Cerliponase Alfa Observational Study in the US
Public ClinicalTrials.gov record NCT04476862. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
Cerliponase Alfa Observational Study
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04476862
- Recruitment status
- Active, not recruiting
- Study type
- Observational
- Phase
- Not listed
- Enrollment
- 35 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- Not listed
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Aug 19, 2020 (Actual)
- Primary completion
- Aug 31, 2030 (Estimated)
- Completion
- Aug 31, 2030 (Estimated)
- Last update posted
- Jun 30, 2026
2020 – 2030
United States locations
- U.S. sites
- 16
- U.S. states
- 14
- U.S. cities
- 15
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Arkansas Children's Hospital | Little Rock | Arkansas | 72205 | — |
| Children's Hospital of Orange County | Orange | California | 92868 | — |
| Children's Hospital of Colorado | Aurora | Colorado | 80045 | — |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | — |
| Advent Health | Orlando | Florida | 32803 | — |
| Children's Healthcare of Atlanta | Atlanta | Georgia | 30329 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Boston Children's Hospital, | Boston | Massachusetts | 02115 | — |
| Children's Hospital Minnesota | Minneapolis | Minnesota | 55404 | — |
| NYU Langone Medical Center | New York | New York | 10017 | — |
| Mt. Sinai School of Medicine | New York | New York | 10029 | — |
| University of Rochester Medical Center | Rochester | New York | 14642 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43130 | — |
| University of Oklahoma Health Sciences Center | Oklahoma City | Oklahoma | 73104 | — |
| Texas Children's Hospital | Houston | Texas | 77030 | — |
| Seattle Children's Hospital | Seattle | Washington | 98145-5005 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04476862, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jun 30, 2026 · Synced Oct 25, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04476862 live on ClinicalTrials.gov.