Tip 3 Gaucher Hastalığı Olan Çocuk ve Ergenlerde Velaglukeraz Alfa Etkinlik ve Güvenlik Çalışması

Sade dilde özet

Bu çalışma, Tip 3 Gaucher Hastalığı (Gaucher disease) olan çocuk ve ergenlerde Velaglukeraz Alfa adlı enzim replasman tedavisini inceliyor. Tip 3 Gaucher tanısı doğrulanmış, Gaucher'e bağlı kansızlığı olan 2-18 yaş arası 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

velaglucerase alfa

Başlangıç

14.09.2012

Tahmini bitiş

15.03.2015

Ülkeler

Egypt, India, Tunisia

İlgili genler

Merkezler

• Alexandria, Egypt
• Cairo, Egypt
• Cairo, Egypt
• Pune, India
• Tunis, Tunisia

Uygunluk kriterleri

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

Inclusion Criteria: Each patient must meet the following criteria to be enrolled in this study.

1. The patient has a confirmed diagnosis of type 3 Gaucher disease.
2. The patient is ≥ 2 and \< 18 years of age at the time of enrollment.
3. The patient is either näive to treatment or has not received treatment (investigational or approved) for Gaucher disease within 12 months prior to study entry.
4. The patient has Gaucher disease-related anemia, defined as hemoglobin concentration below the lower limit of normal for age and sex.

   AND ONE OR MORE OF THE FOLLOWING THREE CRITERIA
   * The patient has at least moderate splenomegaly (2 to 3 cm below the left costal margin) by palpation.
   * The patient has Gaucher disease-related thrombocytopenia, defined as platelet count \< 120 x 10,000 platelets/cubic mm.
   * The patient has a Gaucher disease-related readily palpable enlarged liver.
5. Patients who have undergone splenectomy may still be eligible to participate in the study.
6. Female patients of child-bearing potential must agree to use a medically acceptable method of contraception at all times during the study. Pregnancy testing will be performed at the time of enrollment and as required throughout 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.
7. The patient's parent(s) or the patient's legally authorized representative(s) has provided written informed consent that has been approved by the Institutional Review Board/Independent Ethics Committee (IRB/IEC).

Exclusion Criteria: Patients who meet any of the following criteria will be excluded from this study.

1. The patient is suspected of having type 2 or type 1 Gaucher disease.
2. The patient is \< 2 years of age.
3. The patient has experienced a severe (Grade 3 or higher) infusion-related hypersensitivity reaction (anaphylactic or anaphylactoid reaction) to any enzyme replacement therapy for Gaucher disease (approved or investigational).
4. The patient has received any non-Gaucher disease-related treatment with an investigational drug within 30 days prior to study entry.
5. The patient is a pregnant and/or lactating female.