Cerliponase Alfa Observational Study in the US
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|ClinicalTrials.gov Identifier: NCT04476862|
Recruitment Status : Recruiting
First Posted : July 20, 2020
Last Update Posted : February 11, 2021
|Condition or disease||Intervention/treatment|
|Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2||Drug: Cerliponase Alfa Device: Administration Kit|
|Study Type :||Observational|
|Estimated Enrollment :||35 participants|
|Official Title:||Cerliponase Alfa Observational Study|
|Actual Study Start Date :||August 19, 2020|
|Estimated Primary Completion Date :||August 24, 2030|
|Estimated Study Completion Date :||August 24, 2030|
Cerliponase alfa patients
Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.
Drug: Cerliponase Alfa
Commercially available product provided to patient by participating clinic site.
Other Name: Brineura
Device: Administration Kit
Commercially available administration kit provided to the patient by participating clinic site.
- 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).
- 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).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04476862
|Contact: Trial Specialistfirstname.lastname@example.org|
|United States, Arkansas|
|Arkansas Children's Hospital||Recruiting|
|Little Rock, Arkansas, United States, 72205|
|Principal Investigator: Kapil Arya, MD|
|United States, California|
|Children's Hospital of Orange County||Recruiting|
|Orange, California, United States, 92868|
|Principal Investigator: Raymond Wang, MD|
|United States, Colorado|
|Children's Hospital of Colorado||Recruiting|
|Aurora, Colorado, United States, 80045|
|Principal Investigator: Scott Demarest, MD|
|United States, Minnesota|
|Children's Hospital Minnesota||Recruiting|
|Minneapolis, Minnesota, United States, 55404|
|Principal Investigator: Vikas Bhambhani, MD|
|United States, New York|
|Mt. Sinai School of Medicine||Recruiting|
|New York, New York, United States, 10029|
|Principal Investigator: Jayesh Ganesh, MD|
|University of Rochester Medical Center||Recruiting|
|Rochester, New York, United States, 14642|
|Principal Investigator: Jennifer Vermilion, MD|
|United States, Ohio|
|Nationwide Children's Hospital||Recruiting|
|Columbus, Ohio, United States, 43130|
|Principal Investigator: Emily De Los Reyes, MD|
|Study Director:||Medical Monitor, MD||BioMarin Pharmaceutical|