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

Phase 4

Completed

• Conditions: Gaucher Disease
• Interventions: Drug: Taliglucerase alfa

• Registration Number: NCT01132690
• Lead Sponsor: Pfizer

Brief Summary

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2010-05-26
• Study First Submitted QC: 2010-05-26
• Study First Posted: 2010-05-28
• Study Start: 2010-08
• Primary Completion: 2012-05
• Study Completion: 2012-07
• Disposition First Submitted: 2014-04-25
• Disposition First Submitted QC: 2014-04-25
• Disposition First Posted: 2014-05-13
• Results First Submitted: 2014-10-28
• Results First Submitted QC: 2014-11-10
• Results First Posted: 2014-11-11
• Status Verified: 2018-09
• Last Update Submitted: 2018-09-05
• Last Update Posted: 2018-10-05

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
30 units/kg	Taliglucerase alfa	-
60 units/kg	Taliglucerase alfa	-

Primary Outcome Measures

Name	Time	Method
Hemoglobin	Every 3 months for 12 months	median and interquartile range for change from baseline in haemoglobin

Secondary Outcome Measures

Name	Time	Method
Platelet Count	Baseline and 12 months	Mean and standard deviation of platelet count per cubic mm
Spleen Volume	Baseline and Month 12	Spleen volume measured by MRI
Liver Volume	Baseline and Month 12	Liver volume measured by MRI
Chitotriosidase	Every 3 months for 12 months	Percent change from baseline in chitotriosidase
Chemokine (C-C Motif) Ligand 18 (CCL18)	Every 3 months for 12 months	Percent change from baseline in CCL18

Trial Locations

Locations (3)

Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C); 🇵🇾 Barrio Sajonia Asunción, Paraguay

Shaare Zedek Medical Center; 🇮🇱 Jerusalem, Israel

Morningside Medi-Clinic; 🇿🇦 Morningside, South Africa