Lysosomal Acid Lipase (LAL) Deficiency Registry
Launched by ALEXION PHARMACEUTICALS, INC. · Jul 3, 2012
Trial Information
Current as of July 09, 2025
Recruiting
Keywords
ClinConnect Summary
The Lysosomal Acid Lipase (LAL) Deficiency Registry is a research study aimed at better understanding and improving the care for patients with LAL Deficiency and related conditions. This study is observational, meaning it focuses on collecting information over time from patients and healthcare providers to create a valuable database that can help guide treatment and care decisions. Participation in this registry is voluntary for both patients and doctors.
To be eligible for this study, participants must have a confirmed diagnosis of LAL Deficiency. Patients who are currently involved in another clinical trial sponsored by Alexion cannot join, but those who have completed such trials can enroll. By joining the registry, participants will help contribute to important research that may lead to better treatment options in the future. Additionally, enrolling in this registry does not prevent patients from taking part in other clinical trials later on.
Gender
ALL
Eligibility criteria
- • Patients must have a confirmed diagnosis of LAL Deficiency. An Informed Consent and Authorization must be obtained prior to patient enrollment where required under applicable laws and regulations, or a waiver must be obtained by the Institutional Review Board/Independent Ethics Committee.
- • Patients cannot be currently participating in an Alexion-sponsored clinical trial. Patients who have concluded participation in an Alexion-sponsored sebelipase alfa clinical trial are eligible to enroll in this Registry, and enrollment in the Registry will not exclude a patient from enrolling in a future clinical trial.
About Alexion Pharmaceuticals, Inc.
Alexion Pharmaceuticals, Inc. is a global biopharmaceutical company dedicated to the discovery, development, and commercialization of innovative therapies for patients with rare and complex diseases. With a strong focus on addressing unmet medical needs, Alexion leverages advanced science and technology to create transformative treatments that improve the quality of life for patients suffering from conditions such as complement-mediated disorders, neurology, hematology, and metabolic diseases. Committed to patient-centric approaches, Alexion collaborates with healthcare professionals, patients, and advocacy groups to drive clinical research and foster advancements in rare disease treatment.
Contacts
Jennifer Cobb
Immunology at National Institute of Allergy and Infectious Diseases (NIAID)
Locations
Los Angeles, California, United States
Atlanta, Georgia, United States
Hackensack, New Jersey, United States
Chicago, Illinois, United States
Philadelphia, Pennsylvania, United States
London, Ontario, Canada
Jerusalem, , Israel
Warsaw, , Poland
Barcelona, , Spain
Madrid, , Spain
London, , United Kingdom
Minneapolis, Minnesota, United States
New York, New York, United States
Dallas, Texas, United States
Berlin, , Germany
Amsterdam, , Netherlands
Zaragoza, , Spain
Chicago, Illinois, United States
Phoenix, Arizona, United States
Orlando, Florida, United States
Tacoma, Washington, United States
Genova, , Italy
Manchester, , United Kingdom
Essen, , Germany
Dublin, , Ireland
Mexico City, , Mexico
Houston, Texas, United States
Paris, , France
Detroit, Michigan, United States
Boston, Massachusetts, United States
Olomouc, , Czechia
Prague, , Czechia
Stanford, California, United States
Lyon, , France
Nice, , France
Birmingham, , United Kingdom
Edmonton, , Canada
Bronx, New York, United States
Campinas, , Brazil
Nantes, , France
Munich, , Germany
Providence, Rhode Island, United States
Nancy, , France
Athens, , Greece
Petah Tikva, , Israel
Cambridge, , United Kingdom
Milano, , Italy
Fairfax, Virginia, United States
Zagreb, , Croatia
Valladolid, , Spain
