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

Gaucher Disease Clinical Trial

Official title:

A Multicenter, Double-blind, Randomized Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01132690
• Other study ID #: PB-06-005
• Status: Completed
• Phase: Phase 4
• Start date: August 2010
• Est. completion date: July 2012

Study information

• Verified date: September 2018
• Source: Pfizer
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: July 2012
• Est. primary completion date: May 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years to 17 Years

Eligibility

Inclusion Criteria:

• Males and females 2 to <18 years old.

• Diagnosis of Gaucher disease with leukocyte acid ß-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.

Related Conditions & MeSH terms

• Gaucher Disease

Intervention

Drug:
• Taliglucerase alfa
Taliglucerase alfa for infusion every two weeks for 12 months

Locations

Country	Name	City	State
Israel	Shaare Zedek Medical Center	Jerusalem
Paraguay	Instituto Privado de Hematologia E Investigacion Clinica (I.P.H.I.C)	Barrio Sajonia Asunción
South Africa	Morningside Medi-Clinic	Morningside

Sponsors (1)

Lead Sponsor	Collaborator
Pfizer

Countries where clinical trial is conducted

Israel,  Paraguay,  South Africa, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Hemoglobin	median and interquartile range for change from baseline in haemoglobin	Every 3 months for 12 months
Secondary	Chitotriosidase	Percent change from baseline in chitotriosidase	Every 3 months for 12 months
Secondary	Spleen Volume	Spleen volume measured by MRI	Baseline and Month 12
Secondary	Platelet Count	Mean and standard deviation of platelet count per cubic mm	Baseline and 12 months
Secondary	Chemokine (C-C Motif) Ligand 18 (CCL18)	Percent change from baseline in CCL18	Every 3 months for 12 months
Secondary	Liver Volume	Liver volume measured by MRI	Baseline and Month 12