Cerliponase Alfa Observational Study in the US

Condition(s) studied

Investigational drug(s) / intervention(s)

Cerliponase Alfa: Commercially available product provided to patient by participating clinic site.

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

Study summary

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.

Eligibility

Primary outcome measure(s)

• Safety surveillance of cerliponase alfa — 10 years
  To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).

Trial sites (16)