Genoa, , Italy
Ghent, , Belgium
Clermont Ferrand, , France
Aguascalientes, , Mexico
Zapopan, , Mexico
Copenhagen, , Denmark
Salford, , United Kingdom
Halifax, , Canada
Ljubljana, , Slovenia
Riyadh, , Saudi Arabia
Oviedo, , Spain
Bron, , France
Albacete, , Spain
La Tronche, , France
Jeddah, , Saudi Arabia
Lisbon, , Portugal
New Lambton Heights, , Australia
São Paulo, , Brazil
Turin, , Italy
Asturias, , Spain
Hochheim, , Germany
Chicago, Illinois, United States
Houston, Texas, United States
Ribeirão Preto, , Brazil
Minneapolis, Minnesota, United States
New York, New York, United States
Woluwe Saint Lambert, , Belgium
Guimarães, , Portugal
Murcia, , Spain
Barcelona, , Spain
Atlanta, Georgia, United States
Boston, Massachusetts, United States
Bronx, New York, United States
Napoli, Campania, Italy
Detroit, Michigan, United States
Clermont Ferrand, , France
Jerusalem, , Israel
Albacete, , Spain
Santiago De Compostela, , Spain
New York, New York, United States
Orlando, Florida, United States
Madrid, , Spain
Murcia, , Spain
Bron, , France
Stanford, California, United States
Jackson, Mississippi, United States
Nantes, , France
Phoenix, Arizona, United States
Minneapolis, Minnesota, United States
Malaga, , Spain
Barakaldo, Biscay, Spain
Grenoble, , France
Tacoma, Washington, United States
Valladolid, , Spain
Dijon, , France
Milano, Lombardy, Italy
Zaragoza, , Spain
Berlin, , Germany
Oviedo, Asturias, Spain
Zagreb, , Croatia
Udine, Friuli Venezia Giulia, Italy
Nancy, , France
Andalusia, , Spain
Aragon, , Spain
Basque Country, , Spain
Canary Islands, , Spain
Castile And León, , Spain
Castile La Mancha, , Spain
Catalonia, , Spain
Catalonia, , Spain
Catalonia, , Spain
Galicia, , Spain
Galicia, , Spain
Galicia, , Spain
La Rioja, , Spain
Valencia, , Spain
Miramar, Florida, United States
Hackensack, Nj, 7601, New Jersey, United States
Philadelphia, Pennsylvania, United States
Snyder, Texas, United States
New Lambton Heights, New South Wales, Australia
Ghent, East Flanders, Belgium
Campinas, São Paulo, Brazil
São Paulo, , Brazil
Sofia, Sofia City, Bulgaria
Edmonton, Alberta, Canada
Halifax, Nova Scotia, Canada
London, Ontario, Canada
Quebec, , Canada
Prague, Central Bohemia, Czechia
Olomouc, , Czechia
Bron, Auvergne Rhône Alpes, France
Clermont Ferrand, Auvergne Rhône Alpes, France
Grenoble, Auvergne Rhône Alpes, France
Caen, Normandy, France
La Rochelle, Nouvelle Aquitaine, France
Niort, Nouvelle Aquitaine, France
Nancy, Vandœuvre Lès Nancy, France
Paris, île De France, France
Munich, Bavaria, Germany
Essen, North Rhine Westphalia, Germany
Mainz, Rhineland Palatinate, Germany
Athens, Attica, Greece
Ioannina, Epirus, Greece
Thessaloniki, Macedonia, Greece
Dublin, Leinster, Ireland
Petah Tikva, Central, Israel
Haifa, , Israel
Bari, Apulia, Italy
Genova, Liguria, Italy
Bergamo, Lombardy, Italy
Turin, Piedmont, Italy
Florence, Tuscany, Italy
Padova, Veneto, Italy
Zapopan, Jalisco, Mexico
Aguascalientes, , Mexico
Mexico City, , Mexico
Amsterdam, North Holland, Netherlands
Guimarães, Braga, Portugal
Lisbon, , Portugal
Petersburg, Leningrad Oblast, Russian Federation
Moscow, Moscow Oblast, Russian Federation
Nizhny Novgorod, Nizhny Novgorod Oblast, Russian Federation
Riyadh, , Saudi Arabia
Ljubljana, , Slovenia
Santiago De Compostela, A Coruña, Spain
Logroño, La Rioja, Spain
Burela, Lugo, Spain
Malaga, Málaga, Spain
Reus, Tarragona, Spain
A Coruña, , Spain
Alicante, , Spain
Las Palmas, , Spain
Cambridge, Cambridgeshire, United Kingdom
London, Greater London, United Kingdom
Manchester, Greater Manchester, United Kingdom
Salford, Greater Manchester, United Kingdom
Birmingham, West Midlands, United Kingdom
Campinas, , Brazil
Paris, , France
Patients applied
Trial Officials
Alexion Pharmaceuticals
Study Director
Sponsor GmbH
Timeline
First submit
Trial launched
Trial updated
Estimated completion
Not reported
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