An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease

Gaucher Disease, Type 1 Clinical Trial

Official title:

An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00635427
• Other study ID #: HGT-GCB-044
• Secondary ID: 2008-001965-27
• Status: Completed
• Phase: Phase 3
• Start date: March 13, 2008
• Est. completion date: December 28, 2012

Study information

• Verified date: May 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

The purpose of this study is to evaluate the long-term safety of every other week dosing of Gene-Activated® human glucocerebrosidase (GA-GCB, velaglucerase alfa) intravenously in patients with type 1 Gaucher disease.

Description:

Type 1 Gaucher disease, the most common form,accounts for more than 90% of all cases and does not involve the CNS. Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (GA-GCB,velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. GA-GCB contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the long-term safety of GA-GCB in men, women, and children with Type 1 Gaucher disease.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 95
• Est. completion date: December 28, 2012
• Est. primary completion date: December 28, 2012
• Accepts healthy volunteers: No
• Gender: All
• Age group: 2 Years and older

Eligibility

Inclusion Criteria:

1. The patient has completed study TKT032 or TKT034, or 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 and must have negative results to a pregnancy test performed at the time of enrollment and as required throughout their participation in the study.

3. 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.

4. 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).

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

Exclusion Criteria:

1. The patient has received treatment with any non-Gaucher disease-related investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted.

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 comorbidity(ies) that might affect study data or confound the study results (e.g., malignancies, primary biliary cirrhosis, autoimmune liver 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, or is otherwise unlikely to complete the study, as determined by the Investigator

Related Conditions & MeSH terms

• Gaucher Disease
• Gaucher Disease, Type 1

Intervention

Biological:
• VPRIV®
Intravenous infusion, every other week (EOW)

Locations

Country	Name	City	State
Argentina	Your Health S.A.	Buenos Aires
India	All India Institute of Medical Sciences	New Delhi
India	KEM Hospital	Pune
Israel	Shaare Zedek Medical Center	Jerusalem
Korea, Republic of	Gyeongsang National University Hospital	Jinju	Gyeongsangnam-do
Paraguay	Sociedad Espanola de Socorros Mutuos	Asuncion
Poland	Instytut "Pomnik-Centrum Zdrowia Dziecka"	Warszawa
Russian Federation	State Institution "Hematology Research Centre RAMS"	Moscow
Spain	Hospital Universitario Miguel Servet	Zaragoza
Tunisia	Hospital de La Rabta	Tunis	Jebbari
United Kingdom	Royal Free Hospital	London
United States	Emory University School of Medicine	Atlanta	Georgia
United States	Children's Memorial Hospital	Chicago	Illinois
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Children's Mercy Hospitals & Clinics	Kansas City	Missouri
United States	Los Angeles Medical Center	Los Angeles	California
United States	Children's Hospital of Wisconsin	Milwaukee	Wisconsin
United States	Children's Hospitals and Clinics of Minnesota	Minneapolis	Minnesota
United States	NYU Medical Center	New York	New York
United States	Children's Hospital Oakland	Oakland	California
United States	University of Utah Medical Center	Salt Lake City	Utah

Sponsors (1)

Lead Sponsor	Collaborator
Shire

Countries where clinical trial is conducted

United States,  Argentina,  India,  Israel,  Korea, Republic of,  Paraguay,  Poland,  Russian Federation,  Spain,  Tunisia,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Summary of Treatment Emergent Adverse Events	Safety was evaluated by an analysis of adverse events (AEs), concomitant medication use, clinical laboratory tests, vital signs during the infusion of study drug, physical examination, and the development of anti-velaglucerase alfa. No formal comparisons or statistical tests were applied for the safety analyses, including for differences between the groups.	Baseline to termination of study
Secondary	Change From Baseline to 24 Months in Hemoglobin Concentration for Each Treatment Group		Baseline to 24 months
Secondary	Change From Baseline to 24 Months in Platelet Counts for Each Treatment Group		Baseline to 24 months
Secondary	Change From Baseline to 24 Months in Normalized Liver Volume for Each Treatment Group		Baseline to 24 months
Secondary	Percentage Change From Baseline to 24 Months in Normalized Spleen Volume for Each Treatment Group		Baseline to 24 months