Cerliponase Alfa Observational Study in the US

Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2 Clinical Trial

Official title:

Cerliponase Alfa Observational Study

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT04476862
• Other study ID #: 190-501
• Status: Active, not recruiting
• Start date: August 19, 2020
• Est. completion date: August 31, 2030

Study information

• Verified date: January 2024
• Source: BioMarin Pharmaceutical
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 35
• Est. completion date: August 31, 2030
• Est. primary completion date: August 31, 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Diagnosed with CLN2 disease.

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

3. Written informed consent/assent obtained.

Exclusion Criteria:

1. Currently receiving treatment in another investigational device or drug study.

Related Conditions & MeSH terms

• Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
• Neuronal Ceroid-Lipofuscinoses

Intervention

Drug:
• Cerliponase Alfa
Commercially available product provided to patient by participating clinic site.

Device:
• Administration Kit
Commercially available administration kit provided to the patient by participating clinic site.

Locations

Country	Name	City	State
United States	Children's Healthcare of Atlanta	Atlanta	Georgia
United States	Children's Hospital of Colorado	Aurora	Colorado
United States	Boston Children's Hospital,	Boston	Massachusetts
United States	Rush University Medical Center	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	Texas Children's Hospital	Houston	Texas
United States	Arkansas Children's Hospital	Little Rock	Arkansas
United States	Children's Hospital Minnesota	Minneapolis	Minnesota
United States	Mt. Sinai School of Medicine	New York	New York
United States	NYU Langone Medical Center	New York	New York
United States	University of Oklahoma Health Sciences Center	Oklahoma City	Oklahoma
United States	Children's Hospital of Orange County	Orange	California
United States	Advent Health	Orlando	Florida
United States	University of Rochester Medical Center	Rochester	New York
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Hospital	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety surveillance of cerliponase alfa	To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).	10 years
Secondary	Hypersensitivity	To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.	10 years
Secondary	Severe SAE impact on patient's motor and language functions	To evaluate the effects of Grade III or higher serious adverse events (SAEs) on patient performance on the CLN2 clinical rating scale (motor and language domains).	10 years