RECRUITING Phase 2 ClinicalTrials.gov

Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia

Sponsor: Northside Hospital, Inc. • ClinicalTrials.gov ID: NCT02828592

Registry updated: 2026-10-14

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Description

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.

Interventions

Fludarabine (Drug)
30 mg/m2 IV QD x 5 days (Days -6 to -2)
Cyclophosphamide (Drug)
14.5 mg/kg/day IV x 2 doses (Days -6 \& -5)
Total Body Irradiation (Radiation)
300 cGy x1 dose (Day -1)
Rabbit ATG (Drug)
1.5 mg/kg/day x 3 days (Days -3 to -1)
Cyclophosphamide (Drug)
Post-transplant: 50 mg/kg IV QD (Day +3 to +4)

Eligibility criteria

Inclusion Criteria: * 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 Exclusion Criteria: * 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

Conditions

Severe Aplastic Anemia

Locations

Blood and Marrow Transplant Group of Georgia — Atlanta, Georgia, United States
Northside Hospital — Atlanta, Georgia, United States

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Official Source

Data sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

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