A Multicenter, Randomized, Open-Label Study of Haplo-Cord HSCT for the Treatment of Aplastic Anemia

NCT07499284 | Recruiting DMC

• 1 other identifier: SOOCHOW-WXJ-2025-1075
• Study Type: interventional
• Target: 224
• Locations: 1 country
• Sites: 4

Brief Summary

Aplastic anemia (AA) is a bone marrow failure disorder characterized by pancytopenia and hypoplastic bone marrow caused by the decrease of hematopoietic stem cells. The pathogenesis of AA is complex and involves an abnormal hematopoietic microenvironment, hematopoietic stem cell/progenitor cell deficiencies and immunity disorders. Currently, the standard treatment for AA includes immunosuppressive therapy (IST) based on anti-thymocyte/lymphocyte globulin (ATG/ALG) and cyclosporine A (CsA) or hematopoietic stem cell transplantation (HSCT). Although HLA-identical sibling allogeneic hematopoietic stem cell transplantation is considered the preferred transplant option for patients with severe aplastic anemia (SAA), only less than 30% of patients have an available HLA-matched sibling donor. In recent years, haploidentical hematopoietic cell transplantation (Haplo-HCT) has developed rapidly and has become an important alternative. However, graft failure and graft-versus-host disease (GVHD) remain significant factors limiting its efficacy. Umbilical cord blood (UCB) contains a diverse population of hematopoietic stem cells. Compared with other sources, cord blood-derived hematopoietic stem cells are more primitive, more viable, and possess higher proliferative capacity. Therefore, cord blood transplantation, with its notable clinical therapeutic effects, has become an effective and reliable alternative to peripheral blood or bone marrow transplantation. Currently, some transplant centers worldwide have adopted the coinfusion of UCB units with haplo-HCT (haplo-cord HCT) achieving preliminary efficacy in promoting engraftment and reducing the incidence of GVHD. A retrospective comparative study of haplo-cord HCT versus IST in patients with SAA identified haplo-cord HCT as the sole independent predictor for superior health-related quality of life (HRQoL) (P \< 0.0001). Based on existing research and clinical experience, this study plans to investigate and further evaluate the safety and efficacy of haplo-cord HCT in the treatment of aplastic anemia. Primary endpoints will include overall survival, engraftment rate, disease-free survival, incidence of GVHD, CMV/EBV reactivation rate, donor chimerism dynamics, and immune reconstitution.

Conditions

Aplastic Anaemia

Keywords

Haplo-Cord HCT; Haplo HCT; Aplastic Anemia

Outcome Measures

Primary Outcomes (1)

• Overall survival rate

  We estimated OS from the time of transplant until the date of death of any cause or last follow-up for patients still alive.

  within 1 year following HSCT

Secondary Outcomes (3)

• The cumulative incidence of neutrophil engraftment and platelet engraftment

  on day 28+7 following HSCT

• Disease Free Survival

  within 1 year following HSCT

• The cumulative incidence and grade of graft-versus-host disease (GVHD)

  within 1 year following HSCT

Study Arms (2)

Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT

EXPERIMENTAL

Procedure: Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells

Comparator group: 112 patients with aplastic anemia undergo haplo HCT

ACTIVE COMPARATOR

Procedure: Haploidentical hematopoietic cell transplantation

Interventions

Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells PROCEDURE

1\. Donor Stem Cells Infusion (Haploidentical hematopoietic cell transplantation combined with unrelated cord blood stem cells) Day 0: Intravenous infuse a single unit of unrelated cord blood stem cells( TNC≤2×10⁷/kg, CD34+ cells≤0.8×10⁵/kg, HLA match≥5/10 loci, and either matched ABO type or a cord blood unit of type O). This is followed, 6 hours after the cord blood infusion, by the infusion of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Starting two days before transplantation (Day -2):Aciclovir (ACV): 250mg/㎡/8h.

Experimental group: 112 patients with aplastic anemia undergo haplo-cord HCT

Haploidentical hematopoietic cell transplantation PROCEDURE

1\. Donor Stem Cell Infusion (Haploidentical hematopoietic cell transplantation) Day 0: Intravenous infuse of donor haploidentical stem cells (TNC≥8×10⁸/kg, CD34+ cells≥4.0×10⁶/kg, and HLA match≥6/12 loci). 2. Conditioning Regimen: Day -7 to Day -6: Busulfan (Bu) 3.2mg/kg/day; Day -5 to Day -2: Cyclophosphamide (CTX) 160-200mg/kg. 3. Graft-versus-Host Disease Prophylaxis Regimen: Starting on the day before transplantation (Day -1): Cyclosporin A (CsA) 3mg/kg/day; Day +1 to Day +28: Mycophenolate Mofetil (MMF) 600mg/㎡/12h. 4. Infection Prophylaxis Regimen: Day -7 to Day -3: Ganciclovir (GCV): 5mg/kg/12h; Continuous medication starting from two days before transplantation (Day -2): Aciclovir (ACV): 250mg/㎡/8h.

Comparator group: 112 patients with aplastic anemia undergo haplo HCT

Eligibility Criteria

• Age: 14 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

Sponsors & Collaborators

• The First Affiliated Hospital of Soochow University: lead

Study Sites (4)

Hematology Department, The First Affiliated Hospital of Soochow University

Suzhou, Jiangsu, China 215006, China

RECRUITING

Suzhou Hongci Hematology Hospital

Suzhou, Jiangsu, China 215006, China

RECRUITING

The Affiliated Hospital of Xuzhou Medical University

Xuzhou, Jiangsu, China 221006, China

RECRUITING

The First Affiliated Hospital of Zhejiang Chinese Medical University

Hangzhou, Zhejiang, China 310006, China

RECRUITING

MeSH Terms

Conditions

Anemia, Aplastic

Condition Hierarchy (Ancestors)

Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Bone Marrow Failure Disorders; Bone Marrow Diseases

Study Officials

• Depei Wu

  The First Affiliated Hospital of Soochow University

  STUDY CHAIR

• Xiaojin Wu

  The First Affiliated Hospital of Soochow University

  STUDY CHAIR

Central Study Contacts

Xiaojin Wu

CONTACT

+8613057493105; wuxiaojin@suda.edu.cn

Depei Wu

CONTACT

+8613951102021; wudepei@suda.edu.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 24, 2026
• First Posted: March 30, 2026
• Study Start: October 1, 2025
• Primary Completion (Estimated): September 1, 2028
• Study Completion (Estimated): September 30, 2028
• Last Updated: March 30, 2026

Record last verified: 2025-12

Data Sharing

• IPD Sharing: Will not share

Locations

CN (4)