Cerliponase Alfa Observational Study in the US

• ClinicalTrials.gov Identifier: NCT04476862
• Recruitment Status: Recruiting
• First Posted: July 20, 2020
• Last Update Posted: February 11, 2021
• Sponsor: BioMarin Pharmaceutical
• Information provided by (Responsible Party): BioMarin Pharmaceutical

Study Description

Brief 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.

Condition or disease	Intervention/treatment
Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2	Drug: Cerliponase Alfa; Device: Administration Kit

Study Design

• Study Type: Observational
• Estimated Enrollment: 35 participants
• Observational Model: Cohort
• Time Perspective: Prospective
• Official Title: Cerliponase Alfa Observational Study
• Actual Study Start Date: August 19, 2020
• Estimated Primary Completion Date: August 24, 2030
• Estimated Study Completion Date: August 24, 2030

Groups and Cohorts

Group/Cohort	Intervention/treatment
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.	Drug: Cerliponase Alfa; Commercially available product provided to patient by participating clinic site.; Other Name: Brineura; Device: Administration Kit; Commercially available administration kit provided to the patient by participating clinic site.

Outcome Measures

Primary Outcome Measures :

1. Safety surveillance of cerliponase alfa [ Time Frame: 10 years ]
   To evaluate the long-term safety of cerliponase alfa in patients with neuronal ceroid lipofuscinosis Type 2 (CLN2 disease).

Secondary Outcome Measures :

1. Hypersensitivity [ Time Frame: 10 years ]
   To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.
2. Severe SAE impact on patient's motor and language functions [ Time Frame: 10 years ]
   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).

Eligibility Criteria

• Ages Eligible for Study: Child, Adult, Older Adult
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No
• Sampling Method: Non-Probability Sample

Study Population

CLN2 patients who are currently taking or intend to take cerliponase alfa within 60 days of signing the informed consent form.

Criteria

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.

Contacts and Locations

Contacts

Contact: Trial Specialist; +1.651.523.0310; medinfo@bmrn.com

Locations

United States, Arkansas

Arkansas Children's Hospital; Recruiting

Little Rock, Arkansas, United States, 72205

Principal Investigator: Kapil Arya, MD

United States, California

Children's Hospital of Orange County; Recruiting

Orange, California, United States, 92868

Principal Investigator: Raymond Wang, MD

United States, Colorado

Children's Hospital of Colorado; Recruiting

Aurora, Colorado, United States, 80045

Principal Investigator: Scott Demarest, MD

United States, Minnesota

Children's Hospital Minnesota; Recruiting

Minneapolis, Minnesota, United States, 55404

Principal Investigator: Vikas Bhambhani, MD

United States, New York

Mt. Sinai School of Medicine; Recruiting

New York, New York, United States, 10029

Principal Investigator: Jayesh Ganesh, MD

University of Rochester Medical Center; Recruiting

Rochester, New York, United States, 14642

Principal Investigator: Jennifer Vermilion, MD

United States, Ohio

Nationwide Children's Hospital; Recruiting

Columbus, Ohio, United States, 43130

Principal Investigator: Emily De Los Reyes, MD

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

• Study Director: Medical Monitor, MD; BioMarin Pharmaceutical

More Information

• Responsible Party: BioMarin Pharmaceutical
• ClinicalTrials.gov Identifier: NCT04476862
• Other Study ID Numbers: 190-501
• First Posted: July 20, 2020
• Last Update Posted: February 11, 2021
• Last Verified: February 2021

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: Yes

Keywords provided by BioMarin Pharmaceutical:

Batten's Disease; CLN2; TPP1 enzyme

Additional relevant MeSH terms:

Neuronal Ceroid-Lipofuscinoses; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Lipidoses; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases