Preliminary study of umbilical cord blood transplantation conditioned with TMLI for refractory severe aplastic anemia

Status

Recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR2000041536

Enrollment

Unknown

Registered

2020-12-27

Start date

2021-01-01

Completion date

Unknown

Last updated

2021-11-02

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

refractory severe aplastic anemia

Interventions

Trial group:umbilical cord blood transplantation conditioned with TMLI

Eligibility

Sex/Gender

All

Age

14 Years to 50 Years

Inclusion criteria

Inclusion criteria: 1.Patients aged 14-50 years, male or female. 2.Patients with severe aplastic anemia diagnosed by the Chinese Expert Consensus on the Diagnosis and Treatment of AA (2017 version). 3.Patients who have been treated with immunosuppressants (including cyclosporine) for more than 6 months and have failed. 4.Patients without HLA-matched sibling donor. 5.Patients who have given their consent and signed the informed consent form.

Exclusion criteria

Exclusion criteria: 1. Patients with severe cardiopulmonary, hepatic or renal insufficiency: pulmonary edema, pleural effusion; LVEF 35 µmol/L, AST/ALT more than 2 times the upper limit of normal values; blood creatinine > 150 µmol/L. 2. Presence of any uncontrollable active infection. 3. Recurrent or progressive malignancy. 4. Infectious diseases (e.g. HAV, HBV, HCV and TB patients with active disease, HIV positive patients, etc.). 5. Presence of fixed, irreversible defects that are not expected to be improved by the study protocol (including pulmonary infiltrates, contractures, etc.) 6. Substance abusers. 7. Those participating in any clinical trial within 3 months 8. Those with contraindications to study interventions or unable to understand or comply with the study protocol 9. Patients who are unable to complete the treatment plan and follow-up observations. 10. Patients who, in the judgment of the investigator, do not meet the requirements of the study or are otherwise unable to participate in the study.

Design outcomes

Primary

Measure	Time frame
1 year overall survival;	—

Secondary

Measure	Time frame
incidence of neutrophil engraftment;incidence of platelet engraftment;incidences of acute graft-vs-host-disease (aGVHD);incidences of chronic graft-vs-host-disease (cGVHD);1 year disease-free survival (DFS);	—

Countries

China

Contacts

Public ContactHuilan Liu

The First Affiliated Hospital of USTC, Division of Life Sciences and Medcine, University of Science and Technology of China

huilanl@ustc.edu.cn+86 551-62283347

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026