in the source record yet.
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?
Treatment-emergent adverse events (AEs) (time frame: From enrollment to Follow-up 1 (FU1) visit; duration: for up to 10 years including 1 month Follow-up). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03737214, which also lists 1 linked publication.
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 31 Aug 2026.