Cerliponase Alfa Observational Study in the US

This is a multicenter, observational study for patients with a confirmed diagnosis of neuronal ceroid lipofuscinosis type 2 (CLN2 disease), also known as TPP1 deficiency, who intend to be or are curre...

Lead sponsor: BioMarin Pharmaceutical•16 U.S. locations•View source on ClinicalTrials.gov

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Data from ClinicalTrials.govLast updated on ClinicalTrials.gov: January 14, 2026Terms

Trial snapshot

StatusACTIVE_NOT_RECRUITING

Phase—

Enrollment35

Start dateAug 2020

Last updatedJan 14, 2026

Condition

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2

Locations shown

Arkansas Children's Hospital

Little Rock, Arkansas

Children's Hospital of Orange County

Orange, California

Children's Hospital of Colorado

Aurora, Colorado

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Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: January 14, 2026Data synced to Clareo: May 25, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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