Haplo‑cord versus haplo hematopoietic stem cell transplant for aplastic anemia
A Multicenter, Randomized, Open-Label Study to Analyze the Efficacy of Unrelated Cord Blood Combined With Haploidentical Stem Cell Transplantation in Patients With Aplastic Anemia
This trial will test whether adding unrelated cord blood to a haploidentical stem cell transplant (haplo‑cord) helps people aged 14 and older with aplastic anemia more than a haploidentical transplant alone.
Quick facts
| Phase | Not applicable |
|---|---|
| Study type | Interventional |
| Enrollment | 224 (estimated) |
| Ages | 14 Years and up |
| Sex | All |
| Sponsor | The First Affiliated Hospital of Soochow University Academic / other |
| Locations | 4 sites (Suzhou, Jiangsu and 3 other locations) |
| Trial ID | NCT07499284 on ClinicalTrials.gov |
What this trial studies
This randomized, open‑label multicenter study compares haplo‑cord hematopoietic stem cell transplantation with standard haploidentical transplantation in patients with aplastic anemia. Participants are adults and adolescents aged 14 years or older who are eligible for allogeneic transplantation and meet performance and comorbidity criteria. The trial measures safety and efficacy outcomes, with particular attention to graft failure, graft‑versus‑host disease, and other adverse events. Sites are located in Jiangsu province, China, and the protocol will collect factors associated with treatment outcomes.
Who should consider this trial
Good fit: Ideal candidates are people aged 14 or older with a diagnosis of aplastic anemia who are eligible for allogeneic hematopoietic stem cell transplantation and have KPS ≥70, ECOG ≤2, HCT‑CI ≤2, and an expected survival of at least three months.
Not a fit: Patients with aplastic anemia caused by prior cancer treatment, those with active malignancy, or those who are medically ineligible for transplant are unlikely to benefit from this protocol.
Why it matters
Potential benefit: If successful, haplo‑cord transplantation could lower graft failure and severe graft‑versus‑host disease rates and improve recovery for patients who lack an HLA‑matched sibling donor.
How similar studies have performed: Haploidentical transplantation has increasingly been used with some success as an alternative donor source, but the specific haplo‑cord combination is less extensively tested and remains investigational.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: (1)Aged ≥14 years. (2) Patients diagnosed with aplastic anemia meeting the diagnostic criteria of the Chinese Guidelines for the Diagnosis and Management of Aplastic Anemia (2024 Edition). (3) Patients who were eligible for allogeneic bone marrow or peripheral blood hematopoietic stem cell transplantation in accordance with the Clinical Application and Management Specifications for Allogeneic Hematopoietic Stem Cell Transplantation Technology (2022 Edition). (4) The patient understands the study protocol and voluntarily signs the informed consent form. (5) Life expectancy of ≥3 months; (6) Pre-transplant assessment meets the following criteria: Karnofsky Performance Status (KPS) score≥70, Eastern Cooperative Oncology Group Performance Status (ECOG PS) score≤2, and Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) score≤2. Exclusion Criteria: (1)Patients with aplastic anemia secondary to malignancy treatment or those with concurrent active malignancy. (2) Women who are pregnant or lactating. (3) Patients with psychiatric or psychological disorders that preclude adequate compliance with the treatment protocol. (4) Patients with positive serologic testing for any of the four major infectious diseases (hepatitis B, hepatitis C, syphilis, HIV). (5) Patients with systemic infection or localized severe infection requiring active antimicrobial therapy. (6) Patients with significant dysfunction of major organs (e.g., heart, lung, liver, kidney). (7) Patients with a known allergy or hypersensitivity to any drug or component used in this study. (8) Patients who are currently participating in or plan to participate in any other clinical trial. (9) Any other condition deemed by the investigator to render the patient unsuitable for study participation.
Where this trial is running
Suzhou, Jiangsu and 3 other locations
- Hematology Department, The First Affiliated Hospital of Soochow University — Suzhou, Jiangsu, China (Recruiting)
- Suzhou Hongci Hematology Hospital — Suzhou, Jiangsu, China (Recruiting)
- The Affiliated Hospital of Xuzhou Medical University — Xuzhou, Jiangsu, China (Recruiting)
- The First Affiliated Hospital of Zhejiang Chinese Medical University — Hangzhou, Zhejiang, China (Recruiting)
Study contacts
- Study coordinator: Xiaojin Wu
- Email: wuxiaojin@suda.edu.cn
- Phone: +8613057493105
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.