Hematological MalignanciesPhase 3NCT05290545
Haplo-PBSC+Cord vs Haplo-PBSC+BM for Hematological Malignancies Undergoing Allo-HSCT
A Phase 3 study testing PBSCs, Cord and BMSCs in people with Hematological Malignancies and Hematopoietic Stem Cell Transplantation, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
The objective of this study was to explore whether the combination with umbilical cord blood (UCB) is associated with superior disease-free survival (DFS) in the setting of haploidentical donors (HID) transplantation. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Hematological Malignancies, Hematopoietic Stem Cell Transplantation
- What is being tested?
- PBSCs, Cord and BMSCs
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 314 participants
- Where
- 1 siteChina
- Study dates
- 15 Feb 2022to 30 Mar 2024
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patients with hematologic malignancies undergoing first HID allo-HSCT
- Age 18 to 65 years old with ECOG performance status 0-2
- Received myeloablative conditioning regimens
- Sign informed consent form, have the ability to comply with study and follow-up procedures
Who cannot join
- Received PBSCs as only grafts
- Acute leukemia transformed from a myeloproliferative tumor
- Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
- Respiratory failure ( PaO2 ≤60mmHg)
- Hepatic abnormalities (total bilirubin ≥3 mg/dL, aminotransferase >2 times the upper limit of normal)
- Renal dysfunction (creatinine clearance rate < 30 mL/min)
- ECOG performance status 3, 4 or 5
- With any conditions not suitable for the trial (investigators' decision)
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Hematology,Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
About this study
Official title: A Comparative Study of Haploidentical Transplantation Supported by Third-party Cord Blood and Haploidentical Transplantation in Hematological Malignancies
The objective of this study was to explore whether the combination with umbilical cord blood (UCB) is associated with superior disease-free survival (DFS) in the setting of haploidentical donors (HID) transplantation.
Read the detailed description
The main causes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) failure are primary disease relapse and transplant-related complications, especially relapse. In recent years, with the development of transplantation technology, alternative donors such as HID and UCB have been widely used. But, these alternative donors are associated with high incidences of transplant-related complications and mortalities when compared with human leukocyte antigen (HLA)-matched donors. Some studies suggeted that mixed grafts might overcome the disadvantages of a single alternative graft. UCB transplant (UCBT) supported by third-party HID or HID transplants supported by third-party UCB has been reported to have rapid engraftment and low incidences of graft-versus-host-disease (GVHD), making survival improvement. However, most of these results came from single-arm studies. The comparative studies between haplo-PBSC+Cord and haplo-PBSC+BM are scarce in the setting of HID transplantation. In a retrospective study, the investigators found haplo-PBSC+Cord transplantation has superior DFS than haplo-PBSC+BM in hematological malignancies. To further confirmed this conclusion, the investigators plan to conduct a prospective, multicenter, phase 3 randomized controlled trial.
- Being tested
- PBSCs, Cord, BMSCs
- Keywords
- Hematological malignancies, Haploidentical transplantation, Umbilical cord blood, Disease-free survival
- Medical subject headings
- Hematologic Neoplasms
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PBSCsOther
PBSCs harvest is performed from day 5 of G-CSF to obtain at least 7.0×10^8 total nucleated cells/kg recipient ideal body weight.
CordOther
The criteria for cord selection included the following: (1) ≥3 of 6 HLA loci , (2) blood type matches, (3) contained a minimum cell count of 0.3×10^8 nucleated cells/kg and 0.15×10^6 CD34-positive cells/kg before freezing. The third party UCB will be infused the day after infusion of PBSCs.
BMSCsOther
BMSCs of donor will be collected and infused at least 0.5×10^8 total nucleated cells/kg recipient ideal body weight the day after PBSCs infusion.
| Study group | Role | What they receive |
|---|---|---|
| Haplo-PBSC+Cord group | Experimental | The third party UCB will be infused the day after infusion of PBSCs from HID. Receives: Other: PBSCs; Other: Cord |
| Haplo-PBSC+BM group | Active comparator | The BMSCs from the same HID will be infused the day after infusion of PBSCs. Receives: Other: PBSCs; Other: BMSCs |
What is being measured
The primary outcome is the main question the study is designed to answer.
Disease-free survival (DFS)
Measured: 1 year
Secondary outcomes (5)
Relapse rate
Measured: 1 year
Overall survival (OS)
Measured: 1 year
The cumulative incidence of hematopoietic engraftment.
