Health TrialsbyHealthPanel

Lysosomal Acid Lipase (LAL) Deficiency Registry

ConditionsLysosomal Acid Lipase DeficiencyCholesterol Ester Storage DiseaseWolman DiseaseAcid Cholesteryl Ester Hydrolase Deficiency, Type 2Acid Lipase DeficiencyLIPA DeficiencyLAL-Deficiency
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
AgeNot listed
SponsorAlexion Pharmaceuticals, Inc.

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

None

Disqualifiers

None

Trial population

All patients with a diagnosis of LAL Deficiency.

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

Not listed

Trial groups

300 Participants
are grouped into 1 trial group
Group A: LAL Deficiency patients

Trial outcomes

Primary outcomes

1

Understanding of the variability, progression, identification and natural history of LAL Deficiency.

Time frame
Ongoing

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Alexion Pharmaceuticals, Inc.

Lead sponsor

Source ClinicalTrials.gov
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