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

NCT01132690 · Status: COMPLETED · Phase: PHASE4 · Type: INTERVENTIONAL · Enrollment: 11

Last updated 2018-10-05

Study results available
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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.

Conditions

  • Gaucher Disease

Interventions

DRUG

Taliglucerase alfa

Taliglucerase alfa for infusion every two weeks for 12 months

Sponsors & Collaborators

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
2 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2010-08-31
Primary Completion
2012-05-31
Completion
2012-07-31

Countries

  • Israel
  • Paraguay
  • South Africa

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01132690 on ClinicalTrials.gov