LeukemiaPhase 3NCT04547049
A Study Comparing Haploidentical Hematopoietic Stem Cell Transplantations (HSCTs) From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
A Phase 3 study testing Cytarabine, Busulfan and Cyclophosphamide in people with Leukemia and MDS, run by First Affiliated Hospital of Zhejiang University at 8 sites in China.
Trial summary
An open, multi-center, randomized trial comparing haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies Read more about this study
- Who is it for?
- Ages 13 to 78 years
- What is being tested?
- Cytarabine, Busulfan and Cyclophosphamide
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 232 participants
- Where
- 8 sitesChina
- Study dates
- 1 Sep 2020to 1 Apr 2026
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 78 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Trial locations and contacts
8 sites in 1 country.
8 sites in 1 country
China8 sites
Xiangya Hospital Central South University
Changsha
Xinqiao Hospital, State Key Laboratory of Trauma and Chemical Poisoning, Army Medical University
Chongqing
The First Affiliated Hospital, College of Medicine, Zhejiang University
Hangzhou
Zhejiang Provincial People's Hospital
Hangzhou
The First Affiliated Hospital of Nanjing Medical University
Nanjing
The Affiliated People's Hospital of Ningbo University
Ningbo
The First Affiliated Hospital of Ningbo University
Ningbo
The First Affiliated Hospital of Soochow University
Suzhou
About this study
Official title: An Open, Multi-center, Randomized Trial Comparing Haploidentical 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
Read the detailed description
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.
- Being tested
- Cytarabine, Busulfan, Cyclophosphamide, Me-CCNU, Rabbit antithymocyte globulin
- Keywords
- hematopoietic stem cell transplantation
- Medical subject headings
- Leukemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CytarabineDrug
4 mg/m2/day administered IV day -10 through -9.
BusulfanDrug
3.2 mg/kg/day administered IV day -8 through -6.
CyclophosphamideDrug
1.8 g/m2/day administered IV day -5 through -4.
Me-CCNUDrug
250mg/m2 once administered orally on day -3.
Rabbit antithymocyte globulinDrug
Between 6mg/kg total dose administered IV day -5 through -2 AND 7.5mg/kg total dose administered IV day -5 through -2.
Allogeneic HSCTProcedure or surgery
Day 0
Cyclosporin ADrug
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.
500 mg/day administered intravenously from day -9, ended in complete withdrawal on day +100.
MTXDrug
15 mg/m2 administered intravenously on day +1, 10mg/m2 on day +3, +6, and +9.
| Study group | Role | What they receive |
|---|---|---|
| Non-first-degree donor | Active comparator | Each patient receive graft from a non-first degree donor aged ≤40
Conditoning regimens are decided by each center accoding to disease risk, patient age \& status, and cormobidity index.The centers are required to use the same principle when treating NFD and FD patients. The used protocol in the current study included:
For MAC: use BU-CY-based (described in detail)
For RIC: use BUFlu:
Drug: Fludarabine 30 mg/m²/kg, administered IV d-10 through d-5. Drug: Busulfan, 3.2 mg/kg/day administered IV day -6 (BU2) or -7 through -5 (BU3).
Other conditioning regimen may include:
TBF, FluMel, FBM, Cy-TBI, Flu-TBI. Receives: Drug: Cytarabine; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Me-CCNU; Drug: Rabbit antithymocyte globulin; Procedure: Allogeneic HSCT; Drug: Cyclosporin A; Drug: Mycophenolate Mofetil; Drug: MTX |
| First-degree donor | Active comparator | Each patients receive graft from a first-degree donor aged >50
Conditoning regimens are decided by each center accoding to disease risk, patient age \& status, and cormobidity index.The centers are required to use the same principle when treating NFD and FD patients. The used protocol in the current study included: For MAC: use BU-CY-based (described in detail) For RIC: use BUFlu: Drug: Fludarabine 30 mg/m²/kg, administered IV d-10 through d-5. Drug: Busulfan, 3.2 mg/kg/day administered IV day -6 (BU2) or -7 through -5 (BU3). Other conditioning regimen may include: TBF, FluMel, FBM, Cy-TBI, Flu-TBI. Receives: Drug: Cytarabine; Drug: Busulfan; Drug: Cyclophosphamide; Drug: Me-CCNU; Drug: Rabbit antithymocyte globulin; Procedure: Allogeneic HSCT; Drug: Cyclosporin A; Drug: Mycophenolate Mofetil; Drug: MTX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival (OS)
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.
Measured: 2 years
Secondary outcomes (6)
Progression-free survival (PFS)
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.
Measured: 2 years
Cumulative incidence of transplant-related nonrelapse mortality (NRM)
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.
Measured: 2 years
Cumulative incidence of disease relapse or progression
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.
Measured: 2 years
GVHD-free, relapse-free survival (GRFS)
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.
Measured: 2 years
Cumulative incidence of total acute and grade II-IV acute GVHD
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.
Measured: 180 days
Cumulative incidence of total and and moderate-severe chronic GVHD
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.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Sep 2020
- Study start1 Sep 2020
- Primary completion1 Apr 2026 (estimated)
- Study completion1 Apr 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for LeukemiaStudies of the same condition, open and under way.
