ABOUT THIS STUDY
1. Male or female pediatric subjects with a diagnosis of Type 1 Gaucher disease (evidence of leukocyte acid-glucosidase activity 30% of the mean of the reference range for healthy persons), and meeting one of three weight categories: less than15 kg; 15 to less than 20 kg or 20-25 kg.
2. ERT treatment-naïve, or able to perform the PK assessment within the initial 6 months of initial Elelyso treatment (provided the subject was ERT naïve prior to the start of Elelyso).
3. Have had baseline (ie, prior to the first dose of study medication) PD measurements (spleen volume/size measured by MRI, CT or ultrasound, hemoglobin/platelet counts and growth measures including height and weight), immunogenicity sample collection and Gaucher disease diagnosis history documented in advance of treatment start.
4. Presence of splenomegaly at baseline defined as spleen volume/size measurement of 5 MN.
5. Subjects prescribed the nominal dose of 60 units/kg every two weeks and can tolerate an infusion rate of 1 mL/min.
6. Evidence of a personally signed and dated informed consent document from parent/legal guardian (or adult caregiver) capable of providing informed consent indicating that the subject's parent(s)/legal guardian has been informed of all pertinent aspects of the study before any screening procedures are performed. When age appropriate, written assent must also be obtained.
7. Have parent/legal guardian (or adult caregiver) capable and willing to comply with scheduled visits, treatment plan, laboratory tests and other study procedures.
1. Evidence or history of clinically significant issue or the presence of a medical,
emotional, behavioral or psychological condition that, in the judgment of the
Investigator, would interfere with the subject's participation in the study, cause
harm to the subject or decrease compliance with the study requirements.
2. Treatment with an investigational drug within 30 days (or as determined by the local
requirement) or 5 half-lives preceding the PK sample collection visit, whichever is
3. A diagnosis of Type 2 or 3 Gaucher disease, or the presence of neurological signs and
symptoms characteristic of Type 2 or 3 Gaucher disease.
4. Any change during the registry study to the subjects dose of taliglucerase alfa
infusion (ie, change from 60 units/kg every two weeks to a different dose of
taliglucerase alfa) or a change in infusion duration or rate or a change of ERT
medication (ie, switch from taliglucerase alfa to a different ERT).
5. In the judgment of the Investigator, the subject's vital signs (eg, blood pressure,
pulse) prior to infusion on the day of the PK visit indicate that participation in the
study would not be in the study candidate's best interest.
6. A hemoglobin level of <10 g/dL within 30 days of the PK sample visit or on the day of
the PK sample visit.
7. History of sensitivity to heparin or heparin-induced thrombocytopenia. (Note: applies
only if heparin lock or flush is to be used on the day of the PK sample visit).
8. Parents or legal guardians who are investigational site staff members directly
involved in the conduct of the study and their family members, children of site staff
members otherwise supervised by the Investigator, or subjects who are children of
Pfizer employees directly involved in the conduct of the study.
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