Cerliponase Alfa Observational Study in the US
- Conditions
- Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
- Interventions
- Device: Administration Kit
- Registration Number
- NCT04476862
- Lead Sponsor
- BioMarin Pharmaceutical
- 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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 35
- Diagnosed with CLN2 disease.
- Currently receiving or plan to begin treatment with cerliponase alfa.
- Written informed consent/assent obtained.
- Currently receiving treatment in another investigational device or drug study.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description Cerliponase alfa patients Administration Kit Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form. Cerliponase alfa patients Cerliponase Alfa Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.
- Primary Outcome Measures
Name Time Method 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 Outcome Measures
Name Time Method 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).
Hypersensitivity 10 years To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.
Trial Locations
- Locations (16)
Arkansas Children's Hospital
🇺🇸Little Rock, Arkansas, United States
Children's Hospital of Orange County
🇺🇸Orange, California, United States
Children's Hospital of Colorado
🇺🇸Aurora, Colorado, United States
Children's National Hospital
🇺🇸Washington, District of Columbia, United States
Advent Health
🇺🇸OrLando, Florida, United States
Children's Healthcare of Atlanta
🇺🇸Atlanta, Georgia, United States
Rush University Medical Center
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital,
🇺🇸Boston, Massachusetts, United States
Children's Hospital Minnesota
🇺🇸Minneapolis, Minnesota, United States
NYU Langone Medical Center
🇺🇸New York, New York, United States
Mt. Sinai School of Medicine
🇺🇸New York, New York, United States
University of Rochester Medical Center
🇺🇸Rochester, New York, United States
Nationwide Children's Hospital
🇺🇸Columbus, Ohio, United States
University of Oklahoma Health Sciences Center
🇺🇸Oklahoma City, Oklahoma, United States
Texas Children's Hospital
🇺🇸Houston, Texas, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States