Estudio de extensión abierto de terapia de sustitución enzimática con glucocerebrosidasa humana activada genéticamente® (GA-GCB) en pacientes con enfermedad de Gaucher de tipo IAn Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease

• Conditions: MedDRA version: 9.1Level: LLTClassification code 10018048Term: Gaucher's disease; Enfermedad de Gaucher de tipo IType I Gaucher disease

• Registration Number: EUCTR2008-001965-27-ES
• Lead Sponsor: Shire Human Genetic Therapies, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-09-17
• Last Update Posted: 15 April 2013

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 102

Inclusion Criteria

Patients must meet all of the following criteria to be considered eligible for enrollment:
1. The patient has completed through Week 53 of study TKT032 or TKT034, or through Week 41 of study HGT-GCB-039.
2. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy tests will be performed at the time of enrollment and as required throughout participation in the study. Male patients must agree to use a medically acceptable method of contraception at all times during the study and report a partner’s pregnancy to the investigator.
3. 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).
4. The patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.

Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

Patients who meet any of the following criteria are not eligible for this study:
1. The patient has received treatment with any investigational drug, other than GA-GCB, or device within the 30 days prior to study entry; such use during the study is not permitted. Treatment with imiglucerase, if provided as a part of participation in HGT-GCB-039, is allowed.
2. The patient is pregnant or lactating.
3. The patient, patient’s parent(s), or patient’s legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study.
4. The patient has a significant co-morbidity(ies) that might affect study data or confound the study results (e.g., malignancies, hepatic cirrhosis, autoimmune liver disease, significant infectious disease, etc.).
5. The patient is unable to comply with the protocol, e.g., has a clinically relevant medical condition making implementation of the protocol difficult, has an uncooperative attitude, is unable to return for safety evaluations, is otherwise unlikely to complete the study, or continued treatment with GA-GCB is not in the best interest of the patient, as determined by the Investigator.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified