Actively Recruiting
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Led by Northside Hospital, Inc. · Updated on 2026-04-20
20
Participants Needed
2
Research Sites
624 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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.
CONDITIONS
Official Title
Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Availability of a 3/6 to 5/6 matched related donor with negative HLA cross-match in the host vs. graft direction
- Age 1 to 65 years for previously treated patients and up to 75 years for others
- Karnofsky Performance Status (KPS) of 70% or higher
- Diagnosis of severe aplastic anemia meeting at least two of the following peripheral blood criteria: neutrophils less than 500/mm3, platelets less than 20,000, or absolute reticulocyte count less than 40,000/microL
- Bone marrow must be either markedly hypocellular (less than 25% normal cellularity) or moderately hypocellular with 70% non-myeloid precursors and meeting the peripheral blood criteria above
You will not qualify if you...
- Poor heart function with left ventricular ejection fraction less than 40%
- Poor lung function with FEV1 and FVC less than 50% predicted
- Poor liver function with bilirubin 2 mg/dL or higher
- Poor kidney function with creatinine 2.0 mg/dL or higher or creatinine clearance less than 40 mL/min
- Previous allogeneic transplant
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 2 locations
1
Blood and Marrow Transplant Group of Georgia
Atlanta, Georgia, United States, 30342
Actively Recruiting
2
Northside Hospital
Atlanta, Georgia, United States, 30342
Actively Recruiting
Research Team
M
Melhem Solh, MD
CONTACT
S
Stacey Brown
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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