Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Conditions
Keywords
Batten's Disease, CLN2, TPP1 enzyme
Brief 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.
Interventions
DRUGCerliponase Alfa
Commercially available product provided to patient by participating clinic site.
DEVICEAdministration Kit
Commercially available administration kit provided to the patient by participating clinic site.
Sponsors
BioMarin Pharmaceutical
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
1. Diagnosed with CLN2 disease. 2. Currently receiving or plan to begin treatment with cerliponase alfa. 3. Written informed consent/assent obtained.
Exclusion criteria
1\. Currently receiving treatment in another investigational device or drug study.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| 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). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Hypersensitivity | 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 | 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). |
Countries
United States
Contacts
STUDY_DIRECTORMedical Director, MD
BioMarin Pharmaceutical
Outcome results
None listed