Cerliponase Alfa Observational Study in the US
1 other identifier
observational
35
1 country
16
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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for all trials
Started Aug 2020
Longer than P75 for all trials
16 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 15, 2020
CompletedFirst Posted
Study publicly available on registry
July 20, 2020
CompletedStudy Start
First participant enrolled
August 19, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 31, 2030
ExpectedStudy Completion
Last participant's last visit for all outcomes
August 31, 2030
January 14, 2026
January 1, 2026
10 years
July 15, 2020
January 13, 2026
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Safety surveillance of cerliponase alfa
To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).
10 years
Secondary Outcomes (2)
Hypersensitivity
10 years
Severe SAE impact on patient's motor and language functions
10 years
Study Arms (1)
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.
Interventions
Commercially available product provided to patient by participating clinic site.
Commercially available administration kit provided to the patient by participating clinic site.
Eligibility Criteria
CLN2 patients who are currently taking or intend to take cerliponase alfa within 60 days of signing the informed consent form.
You may qualify if:
- Diagnosed with CLN2 disease.
- Currently receiving or plan to begin treatment with cerliponase alfa.
- Written informed consent/assent obtained.
You may not qualify if:
- \. Currently receiving treatment in another investigational device or drug study.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (16)
Arkansas Children's Hospital
Little Rock, Arkansas, 72205, United States
Children's Hospital of Orange County
Orange, California, 92868, United States
Children's Hospital of Colorado
Aurora, Colorado, 80045, United States
Children's National Hospital
Washington D.C., District of Columbia, 20010, United States
Advent Health
Orlando, Florida, 32803, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329, United States
Rush University Medical Center
Chicago, Illinois, 60612, United States
Boston Children's Hospital,
Boston, Massachusetts, 02115, United States
Children's Hospital Minnesota
Minneapolis, Minnesota, 55404, United States
NYU Langone Medical Center
New York, New York, 10017, United States
Mt. Sinai School of Medicine
New York, New York, 10029, United States
University of Rochester Medical Center
Rochester, New York, 14642, United States
Nationwide Children's Hospital
Columbus, Ohio, 43130, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
Texas Children's Hospital
Houston, Texas, 77030, United States
Seattle Children's Hospital
Seattle, Washington, 98145-5005, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Medical Director, MD
BioMarin Pharmaceutical
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 15, 2020
First Posted
July 20, 2020
Study Start
August 19, 2020
Primary Completion (Estimated)
August 31, 2030
Study Completion (Estimated)
August 31, 2030
Last Updated
January 14, 2026
Record last verified: 2026-01
Data Sharing
- IPD Sharing
- Will not share