Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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ACTIVE_NOT_RECRUITING
35 participants
OBSERVATIONAL
2020-08-19
2030-08-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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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.
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.
Interventions
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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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Currently receiving or plan to begin treatment with cerliponase alfa.
3. Written informed consent/assent obtained.
Exclusion Criteria
ALL
No
Sponsors
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BioMarin Pharmaceutical
INDUSTRY
Responsible Party
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Principal Investigators
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Medical Director, MD
Role: STUDY_DIRECTOR
BioMarin Pharmaceutical
Locations
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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 D.C., 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
Countries
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Other Identifiers
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190-501
Identifier Type: -
Identifier Source: org_study_id
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