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The objective of the LAL-D Registry is to better understand the natural history of LAL-D and evaluate long term treatment outcomes over an extended period using uniform methodology to provide information to 1,2
- Further understand the disease, its progression and any associated complications.1,2
- Evaluate the long-term effectiveness of therapeutic and supportive interventions.1,2
- Improve care through evidence-based patient management.1
Any patient with a diagnosis of LAL-D, may be included in the LAL-D Registry, irrespective of treatment status or treatment choice. Living and deceased patients can be included.1
Data Entry
Participation in the LAL-D Registry requires approval from your Institutional Review Board (IRB) or Ethics Committee (EC). It is at the discretion of the IRB/EC to determine whether a full review is warranted, given that no experimental or interventional procedures are included in the protocol. Appropriate patient authorizations must be obtained prior to submission of data into the Registry.
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Data are submitted to the Registry on electronic case report forms (eCRFs) via a secure web-based system, and are reviewed for quality prior to inclusion in aggregate analyses. Patients are identifiable only to the patient’s physician using a unique ID code: no names or data that directly identify the patients are collected. Once the data have been reviewed, they are available for inclusion in aggregated data analyses.
References
1. NCT01633489. ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/ NCT01633489.
2. Balwani M, Balistreri W, and D'Antiga, et al. Clinical manifestations of lysosomal acid lipase deficiency (LALD):
The international LAL-D registry. Mol. Genet. Metab. 2019;129(2):S26.