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 currently being treated with cerliponase alfa. Patients receiving or expected to receive cerliponase alfa within 60 days of signing the informed consent form (ICF) may be eligible to enroll in the study, assuming all regulatory requirements for sites that have agreed to participate and protocol inclusion criteria are met. Data may be collected for all or some of the assessments as outlined in the protocol, dependent upon the clinic's and/or individual patient's standard of care.

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Heredodegenerative Disorders, Nervous System Lipid Metabolism Disorders Lipid Metabolism, Inborn Errors Lipidoses Metabolic Diseases Metabolism, Inborn Errors Nervous System Diseases Neurodegenerative Diseases Neuronal Ceroid-Lipofuscinoses Nutritional and Metabolic Diseases

Sex eligibility

All sexes

Study type

Observational

Primary location

Arkansas Children's Hospital, Little Rock, Arkansas, United States

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

Diagnosed with CLN2 disease.
Currently receiving or plan to begin treatment with cerliponase alfa.
Written informed consent/assent obtained.

Exclusion criteria

Currently receiving treatment in another investigational device or drug study.

Treatment and study plan

Cerliponase Alfa

Drug

Commercially available product provided to patient by participating clinic site.

Other names: Brineura

Administration Kit

Device

Commercially available administration kit provided to the patient by participating clinic site.

Primary outcomes

Safety surveillance of cerliponase alfa

Time frame: 10 years

To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).

Secondary outcomes

Hypersensitivity

Time frame: 10 years

To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.
Severe SAE impact on patient's motor and language functions

Time frame: 10 years

To evaluate the effects of Grade III or higher serious adverse events (SAEs) on patient performance on the CLN2 clinical rating scale (motor and language domains).

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study title

Cerliponase Alfa Observational Study

Important dates

Study start: 2020
Primary completion: 2030
Study completion: 2030
First posted: Jul 20, 2020
Registry last updated: Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Published trials that share one or more normalized conditions with this study.

Completed