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Cerliponase Alfa Observational Study in the US

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NCT04476862 · readout in 1,473 d

Sponsored by BioMarin Pharmaceutical (industry) · BMRN — their whole pipeline →.

Phase
Status
Active, not recruiting
Study type
Observational
Enrollment
35estimated
Sites
16
Country
United States

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

Dates

each axis at the precision it was filed, with the registry's own basis
DateFiledBasisWhat it is
Start2020-08-19actualWhen the study began enrolling
Primary completion2030-08-31estimatedThe date the last participant is measured for the primary outcome — the readout window
Study completion2030-08-31estimatedThe whole study's end, after follow-up
First posted2020-07-20actualWhen this record first appeared on the registry
Results postedWhen the sponsor posted results to the registry
Record updated2026-06-30actualThe sponsor's own last edit to this record — every projection above is as current as this date

What it studies

Condition

  • Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Interventions

  • Drug: Cerliponase Alfa — also filed as Brineura
  • Device: Administration Kit

Primary outcome

what the primary-completion date above is the date OF
Safety surveillance of cerliponase alfa
measured 10 years

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