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NCT04476862 · ClinicalTrials.gov registry record
Cerliponase Alfa Observational Study in the US
A clinical trial of Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, sponsored by BioMarin Pharmaceutical.
- Active
- Registry status
- 35
- Enrollment target
- 16
- Study locations
NCT04476862: Active study of Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, sponsored by BioMarin Pharmaceutical.
NCT04476862 is a study of Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 that is active but no longer recruiting, run by BioMarin Pharmaceutical. The registered enrollment target is 35 participants. The trial reports 16 study locations across 14 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT04476862, a study of Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2, is active but no longer recruiting, sponsored by BioMarin Pharmaceutical.
- ACTIVE NOT RECRUITING
- Registry status
- 35 participants
- Enrollment target
- 16
- Study locations
Study 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.
Primary Outcome
To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).
Conditions Studied
Interventions
- DRUG Cerliponase Alfa
- DEVICE Administration Kit
Study Locations (16)
New York
- NYU Langone Medical Center - New York
- Mt. Sinai School of Medicine - New York
- University of Rochester Medical Center - Rochester
Arkansas
- Arkansas Children's Hospital - Little Rock
California
- Children's Hospital of Orange County - Orange
Colorado
- Children's Hospital of Colorado - Aurora
District of Columbia
- Children's National Hospital - Washington D.C.
Florida
- Advent Health - Orlando
Georgia
- Children's Healthcare of Atlanta - Atlanta
Illinois
- Rush University Medical Center - Chicago
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2020-08-19 |
| Est. Completion | 2030-08-31 |
What the registry record for NCT04476862 still lists
NCT04476862 is an observational study that tracks outcomes without assigning an intervention. The registry caps enrollment at 35 participants, a relatively small participant target.
The record links to 1 condition, with Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 appearing as the primary indexed condition, and to 2 interventions - of which Cerliponase Alfa is the first listed.
NCT04476862 lists 16 locations in 14 states (New York, Arkansas, California).
Frequently Asked Questions
What is clinical trial NCT04476862 about?
NCT04476862 is a clinical study titled "Cerliponase Alfa Observational Study in the US". 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...
What is the current status of trial NCT04476862?
This trial is currently active not recruiting. The enrollment target is 35 participants. The study started on 2020-08-19. Estimated completion is 2030-08-31.
What conditions does trial NCT04476862 study?
This clinical trial studies the following conditions: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2.
What interventions are being tested in trial NCT04476862?
The interventions under investigation include: Cerliponase Alfa (DRUG), Administration Kit (DEVICE).
Who is sponsoring clinical trial NCT04476862?
This trial is sponsored by BioMarin Pharmaceutical, which has 80 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT04476862 being conducted?
This trial has 16 study locations across Arkansas, California, Colorado, District of Columbia, Florida. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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