Hematopoietic engraftment includes the time of neutrophil and platelet engraftment. Neutrophil engraftment was defined as the first of two consecutive days with an absolute neutrophil count in the peripheral blood exceeding 0.5 × 10^9/L and the platelet engraftment was defined as the first of 3 days with an absolute platelet count exceeding 20 × 10^9 /L without transfusion support.
Measured: 30 days post-transplantation
The cumulative incidence of acute graft-versus-host-disease (GVHD)
Acutue GVHD was defined according to the 1994 consensus conference on acute GVHD grading and graded from I to IV.
Measured: 100 days post-transplantation
The cumulative incidence of chronic GVHD
Chronic GVHD was graded as mild, moderate and severe according to the national institutes of health consensus development project on criteria for clinical trials in chronic GVHD: the 2014 diagnosis and staging working group report.
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Mar 2022
- Study start15 Feb 2022
- Primary completion2 Jan 2023
- Study completion30 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Hematological MalignanciesStudies of the same condition, open and under way.
- More trials run by Nanfang Hospital, Southern Medical 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
- 314 participants (actual)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- First Affiliated Hospital of Guangxi Medical University, Hunan Provincial People's Hospital, The Seventh Affiliated Hospital of Sun Yat-sen University, First People's Hospital of Chenzhou, Dongguan People's Hospital, The First People's Hospital of Guangzhou
- Responsible party
- Principal investigator, Liu Qifa, Nanfang Hospital, Southern Medical University
- Investigators
- Qifa Liu, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05290545
- Sponsor's protocol ID
- NFEC-2021-126
- Status last verified
- Mar 2022
- First submitted
- 3 Mar 2022
- Last updated
- 13 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Yu S, Huang F, Xu N, Zhang Z, Liu C, Xu X, Fan Z, Zeng X, Liu Q, Qiu G, Xi X, Lin R, Liang X, Jiang Y, Dai M, Jin H, Li X, Wang S, Wu M, Sun J, Xuan L, Liu Q. Haploidentical peripheral blood stem cells combined with bone marrow or unrelated cord blood as grafts for haematological malignancies: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2025 Mar;12(3):e190-e200. doi: 10.1016/S2352-3026(24)00372-7. Epub 2025 Feb 4.
PubMed 39919775
Study at a glance
From the registry record, last updated by the sponsor on 13 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Comparative Study of Haploidentical Transplantation Supported by Third-party Cord Blood and Haploidentical Transplantation in Hematological Malignancies
- ClinicalTrials.gov ID
- NCT05290545
- Acronym / sponsor ID
- NFEC-2021-126
- Keywords
- Hematological malignancies, Haploidentical transplantation, Umbilical cord blood, Disease-free survival
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 314 participants (actual)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- First Affiliated Hospital of Guangxi Medical University, Hunan Provincial People's Hospital, The Seventh Affiliated Hospital of Sun Yat-sen University, First People's Hospital of Chenzhou, Dongguan People's Hospital, The First People's Hospital of Guangzhou
- Study start
- 15 Feb 2022 (actual)
- Primary completion
- 2 Jan 2023 (actual)
- Study completion
- 30 Mar 2024 (actual)
- First posted
- 22 Mar 2022 (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.
The objective of this study was to explore whether the combination with umbilical cord blood (UCB) is associated with superior disease-free survival (DFS) in the setting of haploidentical donors (HID) transplantation.
