Using low dose ATG with umbilical cord blood transplantation for severe aplastic anemia

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

This study is testing if using a low dose of a medication called ATG with umbilical cord blood transplantation can help people with severe aplastic anemia recover better and safely.

Quick facts

• Study type: Observational
• Enrollment: 72 (estimated)
• Ages: 1 Year to 50 Years
• Sex: All
• Sponsor: Anhui Provincial Hospital (other gov)
• Locations: 1 site (Hefei, Anhui)
• Trial ID: NCT06039436 on ClinicalTrials.gov

What this trial studies

This study evaluates the safety and efficacy of a conditioning regimen that includes low dose Anti-Thymocyte Globulin (ATG) for patients with acquired Severe Aplastic Anemia (SAA) undergoing Single Umbilical Cord Blood Transplantation (sUCBT). Given the shortage of HLA-compatible donors, umbilical cord blood serves as an alternative treatment for patients who have not responded to immunosuppressive therapies. The study aims to determine the optimal use of low dose ATG to enhance transplantation efficacy while minimizing adverse effects on immune recovery. It is a multicenter observational study that will gather data on patient outcomes following this treatment approach.

Who should consider this trial

Good fit: Ideal candidates are patients aged 1 to 50 years diagnosed with severe aplastic anemia who have not responded to initial therapies and lack available HLA-matched donors.

Not a fit: Patients with inherited bone marrow failure syndromes or those with severe organ failure may not benefit from this study.

Why it matters

Potential benefit: If successful, this approach could improve transplantation success rates and patient outcomes for those with severe aplastic anemia lacking compatible donors.

How similar studies have performed: While the use of ATG in transplantation has been studied, this specific approach with low dose ATG in umbilical cord blood transplantation for SAA is novel and has not been extensively tested.

Eligibility criteria

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.

Where this trial is running

Hefei, Anhui

• The First Affiliated Hospital of University of Science and Technology of China (Anhui Provincial Hospital) — Hefei, Anhui, China (RECRUITING)

Study contacts

• Principal investigator: Xiaoyu Zhu, ph.D — The First Affiliated Hospital of University of Science and Technology of China (Anhui Provicial Hospital)
• Study coordinator: Xiaoyu Zhu, ph.D

Conditions: Severe Aplastic Anemia, Anti-Thymocyte Globulin, Umbilical Cord Blood Transplantation