Tip 1 Gaucher Hastalığında DRX008A (Enzim Replasman Tedavisi) Alan Hastalarda Uzun Dönem Güvenliği Değerlendiren Açık Etiketli Uzatma Çalışması

Sade dilde özet

Bu çalışma, Tip 1 Gaucher Hastalığı (Gaucher disease) olan ve DRX008A adlı enzim replasman tedavisi (eksik enzimin dışarıdan verilmesi) alan hastalarda uzun dönem güvenliği değerlendiriyor. Önceki bir çalışmayı tamamlamış hastalar dahil edilmiştir. Faz 1/Faz 2 aşamasında yürütülmüş ve tamamlanmıştır.

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

Deney bilgileri

Faz

PHASE1/PHASE2

Durum

Tamamlandı

Sponsor

Shire

Müdahale

GA-GCB

Başlangıç

13.09.2004

Tahmini bitiş

31.01.2008

Ülkeler

Israel, Romania, Serbia

İlgili genler

Merkezler

• Jerusalem, Israel
• Bucharest, Romania
• Belgrade, Serbia

Uygunluk kriterleri

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

Inclusion Criteria:

* Patients who have completed through Week 41 visit in the TKT025 study.
* Patients must have voluntarily signed an IRB/EC approved informed consent form after all relevant aspects of the study have been explained and discussed with the patient.
* Patient must be sufficiently cooperative to participate in this clinical study as judged by the Investigator.
* Female and male patients of child bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Female patients must have a negative serum pregnancy test on enrollment.

Exclusion Criteria:

* Patient has received treatment with non-Gaucher disease related investigational drug or device within the past 30 days prior to study entry; such use during the study is not permitted.
* Patient has a clinically relevant medical condition (e.g., HIV, hepatitis B or C) that would make implementation of the protocol difficult and/or confound an assessment of the effects of the experimental therapy and its adverse events.
* Patient, patient's parent(s), or patient's legal guardian is unable to understand the nature, scope and possible consequences of the study.
* Patient is unable to comply with the protocol, e.g. uncooperative attitude, medical condition, inability to return for safety evaluations, or is otherwise unlikely to complete the study, as determined by the Investigator.