- More trials run by First Affiliated Hospital of Zhejiang UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 232 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Zhejiang University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yi Luo, First Affiliated Hospital of Zhejiang University
- Investigators
- Yi Luo, MD, Principal investigator, First Affiliated Hospital of Zhejiang University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04547049
- Sponsor's protocol ID
- NFD-001
- Status last verified
- Sep 2025
- First submitted
- 7 Sep 2020
- Last updated
- 9 Apr 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Apr 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open, Multi-center, Randomized Trial Comparing Haploidentical HSCTs From Young Non-first-degree and Older First-degree Donors in Hematological Malignancies
- ClinicalTrials.gov ID
- NCT04547049
- Acronym / sponsor ID
- NFD-001
- Keywords
- hematopoietic stem cell transplantation
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 232 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Zhejiang UniversityUniversity, hospital or other
- Study start
- 1 Sep 2020 (actual)
- Primary completion
- 1 Apr 2026 (estimated)
- Study completion
- 1 Apr 2026 (estimated)
- First posted
- 14 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An open, multi-center, randomized trial comparing haploidentical HSCTs from young non-first-degree and older first-degree donors in hematological malignancies
Detailed description
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Non-first-degree donor | Active comparator | Drug: Cytarabine, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Me-CCNU, Drug: Rabbit antithymocyte globulin, Procedure: Allogeneic HSCT, Drug: Cyclosporin A, Drug: Mycophenolate Mofetil, Drug: MTXEach patient receive graft from a non-first degree donor aged ≤40 Conditoning regimens are decided by each center accoding to disease risk, patient age \& status, and cormobidity index.The centers are required to use the same principle when treating NFD and FD patients. The used protocol in the current study included: For MAC: use BU-CY-based (described in detail) For RIC: use BUFlu: Drug: Fludarabine 30 mg/m²/kg, administered IV d-10 through d-5. Drug: Busulfan, 3.2 mg/kg/day administered IV day -6 (BU2) or -7 through -5 (BU3). Other conditioning regimen may include: TBF, FluMel, FBM, Cy-TBI, Flu-TBI. |
| First-degree donor | Active comparator | Drug: Cytarabine, Drug: Busulfan, Drug: Cyclophosphamide, Drug: Me-CCNU, Drug: Rabbit antithymocyte globulin, Procedure: Allogeneic HSCT, Drug: Cyclosporin A, Drug: Mycophenolate Mofetil, Drug: MTXEach patients receive graft from a first-degree donor aged >50 Conditoning regimens are decided by each center accoding to disease risk, patient age \& status, and cormobidity index.The centers are required to use the same principle when treating NFD and FD patients. The used protocol in the current study included: For MAC: use BU-CY-based (described in detail) For RIC: use BUFlu: Drug: Fludarabine 30 mg/m²/kg, administered IV d-10 through d-5. Drug: Busulfan, 3.2 mg/kg/day administered IV day -6 (BU2) or -7 through -5 (BU3). Other conditioning regimen may include: TBF, FluMel, FBM, Cy-TBI, Flu-TBI. |
Who may qualify
- Ages
- 13 to 78 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Cytarabine4 mg/m2/day administered IV day -10 through -9. | Drug | First-degree donor, Non-first-degree donor |
| Busulfan3.2 mg/kg/day administered IV day -8 through -6. | Drug | First-degree donor, Non-first-degree donor |
| Cyclophosphamide1.8 g/m2/day administered IV day -5 through -4. | Drug | First-degree donor, Non-first-degree donor |
| Me-CCNU250mg/m2 once administered orally on day -3. | Drug | First-degree donor, Non-first-degree donor |
| Rabbit antithymocyte globulinBetween 6mg/kg total dose administered IV day -5 through -2 AND 7.5mg/kg total dose administered IV day -5 through -2. | Drug | First-degree donor, Non-first-degree donor |
| Allogeneic HSCTDay 0 | Procedure or surgery | First-degree donor, Non-first-degree donor |
| Cyclosporin A2.5 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. | Drug | First-degree donor, Non-first-degree donor |
| Mycophenolate Mofetil500 mg/day administered intravenously from day -9, ended in complete withdrawal on day +100. | Drug | First-degree donor, Non-first-degree donor |
| MTX15 mg/m2 administered intravenously on day +1, 10mg/m2 on day +3, +6, and +9. | Drug | First-degree donor, Non-first-degree donor |
Primary outcome
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.
6 secondary outcomes
Progression-free survival (PFS)
Time frame: 2 years
Cumulative incidence of transplant-related nonrelapse mortality (NRM)
Time frame: 2 years
Cumulative incidence of disease relapse or progression
Time frame: 2 years
GVHD-free, relapse-free survival (GRFS)
Time frame: 2 years
Cumulative incidence of total acute and grade II-IV acute GVHD
Time frame: 180 days
Cumulative incidence of total and and moderate-severe chronic GVHD
Time frame: 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Apr 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Yi Luo, MD principal investigator
First Affiliated Hospital of Zhejiang University
8 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Cytarabine, Busulfan and Cyclophosphamide in people with Leukemia and MDS, run by First Affiliated Hospital of Zhejiang University at 8 sites in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 9 Apr 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (OS) (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Apr 2026.