Status:
ACTIVE_NOT_RECRUITING
Cerliponase Alfa Observational Study in the US
Lead Sponsor:
BioMarin Pharmaceutical
Conditions:
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Eligibility:
All Genders
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 curre...
Eligibility Criteria
Inclusion
- Diagnosed with CLN2 disease.
- Currently receiving or plan to begin treatment with cerliponase alfa.
- Written informed consent/assent obtained.
Exclusion
- 1\. Currently receiving treatment in another investigational device or drug study.
Key Trial Info
Start Date :
August 19 2020
Trial Type :
OBSERVATIONAL
Allocation :
ESTIMATED
End Date :
August 31 2030
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT04476862
Start Date
August 19 2020
End Date
August 31 2030
Last Update
May 25 2025
Active Locations (16)
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1
Arkansas Children's Hospital
Little Rock, Arkansas, United States, 72205
2
Children's Hospital of Orange County
Orange, California, United States, 92868
3
Children's Hospital of Colorado
Aurora, Colorado, United States, 80045
4
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010