A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Pfizer

Status and phase

Completed

Phase 4

Conditions

Gaucher Disease

Treatments

Drug: Taliglucerase alfa

Study type

Interventional

Funder types

Industry

Identifiers

NCT01132690

PB-06-005

Details and patient eligibility

About

This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60 units/kg. Subjects will receive an intravenous (IV) infusion of taliglucerase alfa every two weeks. The total duration of treatment will be 12 months. At the end of the 12-month treatment period eligible subjects will be offered enrollment in an open-label extension study if taliglucerase alfa is not commercially available.

Enrollment

11 patients

Sex

All

Ages

2 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Males and females 2 to <18 years old.
Diagnosis of Gaucher disease with leukocyte acid β-glucosidase activity ≤30% of the mean of the reference range for healthy subjects.
Subjects who have not received enzyme replacement therapy (ERT) in the past or who have not received ERT in the past 12 months and have a negative anti-glucocerebrosidase antibody assay.
Subjects who have not received substrate reduction therapy (SRT) in the past 12 months.
Subjects whose clinical condition, in the opinion of the investigator, requires treatment with enzyme replacement therapy (ERT).

Exclusion criteria

Currently taking another investigational drug for any condition.
Presence of neurological signs and symptoms characteristic of Gaucher disease with complex neuronopathic features other than longstanding oculomotor gaze palsy.
Presence of unresolved anemia due to iron, folic acid, or vitamin B12 deficiency
Previous hypersensitivity reaction to Cerezyme® (imiglucerase) or Ceredase® (alglucerase).
History of allergy to carrots.
Presence of HIV, HBsAg or hepatitis C infections.
Subject's parent(s) or legal guardian(s) are unable to understand the nature, scope and possible consequences of the study.
Presence of any medical, emotional, behavioral or psychological condition that in the judgment of the Investigator would interfere with the subject's compliance with the requirements of the study.