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Search / Trial NCT01633489

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.

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

0 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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