Cerliponase Alfa Observational Study in the US

Cerliponase Alfa Observational Study


Lead Sponsor: BioMarin Pharmaceutical

Source 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.

Overall Status Recruiting
Start Date 2020-08-19
Completion Date 2030-08-24
Primary Completion Date 2030-08-24
Study Type Observational
Primary Outcome
Measure Time Frame
Safety surveillance of cerliponase alfa 10 years
Secondary Outcome
Measure Time Frame
Hypersensitivity 10 years
Severe SAE impact on patient's motor and language functions 10 years
Enrollment 35

Intervention Type: Drug

Intervention Name: Cerliponase Alfa

Description: Commercially available product provided to patient by participating clinic site.

Arm Group Label: Cerliponase alfa patients

Other Name: Brineura

Intervention Type: Device

Intervention Name: Administration Kit

Description: Commercially available administration kit provided to the patient by participating clinic site.

Arm Group Label: Cerliponase alfa patients


Sampling Method:

Non-Probability Sample


Inclusion Criteria: 1. Diagnosed with CLN2 disease. 2. Currently receiving or plan to begin treatment with cerliponase alfa. 3. Written informed consent/assent obtained. Exclusion Criteria: 1. Currently receiving treatment in another investigational device or drug study.



Minimum Age:


Maximum Age:


Healthy Volunteers:


Overall Official
Last Name Role Affiliation
Medical Monitor, MD Study Director BioMarin Pharmaceutical
Overall Contact

Last Name: Trial Specialist

Phone: +1.651.523.0310

Email: [email protected]

Facility: Status: Investigator:
Arkansas Children's Hospital | Little Rock, Arkansas, 72205, United States Recruiting Kapil Arya, MD Principal Investigator
Children's Hospital of Orange County | Orange, California, 92868, United States Recruiting Raymond Wang, MD Principal Investigator
Children's Hospital of Colorado | Aurora, Colorado, 80045, United States Recruiting Scott Demarest, MD Principal Investigator
Advent Health | Orlando, Florida, 32803, United States Recruiting Ki Hyeong Lee Principal Investigator
Children's Healthcare of Atlanta | Atlanta, Georgia, 30329, United States Recruiting Stephanie Keller Principal Investigator
Rush University Medical Center | Chicago, Illinois, 60612, United States Recruiting Elizabeth Berry-Kravis Principal Investigator
Boston Children's Hospital, | Boston, Massachusetts, 02115, United States Recruiting Alexander Rotenberg Principal Investigator
Children's Hospital Minnesota | Minneapolis, Minnesota, 55404, United States Recruiting Vikas Bhambhani, MD Principal Investigator
Mt. Sinai School of Medicine | New York, New York, 10029, United States Recruiting Jayesh Ganesh, MD Principal Investigator
University of Rochester Medical Center | Rochester, New York, 14642, United States Recruiting Jennifer Vermilion, MD Principal Investigator
Nationwide Children's Hospital | Columbus, Ohio, 43130, United States Recruiting Emily De Los Reyes, MD Principal Investigator
University of Oklahoma Health Sciences Center | Oklahoma City, Oklahoma, 73104, United States Recruiting Rene McNall-Knapp Principal Investigator
Texas Children's Hospital | Houston, Texas, 77030, United States Recruiting Gary Clark Principal Investigator
Seattle Children's Hospital | Seattle, Washington, 98145-5005, United States Recruiting Irene Chang Principal Investigator
Location Countries

United States

Verification Date


Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

Label: Cerliponase alfa patients

Description: Patients who are currently on or plan to start taking cerliponase alfa within 60 days of signing the study informed consent form.

Patient Data No
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact [email protected]. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.


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