Severe Aplastic Anemia Clinical Trial
Official title:
Conditioning Regimen Containing Low Dose ATG for The Treatment of Acquired Severe Aplastic Aplasia Receiving Single Cord Blood Transplant: A Multi-center, Single-arm Study
NCT number | NCT06039436 |
Other study ID # | CBSAA001 |
Secondary ID | |
Status | Recruiting |
Phase | |
First received | |
Last updated | |
Start date | October 2023 |
Est. completion date | May 31, 2025 |
To evaluate the safety and efficacy of Single Umbilical Cord blood transplantation (sUCBT) containing low dose ATG based conditioning regimen in the treatment of acquired Severe Aplastic Anemia (SAA).
Status | Recruiting |
Enrollment | 72 |
Est. completion date | May 31, 2025 |
Est. primary completion date | April 30, 2025 |
Accepts healthy volunteers | No |
Gender | All |
Age group | 1 Year to 50 Years |
Eligibility | Inclusion Criteria: - Patients diagnosed with severe aplastic anemia who do not respond to initial diagnosis or immunosuppressive therapy; - Patients meet diagnostic criteria for severe aplastic anemia (Camitta criteria); - Age from 1 to 50 years old, male or female; - Patients without HLA-matched sibling or unrelated donors avaliable; - Patients who have undergone unrelated cord blood transplantation need to have HLA high-resolution matching?TNC (before cord blood freezing)=3.0×107/kg?CD34+(before cord blood freezing)=1.5×107/kg?tubule recovery rate=80%; - patients and their family members or guardians agreed to the study and signed informed consent; - No severe organ failure; - ECOG score=2 and HCT-CI=2; - serum ferritin (SF)<1000ug/L; - women of reproductive agemust have a negative pregnancy test and agree to use effective contraception during treatment and for one year thereafter. Exclusion Criteria: - patients with inherited bone marrow failure syndromes; - Positive for HLA-A, B, Cw, DRB1, DQB1, DPB1 site-specific HLA antibodies for donor cord blood with solid-phase fluorescence assay; - patients who have previously received allogeneic hematopoietic stem cell transplantation or solid organ transplantation; - Uncontrolled bacterial, viral, or fungal infections; - HIV infection or active viral hepatitis B or C infection; - Pregnant or breastfeeding women; - patients with a history of primary malignancy within 3 years prior to transplant treatment; - patients receiving ATG treatment within 2 weeks before transplantation; - patients with drug dependent or uncontrolled mental illness or cognitive impairment; - Participants in other similar clinical studies within 3 months; - Patients allergic to ATG; - patients considered unsuitable for inclusion by the researchers. |
Country | Name | City | State |
---|---|---|---|
China | The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital) | Hefei | Anhui |
Lead Sponsor | Collaborator |
---|---|
Anhui Provincial Hospital |
China,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | The one-year probability of GRFS | GRFS is defined as survival without grade III-IV acute GVHD immunosuppression requiring chronic GVHD or relapse. | one year | |
Secondary | The cumulative incidence of platelet engraftment at 28 days after transplantation | Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10^9/L. | 28 days | |
Secondary | The cumulative incidence of neutrophil engraftment at 28 days after transplantation | Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10^9/L for three consecutive days. | 28 days | |
Secondary | Time of platelet engraftment | Platelet engraftment is defined as independent from platelet transfusion for at least 7 days with a platelet count of more than 20 × 10^9/L. | 28 days | |
Secondary | Time of neutrophil engraftment | Neutrophil engraftment is defined as neutrophil count achieves 0.5 ×10^9/L for three consecutive days. | 28 days | |
Secondary | The cumulative incidence of acute GVHD at 100 days after transplantation | Acute GVHD is defined that occurs within 100 days after transplantation | 100 days | |
Secondary | The cumulative incidence of chronic GVHD at 100 days after transplantation | Chronic GVHD is defined that occurs 100 days after transplantation | one year | |
Secondary | Immune reconstitution after transplantation | The level of immunoglobulin, T cells, B cells and NK cells reconstitution after transplantation | one year | |
Secondary | The 180-day probability of TRM | TRM(transplant-related mortality) is defined that death due to any transplant-related cause within 180 days after transplantation | 180 days | |
Secondary | The incidence of infection | Infection is defined as various post-transplant associated infections | one year | |
Secondary | The one-year probability of OS | OS (overall survival) is defined as the time which begins at diagnosis and up to the time of death | one year | |
Secondary | The one-year probability of DFS | DFS (disease free survival) is defined as the time from treatment until the recurrence of disease (or death) after transplantation | one year |
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