Gaucher Disease and Effects of Taliglucerase Alfa – Phase IV

Study of Elelyso™ (Taliglucerase Alfa) in Pediatric Subjects with Type 1 Gaucher Disease

Protocol Description

The purpose of this international multi-center study is to collect and analyze data about how an intravenous infusion of taliglucerase alfa (Elelyso™) in children with Type 1 Gaucher disease is processed by the body. Elelyso is approved in United States, Canada, Mexico, and other countries, and is available by prescription for Type 1 Gaucher disease. The manufacturer is seeking additional information about it so as to provide the best information to other patients with Gaucher disease in the future.

Study Level: Phase IV

Eligibility Criteria

Subject to certain exclusion criteria, the study is accepting patients of both genders up to age 12 who have a confirmed diagnosis of Type 1 Gaucher disease and are already participating in the Gaucher Disease Enzyme Replacement Therapy Registry.
Boys and Girls: Up to age 12

Requirements

Participants will be seen by the team for a screening visit to determine eligibility, a baseline visit to collect additional information about the child’s Gaucher disease, and additional visits to receive study medications and for blood sampling and other tests.
Visits: 5
Duration: 13 months

Status: Open to Enrollment

Source(s) of Support

Pfizer

Primary Investigator

Gerard Vockley, MD, PhD

Contact Information

For more information about the study or enrollment, please contact:
Nadene Henderson, MS, LCGC
412-692-3475