Clinical Trials Logo

Clinical Trial Summary

Severe Aplastic Anemia (SAA) is a rare condition in which the body stops producing enough new blood cells. SAA can be cured with immune suppressive therapy or a bone marrow transplant. Regular treatment for patients with aplastic anemia who have a matched sibling (brother or sister), or family donor is a bone marrow transplant. Patients without a matched family donor normally are treated with immune suppressive therapy (IST). Match unrelated donor (URD) bone marrow transplant (BMT) is used as a secondary treatment in patients who did not get better with IST, had their disease come back, or a new worse disease replaced it (like leukemia). This trial will compare time from randomization to failure of treatment or death from any cause of IST versus URD BMT when used as initial therapy to treat SAA. The trial will also assess whether health-related quality of life and early markers of fertility differ between those randomized to URD BMT or IST, as well as assess the presence of marrow failure-related genes and presence of gene mutations associated with MDS or leukemia and the change in gene signatures after treatment in both study arms. This study treatment does not include any investigational drugs. The medicines and procedures in this study are standard for treatment of SAA.


Clinical Trial Description

This study is a multi-center randomized phase III trial to compare the failure free survival between those randomized to IST vs 9-10/10 HLA matched URD BMT. The study will also address patient-reported outcomes and gonadal function in each arm and explore critical biological correlates including assessing germline genetic mutations associated with pediatric SAA that may lead to a predisposition to the disease and the risk of development of clonal hematopoiesis following IST vs BMT in pediatric and young adult SAA. This clinical trial will randomize 234 children/AYA over 3.3-4.7 years at a 1:1 ratio between initial treatment with immune suppression therapy (IST) with horse ATG (hATG)/cyclosporine (CsA) versus well- matched (9-10/10 allele) unrelated donor (URD) bone marrow transplantation (BMT) using a regimen of rabbit ATG (rATG)/fludarabine/cyclophosphamide and 200 cGy TBI. Duration of subject participation for all study procedures in this study will be up to 2 years after treatment; a single later timepoint between 3 and 5 years will be collected to follow patients for specific protocol defined late effects and survival. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT05600426
Study type Interventional
Source Boston Children's Hospital
Contact Leah Cheng, MA
Phone 857-218-4731
Email leah.cheng@childrens.harvard.edu
Status Recruiting
Phase Phase 3
Start date January 25, 2023
Completion date December 2029

See also
  Status Clinical Trial Phase
Recruiting NCT02828592 - Haploidentical Bone Marrow Transplant With Post-Transplant Cyclophosphamide for Patients With Severe Aplastic Anemia Phase 2
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