Haploidentical bone marrow transplant for severe aplastic anemia
A Study of T-Cell Replete, HLA-Mismatched Haploidentical Bone Marrow Transplantation With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Lacking HLA-Matched Related Donor
This study is testing if a special type of bone marrow transplant from a mismatched donor can help people with severe aplastic anemia who don't have a matching donor.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 20 (estimated) |
| Ages | 1 Year to 75 Years |
| Sex | All |
| Sponsor | Northside Hospital, Inc. Academic / other |
| Drugs / interventions | Cyclophosphamide, Fludarabine |
| Locations | 2 sites (Atlanta, Georgia and 1 other locations) |
| Trial ID | NCT02828592 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the effectiveness of haploidentical bone marrow transplantation in patients with severe aplastic anemia who lack suitable HLA-matched donors. Participants will undergo a preparative regimen involving Fludarabine, Cyclophosphamide, and Total Body Irradiation, followed by transplantation from a mismatched donor. Post-transplant, Cyclophosphamide will be administered to reduce the risk of graft failure. The study will assess the graft failure rate and overall patient outcomes to determine the viability of this approach.
Who should consider this trial
Good fit: Ideal candidates include patients aged 65 or younger with severe aplastic anemia who have a suitable haploidentical donor.
Not a fit: Patients with poor cardiac, pulmonary, liver, or renal function, or those who have previously undergone an allogeneic transplant may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new option for patients with severe aplastic anemia who do not have matched sibling donors, potentially improving survival rates.
How similar studies have performed: Other studies have shown promising results with haploidentical transplantation, indicating that this approach may be effective for treating severe aplastic anemia.
Eligibility criteria
Show full inclusion / exclusion 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
Where this trial is running
Atlanta, Georgia and 1 other locations
- Blood and Marrow Transplant Group of Georgia — Atlanta, Georgia, United States (Recruiting)
- Northside Hospital — Atlanta, Georgia, United States (Recruiting)
Study contacts
- Principal investigator: Melhem Solh, MD — Blood and Marrow Transplant Group of Georgia
- Study coordinator: Melhem Solh, MD
- Email: msolh@bmtga.com
- Phone: 404-255-1930
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.