An Observational Disease and Clinical Outcomes Registry of Patients With Lysosomal Acid Lipase (LAL) Deficiency
Status: Recruiting
Location: See all (101) locations...
Study Type: Observational
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.
Participation Requirements• 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.
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Stanford
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Miramar
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Orlando
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Atlanta
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Chicago
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Boston
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Detroit
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Minneapolis
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Jackson
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Hackensack, Nj, 7601
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New York
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The Bronx
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Philadelphia
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Philadelphia
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Houston
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Houston
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Snyder
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Tacoma
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New Lambton Heights
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Ghent
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Campinas
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São Paulo
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São Paulo
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Sofia
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Edmonton
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Halifax
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London
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Québec
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Zagreb
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Bron
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Caen
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Clermont-ferrand
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Clermont-ferrand
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Dijon
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Grenoble
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La Rochelle
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Nancy
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Nantes
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Niort
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Paris
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Berlin
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Essen
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Mainz
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Munich
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Athens
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Ioannina
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Thessaloniki
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Dublin
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Haifa
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Jerusalem
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Jerusalem
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Petah Tikva
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Bari
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Bergamo
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Florence
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Genoa
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Milan
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Napoli
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Padua
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Turin
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Udine
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Aguascalientes
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Mexico City
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Zapopan
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Amsterdam
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Amsterdam
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Guimarães
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Lisbon
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Moscow
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Moscow
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Moscow
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Moscow
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Nizhny Novgorod
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Petersburg
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Riyadh
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Riyadh
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Ljubljana
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A Coruña
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Albacete
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Alicante
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Barakaldo
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Barcelona
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Barcelona
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Burela De Cabo
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Las Palmas
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Logroño
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Madrid
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Madrid
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Málaga
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Murcia
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Oviedo
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Reus
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Santiago De Compostela
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Valladolid
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Zaragoza
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Birmingham
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Cambridge
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London
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Manchester
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Salford
Primary
Alexion Pharmaceuticals, Inc. (Sponsor)
clinicaltrials@alexion.com
1-855-752-2356
Start Date: 2013-05-30
Estimated Completion Date: 2029-08-30
Target number of participants: 300
LAL Deficiency patients
Patients are those with a diagnosis of LAL Deficiency (living and deceased), irrespective of treatment status or treatment choice.
Leads: Alexion Pharmaceuticals, Inc.