Clinical trial • Phase III • Rare Disease|Endocrinology
LUCERASTAT for Fabry disease
Phase III trial of LUCERASTAT for Fabry disease. open-label, none/not specified-controlled. 58 participants.
Overview
- Trial Therapeutic Area
- Rare Disease|Endocrinology
- Trial Disease
- Fabry disease
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 07-10-2024
- First CTIS Authorization Date
- 05-11-2024
Trial design
open-label, none/not specified-controlled Phase III trial in Austria, Belgium, Poland and others.
- Open Label
- Yes
- Comparator
- None/Not specified
- Target Sample Size
- 58
- Trial Duration For Participant
- 2910
Eligibility
Recruits 58 No vulnerable populations selected. Adults only (protocol and public title state 'adult subjects'); consent is required: 'Signed and dated ICF prior to any study-mandated procedure;'. Re-consent procedures for later stages are defined (Stage 2/3/4 Reconsent ICFs available). No assent/parental consent described..
- Pregnancy Exclusion
- Pregnant / planning to become pregnant or lactating subject;
- Vulnerable Population
- No vulnerable populations selected. Adults only (protocol and public title state 'adult subjects'); consent is required: 'Signed and dated ICF prior to any study-mandated procedure;'. Re-consent procedures for later stages are defined (Stage 2/3/4 Reconsent ICFs available). No assent/parental consent described.
Inclusion criteria
- {"criterion_text":"- Signed and dated ICF prior to any study-mandated procedure;\n- Subject completed the 6-month, double-blind treatment period in study ID 069A301;"}
Exclusion criteria
- {"criterion_text":"- Pregnant / planning to become pregnant or lactating subject;\n- 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;\n- Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results as per investigator judgment.\n- If at any time during study ID-069A301, the following criteria was met: Subject’s eGFR per the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) creatinine equation < 15 mL/min/1.73 m2.\n- If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of acute kidney injury Common Terminology Criteria for Adverse Event (CTCAE) grade 2 or above;\n- If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of stroke CTCAE grade 3 or above;\n- If at any time during study ID-069A301, the following criteria was met: Subject experienced an event of heart failure leading to in-patient hospitalization or prolongation of ongoing hospitalization."}
Endpoints
Primary endpoints
- {"endpoint_text":"- Treatment-emergent AEs and SAEs","definition_or_measurement_approach":""}
Recruitment
- Planned Sample Size
- 58
- Recruitment Window Months
- 129
- Consent Approach
- Signed and dated ICF prior to any study-mandated procedure. Re-consent for Stage 2/3/4 as applicable (multiple Stage-specific ICFs listed). Subject information and informed consent forms available in multiple language versions (DE, EN, FR, NL, PL, ES, NO as evidenced by ICF document titles). Consent is provided by the adult subject; no assent or parental consent procedures described.
Geography
- Total Number Of Sites
- 15
- Total Number Of Participants
- 45
Austria
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 16-06-2025
- Processing Time Days
- 242
- Number Of Sites
- 1
- Number Of Participants
- 1
Sites
- Site Name
- Medical University Of Vienna
- Department Name
- Universitätsklinik für Innere Medizin III, Klinische Abteilung für Nephrologie und Dialyse
- Contact Person Name
- Gere Sunder-Plassmann
- Contact Person Email
- Gere.Sunder-Plassmann@meduniwien.ac.at
Belgium
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 03-06-2025
- Processing Time Days
- 229
- Number Of Sites
- 2
- Number Of Participants
- 2
Sites
- Site Name
- UZ Leuven
- Department Name
- The Department of Gastro-enterology – Hepatology
- Contact Person Name
- David Cassiman
- Contact Person Email
- david.cassiman@uzleuven.be
- Site Name
- Universiteit Gent
- Department Name
- Hospital - Universitair Gent __Neurology Department
- Contact Person Name
- Dimitri Hemelsoet
- Contact Person Email
- Dimitri.Hemelsoet@uzgent.be
Poland
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 16-06-2025
- Processing Time Days
- 242
- Number Of Sites
- 2
- Number Of Participants
- 13
Sites
- Site Name
- Instytut Pomnik Centrum Zdrowia Dziecka
- Department Name
- Department of Pediatric, Nutrition and Metabolic Diseases
- Contact Person Name
- Anna Tylki-Szymanska
- Contact Person Email
- A.Tylki@IPCZD.PL
- Site Name
- Narodowy Instytut Kardiologii Stefana Kardynala Wyszynskiego Panstwowy Instytut Badawczy
- Department Name
- Klinika Kardiologii i Angiologii Interwencyjnej
- Contact Person Name
- Mariusz Klopotowski
- Contact Person Email
- mklopotowski@ikard.pl
Germany
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 03-06-2025
- Processing Time Days
- 229
- Number Of Sites
- 4
- Number Of Participants
- 13
Sites
- Site Name
- Charite Universitaetsmedizin Berlin KöR
- Department Name
- Abteilung Nephrologie und Internistische Intensivmedizin
- Contact Person Name
- Sima Canaan-Kühl
- Contact Person Email
- Sima.Canaan@charite.de
- Site Name
- Nephrologicum Markgräflerland MVZ GmbH
- Department Name
- Nephrologicum Markgräflerland MVZ GmbH
- Contact Person Name
- Markus Cybulla
- Contact Person Email
- markus.cybulla@nephrologicum-mgl.de
- Site Name
- Universitaetsklinikum Wuerzburg AöR
- Department Name
- FAZIT – Fabry-Zentrum für interdisziplinäre Therapie
- Contact Person Name
