Clinical Trials Logo

Clinical Trial 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.


Clinical Trial Description

Mismatched haploidentical donors will be identified for patients with severe aplastic anemia. These patients will undergo a preparative regimen of Fludarabine/Cyclophosphamide/TBI followed by haploidentical bone marrow transplantation. Post-transplant Cyclophosphamide will be administered on Days 3 & 4. Immunosuppression with Tacrolimus and MMF will begin on Day +5; MMF will be discontinued on Day +35 while Tacrolimus continues until Day +180. Investigators hypothesize that haploidentical transplantation with the above-mentioned preparative regimen will have a <30% graft failure rate. The one-sided exact Binomial test at 5% significance level will be used to test this hypothesis. The size of 20 patients provides the power of 92.5% for confirming the 30-day graft failure rate <30%. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT02828592
Study type Interventional
Source Northside Hospital, Inc.
Contact Melhem Solh, MD
Phone 404-255-1930
Email msolh@bmtga.com
Status Recruiting
Phase Phase 2
Start date September 9, 2016
Completion date August 31, 2026

See also
  Status Clinical Trial Phase
Completed NCT02833805 - NMA Haplo or MUD BMT for Newly Diagnosed Severe Aplastic Anemia Phase 2
Terminated NCT01319851 - Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation N/A
Completed NCT00004143 - Allogeneic Mixed Chimerism Stem Cell Transplant Using Campath for Hemoglobinopathies & Bone Marrow Failure Syndromes Phase 2
Recruiting NCT05012111 - Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Recruiting NCT03836690 - Transfer of Effector Memory T Cells (Tem) Following Allogeneic Stem Cell Transplantation Phase 1
Recruiting NCT06039436 - Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired SAA Receiving sUCBT
Enrolling by invitation NCT05049668 - RACE 2: a Long Term Follow-up of Patients Participating in the RACE Trial
Recruiting NCT01351545 - A Multicenter Access and Distribution Protocol for Unlicensed Cryopreserved Cord Blood Units (CBUs)
Recruiting NCT01472055 - Pharmacokinetic Study of Fludarabine in Pediatric Hematopoietic Stem Cell Transplantation Phase 2
Completed NCT01703169 - Efficacy and Safety of Eltrombopag In Patients With Severe and Very Severe Aplastic Anemia Phase 2
Withdrawn NCT01129323 - Reduced-Intensity Preparative Regimen for Allogeneic Stem Cell Transplantation in Patients With Severe Aplastic Anemia N/A
Completed NCT00516152 - Phase II Study Evaluating Busulfan and Fludarabine as Preparative Therapy in Adults With Hematopoietic Disorders Undergoing MUD SCT Phase 2
Recruiting NCT06069180 - The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA Phase 4
Recruiting NCT03579875 - Alpha/Beta TCD HCT in Patients With Inherited BMF Disorders Phase 2
Recruiting NCT05720234 - Avatrombopag Combined With IST as First-line Treatment for SAA Phase 2
Recruiting NCT04304820 - Early Initiation of Oral Therapy With Cyclosporine and Eltrombopag for Treatment Naive Severe Aplastic Anemia (SAA) Phase 2
Terminated NCT00358657 - Fludarabine Phosphate, Cyclophosphamide, and Total-Body Irradiation Followed by Donor Bone Marrow Transplant and Cyclophosphamide, Mycophenolate Mofetil, Tacrolimus, and Sirolimus in Treating Patients With Primary Immunodeficiency Disorders or Noncancerous Inherited Disorders Phase 2
Completed NCT02998645 - Eltrombopag Combined With Cyclosporine as First Line Therapy in Patients With Severe Acquired Aplastic Anemia Phase 2
Active, not recruiting NCT03825744 - Hetrombopag or Placebo in Treatment-Naive Severe Aplastic Anemia Phase 3
Not yet recruiting NCT06279494 - Sirolimus+Abatacept+Mycophenolate Mofetil for Prophylaxis of aGVHD in Patients Receiving Haplo-HSCT Who Are Intolerant to Calcineurin Inhibitors Phase 1/Phase 2