Tip 1 Gaucher Hastalığında Gen Aktifleştirilmiş İnsan Glukoserebrosidaz (GA-GCB) Enzim Tedavisinin İmiglukeraz ile Karşılaştırılması

Sade dilde özet

Bu çalışma, Tip 1 Gaucher Hastalığı (Gaucher disease) olan hastalarda gen aktifleştirilmiş insan glukoserebrosidaz (GA-GCB, velaglukeraz alfa) enzim tedavisini imiglukeraz ile karşılaştırıyor. Son 12 ayda Gaucher tedavisi almamış, 2 yaş üstü hastalar dahil edilmiştir. Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.

Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.

Deney bilgileri

Faz

Faz 3 (Phase 3)

Durum

Tamamlandı

Sponsor

Shire

Müdahale

velaglucerase alfa, imiglucerase

Başlangıç

29.01.2008

Tahmini bitiş

05.05.2009

Ülkeler

United States, Argentina, India, Israel, Paraguay, Russia, Spain, Tunisia, United Kingdom

İlgili genler

Merkezler

• Durham, United States
• Buenos Aires, Argentina
• Calicut, India
• New Delhi, India
• Pune, India
• Jerusalem, Israel
• Asunción, Paraguay
• Moscow, Russia
• Zaragoza, Spain
• Tunis, Tunisia
• London, United Kingdom

Uygunluk kriterleri

Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.

Inclusion Criteria

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.

Exclusion Criteria

Includes:

* The patient has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease.
* 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 known to be positive for human immunodeficiency virus (HIV).
* The patient is known to be positive for hepatitis B and/or C.
* 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.