RecruitingNCT01633489

Lysosomal Acid Lipase (LAL) Deficiency Registry

An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency

Sponsor

Alexion Pharmaceuticals, Inc.

Enrollment

300 participants

Start Date

May 30, 2013

Study Type

OBSERVATIONAL

Conditions

Lysosomal Acid Lipase Deficiency Wolman Disease Cholesterol Ester Storage Disease Acid Cholesteryl Ester Hydrolase Deficiency, Type 2 Acid Lipase Deficiency LIPA Deficiency LAL-Deficiency

Summary

This is an observational, multi-center, international disease registry designed to collect longitudinal data and create a knowledge base that will be utilized to improve the care and treatment of patients with LAL Deficiency. Participation in the Registry by both physicians and patients is voluntary.

Eligibility

Plain Language Summary

Simplified for easier understanding

This is a patient registry that collects long-term health data from people with Lysosomal Acid Lipase (LAL) deficiency — a rare genetic condition where fats build up in organs including the liver and spleen — to better understand the disease and monitor treatment outcomes. You may be eligible if: - You have a confirmed diagnosis of LAL deficiency - You are able to provide informed consent (or consent has been waived by your ethics board) - You have completed participation in an Alexion-sponsored clinical trial for sebelipase alfa You may NOT be eligible if: - You are currently participating in an active Alexion-sponsored clinical trial Talk to your doctor to see if this trial is right for you.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Locations(104)

Clinical Trial Site

Phoenix, Arizona, United States

Clinical Trial Site

Stanford, California, United States

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Miramar, Florida, United States

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Orlando, Florida, United States

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Atlanta, Georgia, United States

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Chicago, Illinois, United States

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Boston, Massachusetts, United States

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Detroit, Michigan, United States

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Minneapolis, Minnesota, United States

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Jackson, Mississippi, United States

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Hackensack, NJ, 7601, New Jersey, United States

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New York, New York, United States

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The Bronx, New York, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Philadelphia, Pennsylvania, United States

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Houston, Texas, United States

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Houston, Texas, United States

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Snyder, Texas, United States

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Tacoma, Washington, United States

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New Lambton Heights, New South Wales, Australia

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Ghent, East Flanders, Belgium

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Campinas, São Paulo, Brazil

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São Paulo, Brazil

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São Paulo, Brazil

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Sofia, Sofia-Grad, Bulgaria

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Edmonton, Alberta, Canada

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Halifax, Nova Scotia, Canada

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London, Ontario, Canada

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Québec, Canada

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Zagreb, Croatia

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Prague, Central Bohemia, Czechia

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Olomouc, Czechia

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Bron, Auvergne-Rhône-Alpes, France

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Clermont-Ferrand, Auvergne-Rhône-Alpes, France

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Clermont-Ferrand, Auvergne-Rhône-Alpes, France

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Grenoble, Auvergne-Rhône-Alpes, France

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La Rochelle, New Aquitaine, France

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Niort, New Aquitaine, France

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Caen, Normandy, France

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Nancy, Vandœuvre-lès-Nancy, France

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Dijon, France

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Nantes, France

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Paris, Île-de-France Region, France

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Munich, Bavaria, Germany

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Essen, North Rhine-Westphalia, Germany

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Mainz, Rhineland-Palatinate, Germany

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Berlin, Germany

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Athens, Attica, Greece

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Ioannina, Epirus, Greece

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Thessaloniki, Macedonia, Greece

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Dublin, Leinster, Ireland

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Petah Tikva, Central District, Israel

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Haifa, Israel

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Jerusalem, Israel

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Jerusalem, Israel

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Bari, Apulia, Italy

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Naples, Campania, Italy

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Udine, Friuli Venezia Giulia, Italy

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Genoa, Liguria, Italy

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Bergamo, Lombardy, Italy

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Milan, Lombardy, Italy

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Turin, Piedmont, Italy

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Florence, Tuscany, Italy

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Padova, Veneto, Italy

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Zapopan, Jalisco, Mexico

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Aguascalientes, Mexico

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Mexico City, Mexico

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Amsterdam, North Holland, Netherlands

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Amsterdam, North Holland, Netherlands

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Guimarães, Braga District, Portugal

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Lisbon, Portugal

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Petersburg, Leningradskaya Oblast', Russia

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Moscow, Moscow Oblast, Russia

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Moscow, Moscow Oblast, Russia

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Moscow, Moscow Oblast, Russia

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Moscow, Moscow Oblast, Russia

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Nizhny Novgorod, Nizhny Novgorod Oblast, Russia

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Riyadh, Saudi Arabia

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Riyadh, Saudi Arabia

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Ljubljana, Slovenia

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Santiago de Compostela, A Coruña, Spain

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Barakaldo, Biscay, Spain

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Logroño, La Rioja, Spain

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Burela de Cabo, Lugo, Spain

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Málaga, Málaga, Spain

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Oviedo, Principality of Asturias, Spain

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Reus, Tarragona, Spain

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A Coruña, Spain

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Albacete, Spain

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Alicante, Spain

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Barcelona, Spain

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Barcelona, Spain

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Las Palmas, Spain

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Madrid, Spain

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Madrid, Spain

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Murcia, Spain

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Valladolid, Spain

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Zaragoza, Spain

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Cambridge, Cambridgeshire, United Kingdom

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London, Greater London, United Kingdom

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Manchester, Greater Manchester, United Kingdom

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Salford, Greater Manchester, United Kingdom

Clinical Trial Site

Birmingham, West Midlands, United Kingdom

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NCT01633489

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Lysosomal Acid Lipase (LAL) Deficiency Registry

Conditions

Summary

Eligibility

Plain Language Summary

Locations(104)

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