Thalassemia MajorPhase 4NCT04009525
Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
A Phase 4 study testing Busulfan, Cyclophosphamide and Fludarabine in people with Thalassemia Major, run by First Affiliated Hospital of Guangxi Medical University at 1 site in China.
Trial summary
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major. Read more about this study
- Who is it for?
- Ages 2 to 20 years
- Conditions the study is for
- Thalassemia Major
- What is being tested?
- Busulfan, Cyclophosphamide and Fludarabine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 823 participants
- Where
- 1 siteChina
- Study dates
- 5 Jul 2019to 31 Oct 2023
- Phase
- Phase 4
- 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 2 to 20 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with thalassemia major.
- Indication of hematopoietic stem cell transplantation.
- A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.
Who cannot join
- Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
- Uncontrolled bacterial, viral or fungal infections;
- Any other restriction for transplantation.
Trial locations and contacts
1 site in 1 country.
China1 site
First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 510515
About this study
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Read the detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
- Conditions
- Thalassemia Major
- Being tested
- Busulfan, Cyclophosphamide, Fludarabine, Thymoglobulin, cyclosporine A
- Keywords
- thalassemia major, hematopoietic stem cell transplantation
- Medical subject headings
- beta-Thalassemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Also called Bu
Busulfan（4 mg/kg/day,4 days）
CyclophosphamideDrug
Also called Cy
Cyclophosphamide（50 mg/kg/day,4 days）
FludarabineDrug
Also called Flu
Fludarabine（50 mg/m2/day,3 days）
ThymoglobulinDrug
Also called ATG
Thymoglobulin（2.5 mg/kg/day,4 days）
cyclosporine ADrug
Also called CsA
cyclosporine A
Also called MMF
Mycophenolate mofetil（0.25g/day）
TacrolimusDrug
Also called FK506
Tacrolimus
MethotrexateDrug
Also called MTX
Methotrexate
| Study group | Role | What they receive |
|---|---|---|
| MSD-HSCT | Experimental | matched sibling donors hematopoietic stem cell transplantation Receives: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Thymoglobulin; Drug: cyclosporine A; Drug: Mycophenolate mofetil; Drug: Methotrexate |
| URD-HSCT | Experimental | unrelated donor hematopoietic stem cell transplantation Receives: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Thymoglobulin; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate |
| haplo-HSCT | Experimental | haplo-identical hematopoietic stem cell transplantation Receives: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Thymoglobulin; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
2-years overall survival
Measured: 2 years
Thalassemia-free survival
2-years thalassemia-free survival
Measured: 2 years
Secondary outcomes (5)
Engraftment
Myeloid engraftment at day +30
Measured: 30 days
Transplant Related Mortality
Transplant-related mortality by 2 year
Measured: 2 year
Cumulative Incidence of acute Graft Versus Host Disease
Acute graft versus host disease at day +100
Measured: 100 days
Cumulative Incidence of chronic Graft Versus Host Disease
Chronic graft versus host disease by 2 years
Measured: 2 years
Cumulative Incidence of Infectious Complications
Cumulative incidence of bacterial, fungal and viral infections by 2 years
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 posted5 Jul 2019
- Study start5 Jul 2019
- Primary completion31 Jul 2023
- Study completion31 Oct 2023
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 Thalassemia MajorStudies of the same condition, open and under way.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 823 participants (actual)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University People's Hospital, Ruijin Hospital, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, LiuZhou People's Hospital, The First People's Hospital of Yunnan, Liuzhou Workers' Hospital, Xinqiao Hospital of Chongqing, Affiliated Hospital of Youjiang University of Ethnic Medicine, Hainan People's Hospital, Maoming People's Hospital, Red Cross Hospital of Yulin City, Guilin Medical College, Wuzhou People's Hospital, Guangxi Autonomous Region People's Hospital, 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
- Responsible party
- Principal investigator, Yongrong Lai, First Affiliated Hospital of Guangxi Medical University
- Investigators
- Yongrong Lai, MD, Principal investigator, First Affiliated Hospital of Guangxi Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04009525
- Sponsor's protocol ID
- GX-HSCT-MT 2019
- Status last verified
- Aug 2024
- First submitted
- 3 Jul 2019
- Last updated
- 9 Aug 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Liu R, Xiao H, Qin C, Hu J, Luo J, Chen H, Zhang X, Yin X, Nai G, Wang G, Li Z, Tang W, Zhang H, Wang X, Lan M, Liao C, Yang T, Wang S, Huang J, He Y, Wang L, Zhang Z, Liu L, Wei Z, Shi L, Wu M, Zhou Q, Andersson BS, Huang X, Lai Y. A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia. Nat Commun. 2026 Feb 23;17(1):3083. doi: 10.1038/s41467-026-69756-8.
