T-Cell Depleted Alternative Donor Bone Marrow Transplant for Sickle Cell Disease (SCD) and
The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor st
| Condition(s) | Sickle Cell Anemia, Beta-thalassemia Major, Diamond-blackfan Anemia |
|---|---|
| Status | Recruiting |
| Phase | Phase 1, Phase 2 |
| Study type | Interventional |
| Summary | The purpose of this study is to evaluate what effect, if any, mismatched unrelated volunteer donor and/or haploidentical related donor stem cell transplant may have on severe sickle cell disease and other transfusion dependent anemias. By using mismatched unrelated volunteer donor and/or haploidentical related donor stem cells, this study will increase the number of patients who can undergo a stem cell transplant for their specified disease. Additionally, using a T-cell depleted approach should reduce the incidence of graft-versus-host disease which would otherwise be increased in a mismatched transplant setting. |
| Who can participate | Inclusion Criteria 1. Patient, parent, or legal guardian must have given written informed consent and/or assent according to FDA guidelines. 2. Ages 5 years to 40 years, at time of consent. 3. Diagnosis of Sickle Cell Disease (Hemoglobin SS, Sβ0-thalassemia) complicated by any of the following: * Recurrent acute painful episodes (also known as vaso-occlusive crises; VOC) despite supportive care, minimum of 2 new pain events per year requiring hospitalization for parenteral pain management in the previous 2 years. * Recurrent acute chest syndrome (ACS) despite supportive care, minimum of 2 episodes in preceding 2-year period. * Stroke or neurologic event lasting \> 24 hours with an accompanying infarct on MRI in any patient for all ages; Brain MRI with silent infarct without clinical event |
| Ages | 5 Years to 40 Years |
| Sex | All |
| Lead sponsor | Paul Szabolcs |
| Locations | Pittsburgh, Pennsylvania, United States |
| Start date | 2018-08-02 |
| NCT ID | NCT03653338 |
| Official listing | https://clinicaltrials.gov/study/NCT03653338 |