- Peter Nordbeck
- Contact Person Email
- nordbeck_p@ukw.de
- Site Name
- SphinCS GmbH
- Department Name
- SphinCS GmbH
- Contact Person Name
- Eugen Mengel
- Contact Person Email
- eugen.mengel@sphincs.de
France
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 02-06-2025
- Processing Time Days
- 228
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Raymond-Poincare Hospital
- Department Name
- Université de Versailles – Saint Quentin en Yvelines Service de Génétique Médicale
- Contact Person Name
- Dominique P. Germain
- Contact Person Email
- dominique.germain@uvsq.fr
Norway
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 02-06-2025
- Processing Time Days
- 228
- Number Of Sites
- 1
- Number Of Participants
- 2
Sites
- Site Name
- Helse Bergen HF
- Department Name
- Haukeland Universitetssykehus Department of Clinical Medicine
- Contact Person Name
- Camilla Tøndel
- Contact Person Email
- camilla.tondel@helse-bergen.no
Spain
- Earliest CTIS Part Ii Submission Date
- 17-10-2024
- Latest Decision Or Authorization Date
- 02-06-2025
- Processing Time Days
- 228
- Number Of Sites
- 4
- Number Of Participants
- 12
Sites
- Site Name
- Bellvitge University Hospital
- Department Name
- Hospital Universitari de Bellvitge / Nephrology Dpt
- Contact Person Name
- Juan Torras Ambros
- Contact Person Email
- jtorras@bellvitgehospital.cat
- Site Name
- Fundacio Hospital Universitari Vall D’Hebron Institut De Recerca
- Department Name
- Unit of Inherited Metabolic Disorders and Rare Diseases
- Contact Person Name
- Maria Camprodon Gomez
- Contact Person Email
- maria.camprodon@vallhebron.cat
- Site Name
- Hospital Quironsalud Zaragoza
- Department Name
- Quironsalud Floresta.
- Contact Person Name
- Pilar Giraldo Castellano
- Contact Person Email
- giraldocastellano@gmail.com
- Site Name
- Hospital Universitario Ramon Y Cajal
- Department Name
- Hospital Universitario Ramon y Cajal. Servicio de Medicina Interna
- Contact Person Name
- Monica Lopez Rodriguez
- Contact Person Email
- monicaa.lopez@salud.madrid.org
Sponsor
Primary sponsor
- Full Name
- Idorsia Pharmaceuticals Ltd.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- Switzerland
Contract research organisations
- Name
- Almac Clinical Technologies LLC
- Name
- Bioclinica Inc.
- Responsibilities
- ECG analysis/ review, Echocardiography
- Name
- Iqvia Laboratories Limited
- Responsibilities
- Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry
- Name
- Precision for Medicine (HU) Kft.
- Responsibilities
- Contract negotiations, local translations and adaptations, contact for contracts with hospitals, other parties as required for BE, FR, PL, ES
- Name
- Mde Services Group Limited
- Responsibilities
- Patient reimbursement and travel planning
- Name
- Eresearchtechnology Inc.
- Responsibilities
- ePRO
Third parties
- {"country":"United States","full_name":"Indica Labs Inc.","duties_or_roles":"Scoring system provider, coordinate review done by the pathologist, perform QC of data.","organisation_type":"Pharmaceutical company"}
- {"country":"United States","full_name":"Almac Clinical Technologies LLC","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Finland","full_name":"Oriola Finland Oy","duties_or_roles":"contract set-up for NO","organisation_type":"Pharmaceutical company"}
- {"country":"Italy","full_name":"Valos S.r.l. In Forma Abbreviata Va Los S.r.l.","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
- {"country":"Germany","full_name":"Universitaetsklinikum Muenster AöR","duties_or_roles":"anti-ERT antibodies assessment","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Switzerland","full_name":"Swiss BioQuant AG","duties_or_roles":"biomarker analysis","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ePRO","organisation_type":"Pharmaceutical company"}
- {"country":"Switzerland","full_name":"AnaPath Services GmbH","duties_or_roles":"Renal biopsy high magnification imaging, Processing of renal biopsies samples (processing, sectioning, and staining)","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Aixial UK Limited","duties_or_roles":"","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United States","full_name":"Bioclinica Inc.","duties_or_roles":"ECG analysis/ review, Echocardiography","organisation_type":"Laboratory/Research/Testing facility"}
- {"country":"United Kingdom","full_name":"Mde Services Group Limited","duties_or_roles":"Patient reimbursement and travel planning","organisation_type":"Non-Pharmaceutical company"}
- {"country":"Germany","full_name":"Flying Study Team GbR","duties_or_roles":"for Austria and Germany","organisation_type":"Hospital/Clinic/Other health care facility"}
- {"country":"Hungary","full_name":"Precision for Medicine (HU) Kft.","duties_or_roles":"Contract negotiations, local translations and adaptations, contact for contracts with hospitals, other parties as required for BE, FR, PL, ES","organisation_type":"Pharmaceutical company"}
- {"country":"United Kingdom","full_name":"Iqvia Laboratories Limited","duties_or_roles":"Clinical chemistry, Clinical haematology, Serology/ endocrinology, Analytical chemistry","organisation_type":"Non-Pharmaceutical company"}
Investigational products
- Investigational Product Name
- Lucerastat
- Active Substance
- LUCERASTAT
- Modality
- Small molecule
- Routes Of Administration
- ORAL USE
- Route
- Oral
- Authorisation Status
- Authorised
- Orphan Designation
- Yes
- Maximum Dose
- 2000 mg
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