Actively Recruiting
Lysosomal Acid Lipase Deficiency in Risk Groups
Led by AstraZeneca · Updated on 2026-04-14
1200
Participants Needed
2
Research Sites
122 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
A multicenter real-world observational study of the prevalence, diagnostic pathways, and clinical characteristics of lysosomal acid lipase deficiency in pediatric and adolescent risk groups in the Russian Federation (HELIOS)
CONDITIONS
Official Title
Lysosomal Acid Lipase Deficiency in Risk Groups
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age 12 months to 18 years (infantile form is not included)
- Patients not previously tested for lysosomal acid lipase deficiency (LAL-D)
- Presence of at least one major criterion: unexplained liver or spleen enlargement lasting 3 months or more, persistent elevated liver enzymes (ALT or AST ≥ 1.5 times upper limit of normal) after excluding common causes, or abnormal cholesterol levels including high total cholesterol, high LDL-C, and/or low HDL-C
- Presence of at least two minor criteria such as chronic diarrhea, abdominal pain or bloating, loss of appetite, nausea or vomiting, heartburn, weight loss or slowed growth, fatigue, recurrent mouth ulcers, spleen enlargement if not counted as major, anemia or low platelets, liver fat or fibrosis by imaging, poor response to lipid-lowering therapy, or family history of FH-like dyslipidemia without typical genetic markers
- Signed informed consent from parent(s)/legal guardian(s) and the child, if applicable
You will not qualify if you...
- Confirmed alternative cause fully explaining liver disease or dyslipidemia, such as hepatitis A, B, or C, or autoimmune hepatitis, without suspicion of LAL-D
- Diagnosis of Wolman disease
- Long-term use of systemic corticosteroids defined as continuous oral or parenteral use for 14 days or more in the last 6 months
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 2 locations
1
Research site
Nizhny Novgorod, Russia
Actively Recruiting
2
Research Site
Saint Petersburg, Russia
Actively Recruiting
Research Team
A
AstraZeneca Clinical Study Information Center
CONTACT
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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