Cerliponase Alfa Observational Study

Status: Active_not_recruiting
Location: See all (16) locations...
Intervention Type: Drug, Device
Study Type: Observational
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
Participation Requirements
Sex: All
Healthy Volunteers: f
View:

• Diagnosed with CLN2 disease.

• Currently receiving or plan to begin treatment with cerliponase alfa.

• Written informed consent/assent obtained.

Locations
United States
Arkansas
Arkansas Children's Hospital
Little Rock
California
Children's Hospital of Orange County
Orange
Colorado
Children's Hospital of Colorado
Aurora
Washington, D.c.
Children's National Hospital
Washington D.c.
Florida
Advent Health
Orlando
Georgia
Children's Healthcare of Atlanta
Atlanta
Illinois
Rush University Medical Center
Chicago
Massachusetts
Boston Children's Hospital,
Boston
Minnesota
Children's Hospital Minnesota
Minneapolis
New York
Mt. Sinai School of Medicine
New York
NYU Langone Medical Center
New York
University of Rochester Medical Center
Rochester
Ohio
Nationwide Children's Hospital
Columbus
Oklahoma
University of Oklahoma Health Sciences Center
Oklahoma City
Texas
Texas Children's Hospital
Houston
Washington
Seattle Children's Hospital
Seattle
Time Frame
Start Date: 2020-08-19
Completion Date: 2030-08-31
Participants
Target number of participants: 35
Treatments
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.
Sponsors
Leads: BioMarin Pharmaceutical

This content was sourced from clinicaltrials.gov