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)
| Facility | City | Region | Status |
|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | |
| Children's Hospital of Orange County | Orange | California | |
| Children's Hospital of Colorado | Aurora | Colorado | |
| Children's National Hospital | Washington D.C. | District of Columbia | |
| Advent Health | Orlando | Florida | |
| Children's Healthcare of Atlanta | Atlanta | Georgia | |
| Rush University Medical Center | Chicago | Illinois | |
| Boston Children's Hospital, | Boston | Massachusetts | |
| Children's Hospital Minnesota | Minneapolis | Minnesota | |
| NYU Langone Medical Center | New York | New York | |
| Mt. Sinai School of Medicine | New York | New York | |
| University of Rochester Medical Center | Rochester | New York | |
| Nationwide Children's Hospital | Columbus | Ohio | |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | |
| Texas Children's Hospital | Houston | Texas | |
| Seattle Children's Hospital | Seattle | Washington |
More BioMarin Pharmaceutical trials in the USA
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT04476862 on ClinicalTrials.gov ↗ ← All trials in the USA