Cerliponase Alfa Observational Study in the US

Cerliponase Alfa Observational Study

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 Overview

• Status: Active, not recruiting
• Conditions: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
• Intervention / Treatment: Drug: Cerliponase Alfa; Device: Administration Kit

• Study Type: Observational
• Enrollment (Estimated): 35

Contacts and Locations

• Study Contact: Name: Trial Specialist; Phone Number: +1.651.523.0310; Email: medinfo@bmrn.com

Study Locations

• United States
  • Arkansas
    • Little Rock, Arkansas, United States, 72205
      • Arkansas Children's Hospital
  • California
    • Orange, California, United States, 92868
      • Children's Hospital of Orange County
  • Colorado
    • Aurora, Colorado, United States, 80045
      • Children's Hospital of Colorado
  • District of Columbia
    • Washington, District of Columbia, United States, 20010
      • Children's National Hospital
  • Florida
    • Orlando, Florida, United States, 32803
      • Advent Health
  • Georgia
    • Atlanta, Georgia, United States, 30329
      • Children's Healthcare of Atlanta
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Rush University Medical Center
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Boston Children's Hospital,
  • Minnesota
    • Minneapolis, Minnesota, United States, 55404
      • Children's Hospital Minnesota
  • New York
    • New York, New York, United States, 10029
      • Mt. Sinai School of Medicine
    • New York, New York, United States, 10017
      • NYU Langone Medical Center
    • Rochester, New York, United States, 14642
      • University of Rochester Medical Center
  • Ohio
    • Columbus, Ohio, United States, 43130
      • Nationwide Children's Hospital
  • Oklahoma
    • Oklahoma City, Oklahoma, United States, 73104
      • University of Oklahoma Health Sciences Center
  • Texas
    • Houston, Texas, United States, 77030
      • Texas Children's Hospital
  • Washington
    • Seattle, Washington, United States, 98145-5005
      • Seattle Children's Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• 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.

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hypersensitivity	To further assess the occurrence of serious hypersensitivity reactions (including anaphylaxis), serious cardiovascular adverse events, and serious device-related complications.	10 years
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

Collaborators and Investigators

• Sponsor: BioMarin Pharmaceutical
• Investigators: Study Director: Medical Monitor, MD, BioMarin Pharmaceutical

Study record dates

Study Major Dates

• Study Start (Actual): August 19, 2020
• Primary Completion (Estimated): August 31, 2030
• Study Completion (Estimated): August 31, 2030

Study Registration Dates

• First Submitted: July 15, 2020
• First Submitted That Met QC Criteria: July 15, 2020
• First Posted (Actual): July 20, 2020

Study Record Updates

• Last Update Posted (Actual): January 31, 2024
• Last Update Submitted That Met QC Criteria: January 29, 2024
• Last Verified: January 1, 2024

More Information

Terms related to this study

• Keywords: Batten's Disease; CLN2; TPP1 enzyme
• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Metabolism, Inborn Errors; Heredodegenerative Disorders, Nervous System; Lipid Metabolism Disorders; Lipidoses; Lipid Metabolism, Inborn Errors; Neuronal Ceroid-Lipofuscinoses
• Other Study ID Numbers: 190-501

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: Yes