NCT02828592 Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia
| NCT ID | NCT02828592 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Northside Hospital, Inc. |
| Condition | Severe Aplastic Anemia |
| Study Type | INTERVENTIONAL |
| Enrollment | 20 participants |
| Start Date | 2016-09-09 |
| Primary Completion | 2027-08-31 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 20 participants in total. It began in 2016-09-09 with a primary completion date of 2027-08-31.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
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.
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
Contact & Investigator
Melhem Solh, MD
PRINCIPAL INVESTIGATOR
Blood and Marrow Transplant Group of Georgia
Frequently Asked Questions
Who can join the NCT02828592 clinical trial?
This trial is open to participants of all sexes, aged 1 Year or older, up to 75 Years, studying Severe Aplastic Anemia. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT02828592 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT02828592 currently recruiting?
Yes, NCT02828592 is actively recruiting participants. Contact the research team at msolh@bmtga.com for enrollment information.
Where is the NCT02828592 trial being conducted?
This trial is being conducted at Atlanta, United States, Atlanta, United States.
Who is sponsoring the NCT02828592 clinical trial?
NCT02828592 is sponsored by Northside Hospital, Inc.. The principal investigator is Melhem Solh, MD at Blood and Marrow Transplant Group of Georgia. The trial plans to enroll 20 participants.