Ignite Creation Date:
2024-10-26 @ 3:35 PM
Last Modification Date:
2024-10-26 @ 3:35 PM
Study NCT ID:
NCT06517641
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-11
Brief Title:
Clinical Trial of Upfront Haploidentical or Unrelated Donor BMT to Restore Normal Hematopoiesis in Aplastic Anemia
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase II Trial of Non-Myeloablative Conditioning and Transplantation of Haploidentical Related Partially HLA-Mismatched or Matched Unrelated Bone Marrow for Newly Diagnosed Patients With Severe Aplastic Anemia
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-07
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
BMT CTN CureAA
Brief Summary:
BMT CTN 2207 will investigate the use of marrow transplantation for treatment of severe aplastic anemia that has not previously been treated
Detailed Description:
This study is a prospective multicenter Phase II study of hematopoietic stem cell transplantation for previously untreated patients with severe aplastic anemia SAA Severe Aplastic Anemia SAA is a rare condition in which the body stops producing enough new blood cells Patients with aplastic anemia have low white blood cells cells which fight infection low red blood cells cells that carry oxygen throughout the body and low platelets cells that help form clots and prevent bleeding Treatments for SAA seeks to repair this abnormal immune system attack and allow the bone marrow to make the normal amount of blood cells This can be done with a bone marrow transplant or with medications to suppress the immune system
Historically transplant therapy for SAA has been reserved for patients under 40 years old who had an available fully matched related donor The standard treatment for older patients with SAA and patients who do not have a fully matched related donor has been treatment using transfusions medications that suppress the immune system immunosuppressive therapy IST and medications that try to stimulate the bone marrow to produce more cells For these patients transplant was used only if a patient did not respond to these interventions However progress has made transplantation safer and allowed for half-matched related donor or full or partially-matched unrelated donors to be used with success rates similar to fully matched related donors in many situations The goals of this study are to determine if patients with SAA who have not received previous treatment for SAA can be treated effectively with transplant as their first SAA therapy
This is a parallel cohort study comprised of two cohorts based on donor selection haploidentical related donors and unrelated donors The accrual goal is 30 participants enrolled and starting protocol-specified conditioning in each cohort yielding 60 participants in total Participants will be treated with a reduced-intensity preparative regimen of fludarabine 150 mgm2 cyclophosphamide 29 mgkg low dose total body irradiation TBI 400 cGy and Thymoglobulin 45 mgkg Bone marrow will be collected from donors and fresh not cryopreserved cells will be given to patients GVHD prophylaxis will be with post-HSCT cyclophosphamide 100 mgkg tacrolimus and mycophenolate mofetil MMF All patients will receive the same conditioning regimen and GVHD prophylaxis Participants will be followed for 1 year post-transplant
Historically transplant therapy for SAA has been reserved for patients under 40 years old who had an available fully matched related donor The standard treatment for older patients with SAA and patients who do not have a fully matched related donor has been treatment using transfusions medications that suppress the immune system immunosuppressive therapy IST and medications that try to stimulate the bone marrow to produce more cells For these patients transplant was used only if a patient did not respond to these interventions However progress has made transplantation safer and allowed for half-matched related donor or full or partially-matched unrelated donors to be used with success rates similar to fully matched related donors in many situations The goals of this study are to determine if patients with SAA who have not received previous treatment for SAA can be treated effectively with transplant as their first SAA therapy
This is a parallel cohort study comprised of two cohorts based on donor selection haploidentical related donors and unrelated donors The accrual goal is 30 participants enrolled and starting protocol-specified conditioning in each cohort yielding 60 participants in total Participants will be treated with a reduced-intensity preparative regimen of fludarabine 150 mgm2 cyclophosphamide 29 mgkg low dose total body irradiation TBI 400 cGy and Thymoglobulin 45 mgkg Bone marrow will be collected from donors and fresh not cryopreserved cells will be given to patients GVHD prophylaxis will be with post-HSCT cyclophosphamide 100 mgkg tacrolimus and mycophenolate mofetil MMF All patients will receive the same conditioning regimen and GVHD prophylaxis Participants will be followed for 1 year post-transplant
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None