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Adenosine deaminase deficiency is an inherited disorder that results in severe abnormalities of the immune system and leaves children unable to fight infection. This trial aims to treat adenosine deaminase deficiency patients using gene therapy.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Intravenous infusion of transduced cells | Experimental | Intravenous infusion of transduced cells |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Intravenous infusion of transduced cells | Biological | Intravenous infusion of transduced cells |
|
| Measure | Description | Time Frame |
|---|---|---|
| Immunological reconstitution | Measurement of Immunological reconstitution and Metabolic Correction. 5 year follow up of the last patient enrolled into study | 5 years |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of adverse reactions | Incidence of adverse reactions. 5 year follow up of the last patient enrolled into study | 5 years |
| Molecular characterisation of gene transfer | Molecular characterisation of gene transfer. 5 year follow up of the last patient enrolled into study |
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Inclusion Criteria:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Great Ormond Street Hospital for Children NHS Trust | London | WC1N 1EH | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 12089448 | Background | Aiuti A, Slavin S, Aker M, Ficara F, Deola S, Mortellaro A, Morecki S, Andolfi G, Tabucchi A, Carlucci F, Marinello E, Cattaneo F, Vai S, Servida P, Miniero R, Roncarolo MG, Bordignon C. Correction of ADA-SCID by stem cell gene therapy combined with nonmyeloablative conditioning. Science. 2002 Jun 28;296(5577):2410-3. doi: 10.1126/science.1070104. | |
| 8867866 |
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| ID | Term |
|---|---|
| C531816 | Severe combined immunodeficiency due to adenosine deaminase deficiency |
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| 5 years |
| Normalisation of nutritional status, growth, and development | Normalisation of nutritional status, growth, and development. 5 year follow up of the last patient enrolled into study | 5 years |
| Hoogerbrugge PM, van Beusechem VW, Fischer A, Debree M, le Deist F, Perignon JL, Morgan G, Gaspar B, Fairbanks LD, Skeoch CH, Moseley A, Harvey M, Levinsky RJ, Valerio D. Bone marrow gene transfer in three patients with adenosine deaminase deficiency. Gene Ther. 1996 Feb;3(2):179-83. |
| 7749407 | Background | Hershfield MS. PEG-ADA: an alternative to haploidentical bone marrow transplantation and an adjunct to gene therapy for adenosine deaminase deficiency. Hum Mutat. 1995;5(2):107-12. doi: 10.1002/humu.1380050202. |