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NCT Number: NCT07677306

Age-Stratified Conditioning Regimen Efficacy for MDS Haplo-HSCT

This study plan aims to enroll adult patients diagnosed with myelodysplastic syndrome (MDS) who are scheduled to receive T-cell-replete haploidentical hematopoietic stem cell transplantation. After obtaining written informed consent, participants will receive either reduced-toxicity Bu/Flu/Cy/ATG conditioning regimen (for patients aged ≥55 years) or standard myeloablative modified Bu/Cy+ATG conditioning regimen (for patients aged <55 years) followed by unified post-transplant immunosuppression and supportive care. The objective is to prospectively characterize the 1-year transplant-related mortality and comprehensively evaluate hematopoietic engraftment, graft-versus-host disease, infection, relapse, survival outcomes and conditioning-related organ toxicity among all enrolled patients undergoing haploidentical transplantation.

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Key information

Conditions

Myelodysplastic Syndromes Bone Marrow Diseases Hematologic Diseases Hemic and Lymphatic Diseases

Age range

18 year–65 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Peking University People's Hospital, Beijing Lu Daopei Hematology Hospital, Hebei Yanda Lu Daopei Hospital, Nanfang Hospital, Southern Medical University

Beijing, China

Location contact

Xiaojun Huang

CONTACT

[email protected]

8610-8832-6005

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients who had low- and intermediate-risk MDS without ISD nor URD receiving haploidentical hematopoietic stem cell transplantation

Exclusion criteria

  • Patients having ISD or URD; patients having high-risk MDS; patients with active infection; patients with poor compliance; patients with organ failure

Treatment and study plan

RTC conditioning regimen and Modified Bu/Cy conditioning regimen

Drug

RTC preconditioning regimen consisted of cytarabine (2g/m2/day, days -10 to -9), busulfan (3.2 mg/kg/day on days -8 to -6), cyclophosphamide (1.0 g/m2/day, days -5 to -4), fludarabine (30 mg/m2/day, days -6 to -2), semustine (250 mg/m2, day-3), and rabbit antithymocyte globulin (thymoglobulin, 2.5 mg/kg/d, days -5 to -2; Sanofi, France). Bu/Cy preconditioning regimen consisted of tandard institutional myeloablative busulfan + cyclophosphamide + rabbit ATG regimen per department routine dosing guidelines.

Primary outcomes

  1. transplant-related mortality (TRM)

    Time frame: Participants will be followed for an expected average of 1 years

    Death without disease progression or relapse

Secondary outcomes

  1. Hematopoietic engraftment

    Time frame: Day 30 and Day 90 post-transplantation

    Neutrophil engraftment is defined as the first day of sustained absolute neutrophil count (ANC) ≥0.5×10⁹/L for 3 consecutive days; platelet engraftment is defined as the first day of sustained platelet count (PLT) ≥20×10⁹/L without transfusion support for 7 consecutive days. Cumulative incidence of neutrophil engraftment at Day 30 and platelet engraftment at Day 90 will be calculated.

  2. Cumulative incidence of graft-versus-host disease (GVHD)

    Time frame: Day 100 and 3 years after transplantation

    Cumulative incidence of Grade II-IV and Grade III-IV acute GVHD at Day 100, as well as chronic GVHD and moderate-severe chronic GVHD within 3 years after transplantation, graded per Seattle criteria.

  3. Cumulative incidence of CMV and EBV reactivation

    Time frame: 1 year after transplantation

    Defined as any detectable CMV or EBV viral load elevation requiring clinical intervention within 1 year after transplantation.

  4. Cumulative incidence of hematologic relapse

    Time frame: 1 year after transplantation

    Defined as bone marrow blast recurrence or extramedullary disease relapse within 1 year after transplantation.

  5. Disease-free survival (DFS)

    Time frame: 1 year after transplantation

    Defined as the time from transplantation until hematologic relapse or death from any cause.

  6. Overall survival（OS）

    Time frame: 1 year after transplantation

    Defined as the time from treatment until death from any cause or the last follow-up.

  7. Conditioning-related organ toxicities

    Time frame: From conditioning start to Day 30 post-transplantation

    Incidence of grade 1-4 cardiac, renal, hepatic, gastrointestinal and oral mucosal toxicities from conditioning initiation to Day +30 post-transplantation, with separate analysis of Grade 3-4 severe organ injury, hepatic veno-occlusive disease (VOD), and conditioning-related death, graded per WHO organ toxicity scale.

Study contacts

Contact information is provided by the study sponsor or research team.

Yu Wang

CONTACT

[email protected]

8610-8832-6005

Sponsors and collaborators

Lead sponsor

Peking University People's Hospital

Other

Registry information

Official study title

Prospective Cohort Study of Age-Stratified Busulfan-Based Conditioning Regimens in Adult Patients With Myelodysplastic Syndrome Receiving Haploidentical Hematopoietic Stem Cell Transplantation

Important dates

Study start
2026
Primary completion
2027
Study completion
2028
First posted
Jun 30, 2026
Registry last updated
Jun 30, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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