An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID
Enrolling by invitation · NCT04049084 · Registry updated 2025-12-18Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| An Observational LTFU Study for Patients Previously Treated With Autologous ex Vivo Gene Therapy for ADA-SCID | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, DNA repair disease, Infant, Newborn, Diseases, Nutritional and Metabolic Diseases, Primary Immunodeficiency Diseases, hereditary disease, immune system disorder, inborn error of immunity, metabolic disease, severe combined immunodeficiency, severe combined immunodeficiency, autosomal recessive, T cell-negative, B cell-negative, NK cell-negative, due to adenosine deaminase deficiency | Enrolling by invitation | Not stated | 2025-12-18 | NCT04049084 |
2 results · Page 1 of 1
Organizations
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| Organizations | Linked trials |
|---|---|
| Great Ormond Street Hospital for Children NHS Foundation Trust | 34 |
| University of California, Los Angeles | 355 |
2 results · Page 1 of 1
Research sites
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| Research sites | City | Country | Linked trials |
|---|---|---|---|
| Mattel Children's Hospital UCLA/Ronald Reagan UCLA Medical Center | Los Angeles | United States | 1 |
| UCL Great Ormond Street Institute of Child Health | London | United Kingdom | 3 |