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Complete DiGeorge Anomaly clinical trials

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NCT ID: NCT05329935 Recruiting - Clinical trials for Complete DiGeorge Syndrome

Congenital Athymia Patient Registry

Start date: May 25, 2022
Phase:
Study type: Observational

This Congenital Athymia Patient Registry is an observational exposure-based registry study. It uses a prospective cohort design to follow patients who have been treated with RETHYMIC. Clinical studies conducted with investigational RETHYMIC showed that treatment can result in immune reconstitution and prolong life. This treatment-based registry is being conducted to learn more about the reconstitution process following treatment and the impact of treatment on longer-term survival and the occurrence of adverse events of special interest (AESI).

NCT ID: NCT01220531 Approved for marketing - DiGeorge Syndrome Clinical Trials

Thymus Transplantation Safety-Efficacy

Start date: n/a
Phase:
Study type: Expanded Access

Complete DiGeorge anomaly (cDGA) is a disorder in which there is no thymus function. With no thymus function, bone marrow stem cells do not develop into educated T cells, which fight infection. Without successful treatment, patients with cDGA must remain in reverse isolation to prevent infection and subsequent death. Cultured thymus tissue with and without immunosuppression (drugs given before and after implantation) has resulted in the development of good T cell function in subjects with complete DiGeorge anomaly. This expanded access study continues cultured thymus tissue safety and efficacy research for the treatment of complete DiGeorge anomaly. Eligible participants receive cultured thymus tissue. Immune function testing is continued for one year post-implantation.

NCT ID: NCT00579709 Completed - DiGeorge Syndrome Clinical Trials

Thymus Transplantation With Immunosuppression

884
Start date: July 2002
Phase: Phase 1
Study type: Interventional

The research purpose is to determine if thymus transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly. The research includes studies to evaluate whether thymus transplantation results in complete DiGeorge anomaly subjects developing a normal immune system.

NCT ID: NCT00579527 Completed - Clinical trials for Complete DiGeorge Syndrome

Phase I/II Thymus Transplantation With Immunosuppression #950

#950
Start date: December 19, 2005
Phase: Phase 1/Phase 2
Study type: Interventional

The study purpose is to determine if cultured thymus tissue implantation (CTTI) (previously described as transplantation) with tailored immunosuppression based on the recipient's pre-implantation T cell population is a safe and effective treatment for complete DiGeorge anomaly. This study will also evaluate whether cultured thymus tissue implantation and parathyroid transplantation with immunosuppression is a safe and effective treatment for complete DiGeorge anomaly and hypoparathyroidism.

NCT ID: NCT00576836 Completed - DiGeorge Syndrome Clinical Trials

Thymus Transplantation Dose in DiGeorge #932

Start date: September 2, 2004
Phase: Phase 2
Study type: Interventional

One purpose of this study is to determine whether the amount of cultured thymus tissue implanted into DiGeorge anomaly infants has any effect on the immune outcome. Another purpose of this study is to determine whether parental parathyroid transplantation (in addition to cultured thymus tissue implantation (CTTI) can help both the immune and the calcium problems in DiGeorge infants with hypocalcemia. [Funding Source - FDA Office of Orphan Products Development (OOPD)]