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A Prospective Multicenter Study on the TBI-Free Conditioning Regimen for Umbilical Cord Blood Hematopoietic Stem Cell Transplantation in Children and Adolescents with Germline Predisposition-Associated Bone Marrow Failure

A Prospective Multicenter Study on the TBI-Free Conditioning Regimen for Umbilical Cord Blood Hematopoietic Stem Cell Transplantation in Children and Adolescents with Germline Predisposition-Associated Bone Marrow Failure

Status
Active, not recruiting
Phases
Unknown
Study type
Interventional
Source
ChiCTR
Registry ID
ChiCTR2500102683
Enrollment
Unknown
Registered
2025-05-19
Start date
2025-06-18
Completion date
Unknown
Last updated
2025-05-26

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

Conditions

Germline Predisposition-Associated Bone Marrow Failure

Interventions

Experimental Group:ATG+ Thiotepa + Fudarabine + Busulfan + Cyclophosphamide

Sponsors

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Lead Sponsor

Eligibility

Sex/Gender
All
Age
No minimum to 18 Years

Inclusion criteria

Inclusion criteria: 1.Both men and women aged =60 points. 5. Informed consent provided by the patient's legal guardian.

Exclusion criteria

Exclusion criteria: If any of the following conditions are met, the patient will be excluded; 1.Patients with Fanconi anemia or Dyskeratosis congenita; 2.Patients who have progressed to high - risk myelodysplastic syndromes (MDS), acute myeloid leukemia (AML), chronic myelomonocytic leukemia (CMML), or other malignant hematological diseases; 3.Patients with severe organ dysfunction or disease; 4.Patients who are HIV - positive, have active hepatitis B/C, or active tuberculosis, or have other severe infections that may be life - threatening; 5.Any other conditions deemed by the investigator as unsuitable for participation in this study.

Design outcomes

Primary

MeasureTime frame
Response Rate;Overall Survival;

Secondary

MeasureTime frame
Progression Rate;Relapse free survival;Neutrophil Engraftment?Platelet Engraftment;Relapse;Omplete ResponsePartial Response;

Countries

China

Contacts

Public ContactXiaofan Zhu

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College.

xfzhu@ihcams.ac.cn+86 22 23909001

Outcome results

None listed

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