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

Completed · Phase 3

Conditions studied: Gaucher Disease, Type 1

In brief

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.

Key facts

Study ID

NCT00635427

Run by

Shire

People needed

95

Starts

2008-03-13

Expected to finish

2012-12-28

Last updated by the study team

2021-06-10

Who can join

Age: 2 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• The patient has completed study TKT032 or TKT034, or study HGT-GCB-039.
• 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 agree to use a medically acceptable method of contraception at all times during the study and report a partner's pregnancy to the investigator.
• 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

You may not qualify if…

• 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.
• The patient is pregnant or lactating.
• 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.
• 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.).
• 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

Where it is running

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

Full record on ClinicalTrials.gov

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