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Study of GA-GCB Enzyme Replacement Therapy in Type 1 Gaucher Disease Patients Previously Treated With Imiglucerase
Bu çalışma, daha önce imiglukeraz ile tedavi edilmiş Tip 1 Gaucher Hastalığı (Gaucher disease) olan hastalarda GA-GCB (velaglukeraz alfa) adlı enzim replasman tedavisini inceliyor. En az 30 ay boyunca düzenli imiglukeraz almış hastalar dahil edilmiştir. Faz 2/Faz 3 aşamasında yürütülmüş ve tamamlanmıştır.
Yukarıdaki özet, resmî kaydın sade dile aktarılmış halidir. Orijinal İngilizce başlık referans için gösterilir.
Kriterler ClinicalTrials.gov kaydından orijinal İngilizce haliyle alınmıştır. Uygunluk kararını yalnızca deneyi yürüten ekip verebilir.
Inclusion Criteria: Includes: * The participant has a documented diagnosis of type 1 Gaucher disease, as determined by deficient glucocerebrosidase (GCB) activity relative to normal as measured in leukocytes or by genotype analysis and the participant/legal guardian is willing and able to provide written informed consent prior to initiating any study-related procedures * The participant has received consistent treatment with imiglucerase at a dose ≤ 60 U/kg and ≥ 15 U/kg every other week for a minimum of 30 consecutive months. Participants who are anti-imiglucerase antibody positive will be allowed to enter this study * The participant is at least 2 years of age * Female participants of child-bearing potential agree to use a medically acceptable method of contraception. Male participants must agree to use a medically acceptable method of birth control * Participant must be sufficiently co-operative to participate in the study as judged by the Investigator. Exclusion Criteria: Includes: * The participant has type 2 or 3 Gaucher disease or is suspected of having type 3 Gaucher disease * The participant has received treatment with any investigational drug or device within the 30 days prior to study entry; such use during the study is not permitted * Participant is HIV positive * Participant is hepatitis B/C positive * The participant presents with sustained iron, folic acid and/or vitamin B12 deficiency-related anemia during Screening * The participant, participant's parent(s), or participant's legal guardian(s) is/are unable to understand the nature, scope, and possible consequences of the study * The participant has a significant comorbidity that might affect study data or confound the study results * The participant is unable to comply with the protocol or is otherwise unlikely to complete the study, as determined by the Investigator * The participant has experienced an anaphylactic/anaphylactoid reaction during treatment with imiglucerase * The participant has received miglustat during the 6 months prior to study enrollment * The participant has an active, clinically significant spleen infarction * The participant has active, progressive bone necrosis * The participant is a pregnant and/or lactating female