Lysosomal Acid Lipase Deficiency in Risk Groups
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Phase
—
Status
Terminated
Study type
Observational
Enrollment
25actual
Sites
2
Country
Russia
Every value on this page is a field the sponsor filed with ClinicalTrials.gov, reproduced. Dates are their own estimates, revised as a study runs, unless the registry marks them actual. sources →
Source: ClinicalTrials.gov, retrieved 2026-08-19
Why the sponsor stopped it
This decision is based on a reassessment of the current need for this study, impracticality of opening study sites in outpatient clinics due to administrative and legal constraints.
As filed on the registry record, in the sponsor's own words.
Dates
each axis at the precision it was filed, with the registry's own basis| Date | Filed | Basis | What it is |
|---|---|---|---|
| Start | 2026-02-25 | actual | When the study began enrolling |
| Primary completion | 2026-06-04 | actual | The date the last participant is measured for the primary outcome — the readout window |
| Study completion | 2026-06-04 | actual | The whole study's end, after follow-up |
| First posted | 2026-03-06 | actual | When this record first appeared on the registry |
| Results posted | — | When the sponsor posted results to the registry | |
| Record updated | 2026-07-08 | actual | The sponsor's own last edit to this record — every projection above is as current as this date |
What it studies
Condition
- Lysosomal Acid Lipase Deficiency
Primary outcome
what the primary-completion date above is the date OFTo estimate the proportion of patients with genetically confirmed LAL-D (defined by decreased LAL activity plus presence of biallelic pathogenic LIPA variants) among 12-month-to-18-year-old patients identified by predefined red flags.
measured Day 60 (Visit 2)
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