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.
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.