X'e Bağlı Ağır Kombine İmmün Yetmezliğin (XSCID) Tedavisinde Kök Hücre Gen Terapisi

Sade dilde özet

Bu çalışma, X'e bağlı Ağır Kombine İmmün Yetmezlik (X-Linked Severe Combined Immunodeficiency, XSCID) tedavisinde bir kök hücre gen terapisini (geni aktarılmış CD34+ kök hücreler) inceliyor. IL2RG geninde mutasyonu olan, 1,5-20 yaş arası hastalar dahil edilmiştir. Faz 1 aşamasında yürütülmüş ve tamamlanmıştır.

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

Deney bilgileri

Faz

Faz 1 (Phase 1)

Durum

Tamamlandı

Sponsor

National Institute of Allergy and Infectious Diseases (NIAID)

Müdahale

Gene-Transduced Autologous CD34+ Stem Cells

Başlangıç

10.12.2001

Tahmini bitiş

25.07.2011

Ülkeler

United States

İlgili genler

Merkezler

• Bethesda, United States

Uygunluk kriterleri

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

* INCLUSION CRITERIA:

Patients must have XSCID as defined by either a deleterious mutation in IL2RG, the absence of or less than 5% of normal detectable gc protein, or evidence of functionally defective gc protein.

Patients must be between 1.5 and 20 years of age.

Patients must weigh at least 12 kg.

Patients will have evidence of combined B-cell and T-cell immune deficiency over at least a 6 month period despite previous allogeneic BMT at least 12 months prior to study entry. T-cell immune deficiency is defined as one or more of the following: Total T-cell count less than 500/ul; less than 50% of normal value for in vitro mitogen stimulation; or absent proliferation in vitro to antigens. B-cell immune deficiency is defined as one or more of the following: IgM, IgA or IgE values which are 2 or more standard deviations below the established value for normal, IgG values falling to less than 30% of normal during unintended interruptions or delay in the periodic administration of IVIG; or documented failure to respond to a specific antigen challenge.

Patients must have less than or equal to 3% of their mobilized CD34+ cells deriving from their allogeneic bone marrow donor.

Willingness to remain hospitalized for several days to several weeks.

Have a primary care physician at home.

Consent to permit blood and/or tissue samples for storage.

EXCLUSION CRITERIA:

Any current or preexisting hematologic malignancy.

Current treatment with any chemotherapeutic agent.

Current treatment with any immunosuppressive agent, excluding corticosteroids.

Documented HIV-1 infection.

Documented Hepatitis B infection.

Childhood malignancy (occurring before 18 years of age) in the patient or a first degree relative, or known genotype of the subject conferring a predisposition to cancer.