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Active, Not Recruiting

NCT Number: NCT04547049

A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies

An open, multi-center, randomized trial comparing haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies

Active, Not Recruiting

This study is active but is not currently recruiting participants.

Key information

Conditions

Leukemia Hematologic Diseases Hemic and Lymphatic Diseases MDS Neoplasms Neoplasms by Histologic Type

Age range

13 year–78 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Xiangya Hospital Central South University, Changsha, China

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About this study

This is an open, multi-center, randomized trial comparing the clinical outcomes of haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies. This study is indicated for patients with hematological malignancies including acute leukemias and MDS who are eligible to haploidentical HSCTs. 2 groups of patients will be enrolled with 116 in each group. The clinical criteria including survival, relapse, transplantation-related mortality will be monitored.

Who can participate

Healthy volunteers accepted: No

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

Patient Inclusion Criteria:

  • Patient age 13-78 years
  • Absence of a suitable HLA identical related or unrelated hematopoietic stem cell donor
  • Absence of a suitable partially HLA-mismatched (haploidentical), first-degree related donor aged between 18 and 50
  • Presence of both HLA haploidentical young non-first-degree (age ≤ 40) and older first-degree (age >50) donors

Eligible diagnoses:

AML（excluding APL） with at least one of the following:

  • median- or high- risk according to the WHO prognostic stratification system
  • failure to achieve CR after 2 cycles of induction chemotherapy
  • AML arising from MDS or a myeloproliferative disorder, or secondary AML
  • patients in CR2 or beyond

Mixed-phenotype acute leukemia (MPAL) in morphological remission Acute lymphoblastic leukemia (T or B) in morphological remission

MDS with at least one of the following:

  • IPSS score of INT-2 or greater
  • IPSS score of INT-1 with life-threatening cytopenias, including those generally requiring greater than weekly transfusions
  • A first allo-HCT
  • Adequate end-organ function
  • ECOG performance status < 2
  • No other contraindications for HSCT
  • Signature of the informed consent

Patient Exclusion Criteria

  • Availability of suitable HLA identical related or unrelated hematopoietic stem cell donors
  • Availability of suitable partially HLA-mismatched (haploidentical), first-degree related donor aged between 18 and 50
  • Not the first allo-HCT
  • Presence of uncontrolled bacterial, viral, or fungal infection
  • Patients with severe heart, lung, liver and kidney insufficiency
  • HIV-positive patients
  • Women of childbearing potential who are pregnant (β-HCG+) or breast feeding
  • Patients with a psychiatric history
  • ECOG performance status ≥ 2
  • Patients with malignancies other than the primary disease
  • Refusal to sign the informed consent

Donor Inclusion Criteria:

  • The donor and recipient must be HLA haploidentical
  • Meets institutional selection criteria and medically fit to donate
  • Lack of recipient anti-donor HLA antibody

Donor Exclusion Criteria:

  • The donor and recipient are HLA identical
  • Has not donated blood products to recipient

Treatment and study plan

Cytarabine

Drug

4 mg/m2/day administered IV day -10 through -9.

busulfan

Drug

3.2 mg/kg/day administered IV day -8 through -6.

Cyclophosphamide

Drug

1.8 g/m2/day administered IV day -5 through -4.

Me-CCNU

Drug

250mg/m2 once administered orally on day -3.

rabbit antithymocyte globulin

Drug

Between 6mg/kg total dose administered IV day -5 through -2 AND 7.5mg/kg total dose administered IV day -5 through -2.

allogeneic HSCT

Procedure

Day 0

cyclosporin A

Drug

2.5 mg/kg/day administered intravenously from day -7, target: 200-300ng/mL. Usually tapered during the second month, and ended in complete withdrawal during the ninth month after transplantation.

Mycophenolate mofetil

Drug

500 mg/day administered intravenously from day -9, ended in complete withdrawal on day +100.

MTX

Drug

15 mg/m2 administered intravenously on day +1, 10mg/m2 on day +3, +6, and +9.

Primary outcomes

  1. Overall survival (OS)

    Time frame: 2 years

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

Secondary outcomes

  1. Progression-free survival (PFS)

    Time frame: 2 years

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

  2. Cumulative incidence of transplant-related nonrelapse mortality (NRM)

    Time frame: 2 years

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

  3. Cumulative incidence of disease relapse or progression

    Time frame: 2 years

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

  4. GVHD-free, relapse-free survival (GRFS)

    Time frame: 2 years

    All patients will be tracked from Day 0 to date of first objective disease progression, death from any cause, or last patient evaluation. Patients who have not progressed or died will be censored at the last date they were assessed and deemed free of relapse or progression.

    GRFS is defined as survival with no evidence of relapse/progression, grade III to IV aGVHD, and systemic therapy-requiring cGVHD.

  5. Cumulative incidence of total acute and grade II-IV acute GVHD

    Time frame: 180 days

    Date of symptom onset, maximum clinical grade, and dates and types of treatment will be recorded. Dates of symptom onset of grade II or higher acute GVHD and grade III-IV acute GVHD will be recorded.

  6. Cumulative incidence of total and and moderate-severe chronic GVHD

    Time frame: 2 years

    Date of symptom onset, maximum clinical grade, and dates and types of treatment will be recorded. Dates of symptom onset of chronic GVHD and severe chronic GVHD will be recorded.

Sponsors and collaborators

Lead sponsor

First Affiliated Hospital of Zhejiang University

Other

Registry information

Official study title

An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies

Important dates

Study start
2020
Primary completion
2026
Study completion
2026
First posted
Sep 14, 2020
Registry last updated
Apr 9, 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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