Allogeneic Hematopoietic Stem Cell Transplant for Patients With Inborn Errors of Immunity
Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications. Objective: To see if stem cell transplant can be suc
| Condition(s) | Lymphoproliferative Disorders, Autoimmune Lymphoproliferative, Immune System Diseases, Common Variable Immunodeficiency, Primary T-cell Immunodeficiency Disorders |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | Background: During a transplant, blood stem cells from one person are given to someone else. The cells grow into the different cells that make up the immune system. This can cure people with certain immunodeficiencies. But transplant has many risks and complications. Objective: To see if stem cell transplant can be successfully performed in people with primary immunodeficiency disease and cure them. Eligibility: People ages 4-69 for whom a primary immunodeficiency (PID) or Primary Immune Regulatory Disorder (PIRD), has caused significant health problems and either standard management has not worked or there are no standard management options, along with their donors Design: Donors will be screened under protocol 01-C-0129. They will donate blood or bone marrow. Participants will be screene |
| Who can participate | * INCLUSION CRITERIAl: * Age \>= 4 years and \<=69 yo with Weight \>=12 kilograms * Mutation in a known monogenic (IEI) gene performed by a CLIA certified laboratory, who have failed standard medical management, or when no standard medical management is available. OR Patients without a known IEI mutation may be eligible if they have a clinical history that is characteristic of an individual with an immune defect including a history of infections requiring prolonged courses of therapy or evidence of immune dysregulation manifested by autoimmune/autoinflammatory disease, atopy, hemophagocytic lymphohistiocytosis, hypogammaglobulinemia, or impaired response to vaccination. A virally-driven malignancy alone will also constitute basis for inclusion. * Availability of an 8/8, 7/8, or 6/8 HLA-mat |
| Ages | 4 Years to 69 Years |
| Sex | All |
| Lead sponsor | National Cancer Institute (NCI) |
| Locations | Bethesda, Maryland, United States |
| Start date | 2020-09-22 |
| NCT ID | NCT04339777 |
| Official listing | https://clinicaltrials.gov/study/NCT04339777 |