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Cerliponase Alfa Observational Study in the US

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

Tracking Information
First Submitted Date July 15, 2020
First Posted Date July 20, 2020
Last Update Posted Date February 11, 2021
Actual Study Start Date August 19, 2020
Estimated Primary Completion Date August 24, 2030   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: July 15, 2020)
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).
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: July 15, 2020)
  • 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.
  • 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).
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Cerliponase Alfa Observational Study in the US
Official Title Cerliponase Alfa Observational Study
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.
Detailed Description Not Provided
Study Type Observational
Study Design Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Intervention
  • 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.
Study Groups/Cohorts 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.
Interventions:
  • Drug: Cerliponase Alfa
  • Device: Administration Kit
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Recruiting
Estimated Enrollment
 (submitted: July 15, 2020)
35
Original Estimated Enrollment Same as current
Estimated Study Completion Date August 24, 2030
Estimated Primary Completion Date August 24, 2030   (Final data collection date for primary outcome measure)
Eligibility 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.

Sex/Gender
Sexes Eligible for Study: All
Ages Child, Adult, Older Adult
Accepts Healthy Volunteers No
Contacts
Contact: Trial Specialist +1.651.523.0310 medinfo@bmrn.com
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT04476862
Other Study ID Numbers 190-501
Has Data Monitoring Committee No
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: Yes
IPD Sharing Statement
Plan to Share IPD: No
Responsible Party BioMarin Pharmaceutical
Study Sponsor BioMarin Pharmaceutical
Collaborators Not Provided
Investigators
Study Director: Medical Monitor, MD BioMarin Pharmaceutical
PRS Account BioMarin Pharmaceutical
Verification Date February 2021