Rare liver disease patients tracked in global effort to improve care

NCT ID NCT01633489

Recruiting now Knowledge-focused Sponsor: Alexion Pharmaceuticals, Inc. Source: ClinicalTrials.gov ↗

Summary

This study is creating a worldwide registry to collect long-term health information from people diagnosed with Lysosomal Acid Lipase (LAL) Deficiency, a rare genetic disorder that causes severe liver and heart problems. By tracking up to 300 patients over time, researchers aim to better understand how the disease progresses and how treatments work in real-world settings. Participation is voluntary and helps build knowledge to improve future care for this serious condition.

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes NO responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Contacts and locations

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Study contacts

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Phoenix, Arizona, 85016, United States
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Stanford, California, 94305, United States

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Miramar, Florida, 33025, United States
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Orlando, Florida, 32806, United States
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Atlanta, Georgia, 30322, United States
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Chicago, Illinois, 60611, United States

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Boston, Massachusetts, 02115, United States

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Detroit, Michigan, 48236, United States

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Minneapolis, Minnesota, 55404, United States
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Jackson, Mississippi, 39216, United States
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Hackensack, NJ, 7601, New Jersey, 07601, United States
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New York, New York, 10016, United States

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The Bronx, New York, 10467, United States

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Philadelphia, Pennsylvania, 19104-6061, United States
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Philadelphia, Pennsylvania, 19104, United States
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Philadelphia, Pennsylvania, 19107, United States
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Houston, Texas, 77030, United States
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Houston, Texas, 77030, United States

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Snyder, Texas, 79549, United States
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Tacoma, Washington, 98405, United States
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New Lambton Heights, New South Wales, 2305, Australia

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Ghent, East Flanders, 9000, Belgium

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Campinas, São Paulo, 13083-878, Brazil

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São Paulo, 04020-041, Brazil

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São Paulo, 04543-011, Brazil

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Sofia, Sofia-Grad, 1612, Bulgaria

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Edmonton, Alberta, T6G 2H7, Canada

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Halifax, Nova Scotia, B3H 1V8, Canada

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London, Ontario, NGA 5W9, Canada

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Québec, G1R 2J6, Canada

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Zagreb, 10000, Croatia

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Prague, Central Bohemia, 128 00, Czechia

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Olomouc, 779 00, Czechia

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Bron, Auvergne-Rhône-Alpes, 69500, France

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Clermont-Ferrand, Auvergne-Rhône-Alpes, 63003, France

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Clermont-Ferrand, Auvergne-Rhône-Alpes, 63100, France

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Grenoble, Auvergne-Rhône-Alpes, 38043, France

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Caen, Normandy, 14033, France

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La Rochelle, Nouvelle-Aquitaine, 17000, France

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Niort, Nouvelle-Aquitaine, 79021, France

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Nancy, Vandœuvre-lès-Nancy, 54500, France

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Dijon, 21000, France

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Nantes, 44093, France

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Paris, Île-de-France Region, 75743, France

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Munich, Bavaria, 81377, Germany

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Essen, North Rhine-Westphalia, 45122, Germany

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Mainz, Rhineland-Palatinate, 55131, Germany

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Berlin, 13353, Germany

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Athens, Attica, 115 27, Greece

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Ioannina, Epirus, 455 00, Greece

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Thessaloniki, Macedonia, 564 03, Greece

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Dublin, Leinster, D01 XD99, Ireland

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Petah Tikva, Central District, 49202, Israel

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Haifa, 3436212, Israel

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Jerusalem, 9103102, Israel

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Jerusalem, 91120, Israel

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Bari, Apulia, 70126, Italy

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Naples, Campania, 80131, Italy

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Udine, Friuli Venezia Giulia, 33100, Italy

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Genoa, Liguria, 16147, Italy

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Bergamo, Lombardy, 24127, Italy

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Milan, Lombardy, 20122, Italy

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Turin, Piedmont, 10126, Italy

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Florence, Tuscany, 50139, Italy

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Padua, Veneto, 35128, Italy

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Zapopan, Jalisco, 45116, Mexico

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Aguascalientes, 20230, Mexico

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Mexico City, 06720, Mexico

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Amsterdam, North Holland, 1105, Netherlands
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Amsterdam, North Holland, 1105, Netherlands

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Guimarães, Braga District, 4835, Portugal

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Lisbon, 1649, Portugal

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Petersburg, Leningradskaya Oblast', 194100, Russia

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Moscow, Moscow Oblast, 115446, Russia

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Moscow, Moscow Oblast, 119049, Russia

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Moscow, Moscow Oblast, 119192, Russia

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Moscow, Moscow Oblast, 119296, Russia

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Nizhny Novgorod, Nizhny Novgorod Oblast, 603950, Russia

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Riyadh, 11211, Saudi Arabia

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Riyadh, 11426, Saudi Arabia

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Ljubljana, 1111, Slovenia

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Santiago de Compostela, A Coruña, 15706, Spain
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Barakaldo, Biscay, 48903, Spain
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Logroño, La Rioja, 26006, Spain
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Burela de Cabo, Lugo, 27880, Spain
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Málaga, Málaga, 29010, Spain
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Oviedo, Principality of Asturias, 33011, Spain

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Reus, Tarragona, 43204, Spain
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A Coruña, 15006, Spain
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Albacete, 02006, Spain

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Alicante, 03203, Spain
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Barcelona, 08036, Spain
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Barcelona, 08305, Spain
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Las Palmas, 35019, Spain
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Madrid, 28046, Spain
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Madrid, 28914, Spain
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Murcia, 30120, Spain
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Valladolid, 47012, Spain

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Zaragoza, 50009, Spain

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Cambridge, Cambridgeshire, CB2 0QQ, United Kingdom

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London, Greater London, WC1N 3BG, United Kingdom

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Manchester, Greater Manchester, M13 9WL, United Kingdom

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Salford, Greater Manchester, M6 8HD, United Kingdom

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Birmingham, West Midlands, B4 6NH, United Kingdom

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Conditions

Explore the condition pages connected to this study.

ACID CHOLESTERYL ESTER HYDROLASE DEFICIENCY, TYPE 2 ACID LIPASE DEFICIENCY CHOLESTEROL ESTER STORAGE DISEASE LAL-DEFICIENCY LIPA DEFICIENCY LYSOSOMAL ACID LIPASE DEFICIENCY WOLMAN DISEASE