TCR Alpha Beta T-cell Depleted Haploidentical HCT in the Treatment of Primary Immunodefici
This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other h
| Condition(s) | Primary Immune Deficiency Disorders, Metabolic Disease |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | This research is being done to learn if a new type of haploidentical transplantation using TCR alpha beta and CD19 depleted stem cell graft from the donor is safe and effective to treat the patient's underlying condition. This study will use stem cells obtained via peripheral blood or bone marrow from parent or other half-matched family member donor. These will be processed through a special device called CliniMACS, which is considered investigational. |
| Who can participate | Inclusion Criteria: 1. Patient with any form of primary immune deficiency/dysregulatory disorders characterized by aberrant immune function, abnormal hematopoiesis, systemic or organ specific autoimmunity and/or non-malignant lymphoproliferation. This includes, but not limited to: I. Disorders of phagocytes: Chronic granulomatous disease, Leukocyte adhesion deficiency, defects of IL-10 pathway, MonoMac syndrome II. Defects of cellular and humoral immunity: Severe Combined Immunodeficiency Disorder (infants with classic SCID up to 2 years of age will be excluded due to other open protocol), X-linked hyper-IgM syndrome, DOCK8 deficiency, ZAP70 deficiency, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, NEMO deficiency. III. Disorder of immune dysregulation: Immunodysregula |
| Ages | 21 Years |
| Sex | All |
| Lead sponsor | Johns Hopkins All Children's Hospital |
| Locations | St. Petersburg, Florida, United States |
| Start date | 2020-07-22 |
| NCT ID | NCT04414046 |
| Official listing | https://clinicaltrials.gov/study/NCT04414046 |