Clinical trial NCT04476862: Cerliponase Alfa Observational Study in the US
Official title: Cerliponase Alfa Observational Study
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2
Keywords: Batten's Disease
CLN2
TPP1 enzyme
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2020-07-20
Enrollment: 35
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

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.


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.

Primary outcomes:
measure: Safety surveillance of cerliponase alfa
timeFrame: 10 years
Secondary outcomes:
measure: Hypersensitivity
timeFrame: 10 years
measure: Severe SAE impact on patient's motor and language functions
timeFrame: 10 years
Other outcomes:
Locations:
city: Little Rock
country: United States
facility: Arkansas Children's Hospital
state: Arkansas
city: Orange
country: United States
facility: Children's Hospital of Orange County
state: California
city: Aurora
country: United States
facility: Children's Hospital of Colorado
state: Colorado
city: Washington
country: United States
facility: Children's National Hospital
state: District of Columbia
city: OrLando
country: United States
facility: Advent Health
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Chicago
country: United States
facility: Rush University Medical Center
state: Illinois
city: Boston
country: United States
facility: Boston Children's Hospital,
state: Massachusetts
city: Minneapolis
country: United States
facility: Children's Hospital Minnesota
state: Minnesota
city: New York
country: United States
facility: NYU Langone Medical Center
state: New York
city: New York
country: United States
facility: Mt. Sinai School of Medicine
state: New York
city: Rochester
country: United States
facility: University of Rochester Medical Center
state: New York
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Oklahoma City
country: United States
facility: University of Oklahoma Health Sciences Center
state: Oklahoma
city: Houston
country: United States
facility: Texas Children's Hospital
state: Texas
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
Study officials:
affiliation: BioMarin Pharmaceutical
name: Medical Director, MD
role: STUDY_DIRECTOR
References: