Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Recruiting now · Phase 2
Conditions studied: Severe Aplastic Anemia
In brief
Severe aplastic anemia is a rare and serious form of bone marrow failure related to an immune-mediated mechanism that results in severe pancytopenia and high risk for infections and bleeding. Patients with matched sibling donors for transplantation have a 80-90% chance of survival; however, a response rate with just immunosuppression for those patients lacking suitable HLA-matched related siblings is only 60%. With immunosuppression, only 1/3 of patients are cured, 1/3 are dependent on long term immunosuppression, and the other 1/3 relapse or develop a clonal disorder. Recent studies have shown that using a haploidentical donor for transplantation has good response rates and significantly lower rates of acute and chronic GVHD.
Key facts
- Study ID
- NCT02828592
- Run by
- Northside Hospital, Inc.
- People needed
- 20
- Starts
- 2016-09-09
- Expected to finish
- 2028-08-31
- Last updated by the study team
- 2026-04-20
Who can join
Age: 1 and older, up to 75. Sex: any. Healthy volunteers: not accepted.
You may qualify if…
- Availability of 3/6 - 5/6 matched (HLA-A, B, DR) related donor who must have negative HLA cross-match in the host vs. graft direction
- Age <= 65 years for previously treated and <= 75 years for previously treated patients
- KPS >= 70%
- Aplastic Anemia that meets the following criteria:
- Peripheral Blood (must fulfill 2 of 3):
- <500 PMN/mm3
- <20,000 platelets
- absolute reticulocyte count <40,000/microL
- Bone Marrow (must be either):
- markedly hypocellular (<25% of normal cellularity)
- moderately hypocellular with 70% non-myeloid precursors and patient meets peripheral blood criteria above
You may not qualify if…
- poor cardiac function (LVEF <40%)
- poor pulmonary function (FEV1 \& FVC <50% predicted)
- poor liver function (bili >= 2mg/dL)
- poor renal function (creatinine >= 2.0mg/dL or creatinine clearance <40mL/min)
- prior allogeneic transplant
Where it is running
- Blood and Marrow Transplant Group of Georgia — Atlanta, Georgia, United States (enrolling)
- Northside Hospital — Atlanta, Georgia, United States (enrolling)
Full record on ClinicalTrials.gov
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