Cerliponase alfa
(190-501)
Trial Status Active, Not Recruiting
Condition
CLN2 Disease
Technology
- Enzyme
Title
Cerliponase Alfa Observational Study in the US
Study Description
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.
Primary Outcome Measures*
- To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis type 2
Select Inclusion Criteria*
- Diagnosed with CLN2 disease
- Currently receiving or plan to begin treatment with cerliponase alfa
- Parent/guardian provides informed written consent if required
Secondary Outcome Measures*
- Hypersensitivity
- Impact of severe adverse events on motor and language functions
Select Exclusion Criteria*
- Currently receiving treatment in another investigational device or drug study
* Additional measures/criteria may apply.
CLN2, late infantile neuronal ceroid lipofuscinosis type 2; TPP1, tripeptidyl peptidase 1
For reference and comprehensive trial information, visit ClinicalTrials.gov NCT04476862
Pre-IND
Phase 1
Phase 2
Phase 3
Phase 4