LAL Deficiency Registry
Research type
Research Database
Full title
An Observational Disease and Clinical Outcomes Registry of Patients wi
IRAS ID
121489
Contact name
Florian Abel
Contact email
Research summary
An Observational Disease and Clinical Outcomes Registry of Patients with Lysosomal Acid Lipase (LAL) Deficiency
REC name
HSC REC B
REC reference
13/NI/0129
Date of REC Opinion
5 Dec 2013
REC opinion
Further Information Favourable Opinion
Data collection arrangements
LAL Deficiency is a very rare disorder with limited understanding of the signs, symptoms, and progression. There is no planned end date for the Registry. Prospective and retrospective data will be collected. Demographic, socioeconomic, clinical, and treatment data, or other data deemed relevant to the management of patients will be submitted to the Registry based on routine clinical practice, as determined by the patient’s physician; there is no visit schedule set by the Registry.
No directly identifiable data will be collected. All patients enrolled in the Registry will be issued a unique 9-digit identification (ID) number that will be used and only the patient's physician will have access to the patient's identity. Data will be submitted to the Registry on electronic case report forms.The Registry system is hosted and maintained in highly secure, certified facilities that have been audited and approved for health data privacy and security regulations.
Research programme
This is an observational, multi-center, international 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 and carriers of the disorder. Data may be used to respond to requests from national regulatory or reimbursement authorities and agencies, as well as from physicians interested in learning more about LAL Deficiency (e.g. the signs, symptoms, or progression of the disease). Data may be analyzed and reported based on requests from participating physicians to answer research questions they have about LAL Deficiency or its management(subject to approval by a governing Board of expert physicians overseeing the program) in order to improve the management and care of patients with the disorder.
Research database title
An Observational Disease and Clinical Outcomes Registry of Patients with Lysosomal Acid Lipase (LAL) Deficiency
Establishment organisation
Alexion Pharmaceuticals, Inc.
Establishment organisation address
100 College Street
New Haven, CT
USA
06510