Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
Cerliponase Alfa Observational Study in the US
NCT04476862 · View on ClinicalTrials.gov ↗
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.
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 |
Interested in This Trial?
Always speak with your doctor before enrolling in a clinical trial.
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04476862
The ClinicalTrials.gov registry entry for NCT04476862 describes a study currently listed as active not recruiting. It is categorized as an unspecified phase, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is BioMarin Pharmaceutical, which has 31 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
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. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04476862 reports 16 study locations spanning 14 distinct geographic areas — top geographies include New York, Arkansas, California. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
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. These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04476862?
The interventions under investigation include: Cerliponase Alfa (DRUG), Administration Kit (DEVICE). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04476862?
This trial is sponsored by BioMarin Pharmaceutical, which has 31 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
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.
Learn More About Clinical Trials
How Clinical Trials Work
Understand phases 1-4, trial design, randomization, and the informed consent process.
Patient Rights in Clinical Trials
Your rights as a participant: consent, withdrawal, privacy, and who to contact.
Finding the Right Clinical Trial
A practical guide to searching trials, understanding eligibility, and evaluating options.
All Guides
Browse our complete library of clinical trial educational resources.
Read our methodology — how this data is sourced, computed, and verified.