PubMed 41730859
Study at a glance
From the registry record, last updated by the sponsor on 9 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major: A Multicenter, Prospective Clinical Study
- ClinicalTrials.gov ID
- NCT04009525
- Acronym / sponsor ID
- GX-HSCT-MT 2019
- Conditions
- Thalassemia Major
- Keywords
- thalassemia major, hematopoietic stem cell transplantation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 823 participants (actual)
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical UniversityUniversity, hospital or other
- Collaborators
- Peking University People's Hospital, Ruijin Hospital, The 923rd Hospital of Joint Logistics Support Force of People's Liberation Army, LiuZhou People's Hospital, The First People's Hospital of Yunnan, Liuzhou Workers' Hospital, Xinqiao Hospital of Chongqing, Affiliated Hospital of Youjiang University of Ethnic Medicine, Hainan People's Hospital, Maoming People's Hospital, Red Cross Hospital of Yulin City, Guilin Medical College, Wuzhou People's Hospital, Guangxi Autonomous Region People's Hospital, 920th Hospital of Joint Logistics Support Force of People's Liberation Army of China
- Study start
- 5 Jul 2019 (actual)
- Primary completion
- 31 Jul 2023 (actual)
- Study completion
- 31 Oct 2023 (actual)
- First posted
- 5 Jul 2019 (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 only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(allo-HSCT). We conduct a prospective multicenter study to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Detailed description
Allogeneic hematopoietic stem cell transplantation (allo-HSCT) is one of the established curative option for thalassemia major (TM). Previous study has predicted that more than 90% of TM patients can survive after allo-HSCT with a thalassemia-free survival (TFS) in around 80% of them.The purpose of this study is to evaluate the efficacy of allo-HSCT in the treatment of thalassemia major.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| MSD-HSCT | Experimental | Drug: Busulfan, Drug: Cyclophosphamide, Drug: Fludarabine, Drug: Thymoglobulin, Drug: cyclosporine A, Drug: Mycophenolate mofetil, Drug: Methotrexatematched sibling donors hematopoietic stem cell transplantation |
| URD-HSCT | Experimental | Drug: Busulfan, Drug: Cyclophosphamide, Drug: Fludarabine, Drug: Thymoglobulin, Drug: Mycophenolate mofetil, Drug: Tacrolimus, Drug: Methotrexateunrelated donor hematopoietic stem cell transplantation |
| haplo-HSCT | Experimental | Drug: Busulfan, Drug: Cyclophosphamide, Drug: Fludarabine, Drug: Thymoglobulin, Drug: Mycophenolate mofetil, Drug: Tacrolimus, Drug: Methotrexatehaplo-identical hematopoietic stem cell transplantation |
Who may qualify
- Ages
- 2 to 20 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (3 listed)
- Diagnosed with thalassemia major.
- Indication of hematopoietic stem cell transplantation.
- A cardiac ejection fraction of >50%; normal pulmonary function tests and pulmonary examination results; and normal kidney function.
Key exclusion criteria (3 listed)
- Aspartate aminotransferase levels > 4-fold the upper limit of the normal range for our institution's lab criteria;
- Uncontrolled bacterial, viral or fungal infections;
- Any other restriction for transplantation.
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 |
|---|---|---|
| BusulfanAlso: BuBusulfan（4 mg/kg/day,4 days） | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
| CyclophosphamideAlso: CyCyclophosphamide（50 mg/kg/day,4 days） | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
| FludarabineAlso: FluFludarabine（50 mg/m2/day,3 days） | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
| ThymoglobulinAlso: ATGThymoglobulin（2.5 mg/kg/day,4 days） | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
| cyclosporine AAlso: CsAcyclosporine A | Drug | MSD-HSCT |
| Mycophenolate mofetilAlso: MMFMycophenolate mofetil（0.25g/day） | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
| TacrolimusAlso: FK506Tacrolimus | Drug | URD-HSCT, haplo-HSCT |
| MethotrexateAlso: MTXMethotrexate | Drug | MSD-HSCT, URD-HSCT, haplo-HSCT |
Primary outcome
Overall survival
Time frame: 2 years
2-years overall survival
Thalassemia-free survival
Time frame: 2 years
2-years thalassemia-free survival
5 secondary outcomes
Engraftment
Time frame: 30 days
Transplant Related Mortality
Time frame: 2 year
Cumulative Incidence of acute Graft Versus Host Disease
Time frame: 100 days
Cumulative Incidence of chronic Graft Versus Host Disease
Time frame: 2 years
Cumulative Incidence of Infectious Complications
Time frame: 2 years
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 9 Aug 2024.
- Derived from this study: PubMed 41730859Liu R, Xiao H, Qin C, Hu J, Luo J, Chen H, Zhang X, Yin X, Nai G, Wang G, Li Z, Tang W, Zhang H, Wang X, Lan M, Liao C, Yang T, Wang S, Huang J, He Y, Wang L, Zhang Z, Liu L, Wei Z, Shi L, Wu M, Zhou Q, Andersson BS, Huang X, Lai Y. A multi-center clinical trial of allogeneic hematopoietic stem cell transplantation in transfusion-dependent thalassemia. Nat Commun. 2026 Feb 23;17(1):3083. doi: 10.1038/s41467-026-69756-8.
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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.
Yongrong Lai, MD principal investigator
First Affiliated Hospital of Guangxi 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 4 study testing Busulfan, Cyclophosphamide and Fludarabine in people with Thalassemia Major, run by First Affiliated Hospital of Guangxi 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 3 inclusion and 3 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 9 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Overall survival (time frame: 2 years). The study lists 2 primary outcomes in all. 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 NCT04009525, 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 9 Aug 2024.