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

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

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

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

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