Haploidentical HCT for Severe Aplastic Anemia
This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched
| Condition(s) | Aplastic Anemia, Bone Marrow Failure Syndrome |
|---|---|
| Status | Recruiting |
| Phase | Phase 2 |
| Study type | Interventional |
| Summary | This study is a prospective, single center phase II clinical trial in which patients with Severe Aplastic Anemia (SAA) ) will receive a haploidentical transplantation. The purpose of this study is to learn more about newer methods of transplanting blood forming cells donated by a family member that is not fully matched to the patient. This includes studying the effects of the chemotherapy, radiation, the transplanted cell product and additional white blood cell (lymphocyte) infusions on the patient's body, disease and overall survival. The primary objective is to assess the rate of engraftment at 30 days and overall survival (OS) and event free survival (EFS) at 1 year post-hematopoietic cell transplantation (HCT). Primary Objectives * To estimate the rate of engraftment at 30 days after T |
| Who can participate | Inclusion Criteria for Transplant Recipient 1. Age less than or equal to 21 years at time of enrollment. 2. Confirmed diagnosis of SAA or a single lineage cytopenia (a) SAA or single lineage cytopenia will be defined as follows: * i. Bone marrow cellularity \< 25% or hypocellular marrow for age, AND * ii. One or more of the following (in peripheral blood): (i) Neutrophils \< 0.5 x10\^9/L (ii) Platelets \< 20 x10\^9/L, or platelet transfusion dependence (iii) Hemoglobin \<8g/dL, or red blood cell transfusion dependence 3. Does not have a suitable HLA-matched sibling donor (MSD) or volunteer 10/10 HLA-matched unrelated donor (MUD) available in the necessary time for progenitor cell donation. 4. Failed at least one trial of immunosuppressive therapy (IST) by being refractory (persistence of s |
| Ages | 21 Years |
| Sex | All |
| Lead sponsor | St. Jude Children's Research Hospital |
| Locations | Memphis, Tennessee, United States |
| Start date | 2021-01-21 |
| NCT ID | NCT04558736 |
| Official listing | https://clinicaltrials.gov/study/NCT04558736 |