A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared With Imiglucerase in Patients With Type I Gaucher Disease

Status: Completed
Location: See all (11) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to the deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this non-inferiority study is to evaluate the efficacy and safety of GA-GCB (velaglucerase alfa) administered every other week in comparison to imiglucerase in treatment naive patients with type 1 Gaucher disease.

Eligibility
Participation Requirements
Sex: All
Minimum Age: 2
Healthy Volunteers: No
View:

• Includes:

• The patient has a documented diagnosis and clinical manifestation of type 1 Gaucher disease

• The patient is at least 2 years of age.

• The patient has not received treatment for Gaucher disease (investigational products, miglustat, or imiglucerase) within 12 months prior to study entry, as documented in the patient's medical history.

• Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study. Male patients must use a medically acceptable method of birth control throughout their participation in the study and must report their partner's pregnancy.

• The patient, the patient's parent(s) or legal guardian(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

• The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

Locations
United States
North Carolina
Duke Children's Hospital & Health Center
Durham
Other Locations
Argentina
Your Health S.A.
Buenos Aires
India
Malabar Institute of Medical Sciences Ltd.
Calicut
All India Institute of Medical Sciences
New Delhi
KEM Hospital Research Centre
Pune
Israel
Shaare Zedek Medical Center
Jerusalem
Paraguay
Sociedad Espanola de Socorros Mutuos
Asuncion
Russian Federation
National Research Center for Haematology
Moscow
Spain
Hospital Universitario Miguel Servet
Zaragoza
Tunisia
La Rabta Hospital
Tunis
United Kingdom
The Royal Free Hospital
London
Time Frame
Start Date: January 29, 2008
Completion Date: May 5, 2009
Participants
Target number of participants: 34
Treatments
Experimental: GA-GCB
VPRIV™ ,velaglucerase alfa
Active Comparator: imiglucerase
Sponsors
Leads: Shire

This content was sourced from clinicaltrials.gov

Similar Clinical Trials