Detailed description
The main causes of allogeneic hematopoietic stem cell transplantation (allo-HSCT) failure are primary disease relapse and transplant-related complications, especially relapse. In recent years, with the development of transplantation technology, alternative donors such as HID and UCB have been widely used. But, these alternative donors are associated with high incidences of transplant-related complications and mortalities when compared with human leukocyte antigen (HLA)-matched donors. Some studies suggeted that mixed grafts might overcome the disadvantages of a single alternative graft. UCB transplant (UCBT) supported by third-party HID or HID transplants supported by third-party UCB has been reported to have rapid engraftment and low incidences of graft-versus-host-disease (GVHD), making survival improvement. However, most of these results came from single-arm studies. The comparative studies between haplo-PBSC+Cord and haplo-PBSC+BM are scarce in the setting of HID transplantation. In a retrospective study, the investigators found haplo-PBSC+Cord transplantation has superior DFS than haplo-PBSC+BM in hematological malignancies. To further confirmed this conclusion, the investigators plan to conduct a prospective, multicenter, phase 3 randomized controlled trial.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Haplo-PBSC+Cord group | Experimental | Other: PBSCs, Other: CordThe third party UCB will be infused the day after infusion of PBSCs from HID. |
| Haplo-PBSC+BM group | Active comparator | Other: PBSCs, Other: BMSCsThe BMSCs from the same HID will be infused the day after infusion of PBSCs. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- Patients with hematologic malignancies undergoing first HID allo-HSCT
- Age 18 to 65 years old with ECOG performance status 0-2
- Received myeloablative conditioning regimens
- Sign informed consent form, have the ability to comply with study and follow-up procedures
Key exclusion criteria (8 listed)
- Received PBSCs as only grafts
- Acute leukemia transformed from a myeloproliferative tumor
- Cardiac dysfunction (particularly congestive heart failure, unstable coronary artery disease and serious cardiac ventricular arrhythmias requiring antiarrhythmic therapy)
- Respiratory failure ( PaO2 ≤60mmHg)
- Hepatic abnormalities (total bilirubin ≥3 mg/dL, aminotransferase >2 times the upper limit of normal)
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 |
|---|---|---|
| PBSCsPBSCs harvest is performed from day 5 of G-CSF to obtain at least 7.0×10^8 total nucleated cells/kg recipient ideal body weight. | Other | Haplo-PBSC+BM group, Haplo-PBSC+Cord group |
| CordThe criteria for cord selection included the following: (1) ≥3 of 6 HLA loci , (2) blood type matches, (3) contained a minimum cell count of 0.3×10^8 nucleated cells/kg and 0.15×10^6 CD34-positive cells/kg before freezing. The third party UCB will be infused the day after infusion of PBSCs. | Other | Haplo-PBSC+Cord group |
| BMSCsBMSCs of donor will be collected and infused at least 0.5×10^8 total nucleated cells/kg recipient ideal body weight the day after PBSCs infusion. | Other | Haplo-PBSC+BM group |
Primary outcome
Disease-free survival (DFS)
Time frame: 1 year
5 secondary outcomes
Relapse rate
Time frame: 1 year
Overall survival (OS)
Time frame: 1 year
The cumulative incidence of hematopoietic engraftment.
Time frame: 30 days post-transplantation
The cumulative incidence of acute graft-versus-host-disease (GVHD)
Time frame: 100 days post-transplantation
The cumulative incidence of chronic GVHD
Time frame: 1 year
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Aug 2024.
- Derived from this study: PubMed 39919775Yu S, Huang F, Xu N, Zhang Z, Liu C, Xu X, Fan Z, Zeng X, Liu Q, Qiu G, Xi X, Lin R, Liang X, Jiang Y, Dai M, Jin H, Li X, Wang S, Wu M, Sun J, Xuan L, Liu Q. Haploidentical peripheral blood stem cells combined with bone marrow or unrelated cord blood as grafts for haematological malignancies: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2025 Mar;12(3):e190-e200. doi: 10.1016/S2352-3026(24)00372-7. Epub 2025 Feb 4.
- Individual participant dataSharing planned: No
Related trials for Hematological Malignancies
Not yet recruitingPhase 4
MACE and BTK Inhibitors
Hematologic Cancer, MACE
RecruitingEarly Phase 1
HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT
Hematologic Malignancy
Recruiting
An Exploratory Study to Evaluate Immune Determinants of the Response to Adoptive Cell Therapy (ACT) in Solid and Hematologic Tumors
Solid Tumor, Adult, Solid Tumor, Childhood
RecruitingObservational study
Natural History Study to Determine Drug Metabolism Phenotype and Appropriate Germline Source DNA in Patients Undergoing Allogeneic Hematopoietic Stem Cell Transplant
Leukemia, Lymphoma
Active, not recruitingPhase 2
Testing AZD5363 as a Potential Targeted Treatment in Cancers With AKT Genetic Changes (MATCH-Subprotocol Y)
Advanced Lymphoma, Advanced Malignant Solid Neoplasm
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.
Qifa Liu principal investigator
Nanfang Hospital, Southern Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing PBSCs, Cord and BMSCs in people with Hematological Malignancies and Hematopoietic Stem Cell Transplantation, run by Nanfang Hospital, Southern Medical University at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 8 exclusion criteria. 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: finished, as of the sponsor's last update on 13 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Disease-free survival (DFS) (time frame: 1 year). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT05290545, which also lists 1 linked publication.
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 13 Aug 2024.