Thalassemia MajorPhase 4NCT03171831
Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
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 purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major. Read more about this study
- Who is it for?
- Ages 2 to 18 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
- 30 participants
- Where
- 1 siteChina
- Study dates
- 1 Apr 2017to 1 Apr 2022
- 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 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed with thalassemia major
- Lacking of a HLA-identical sibling donor or unrelated donor
- Indication of haploidentical hematopoietic stem cell transplantation
- No restrictions for transplantation
Who cannot join
- Any restriction for transplantation
- No indication of haploidentical hematopoietic stem cell transplantation
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
First Affiliated Hospital of Guangxi Medical University
Nanning, Guangxi, 510515
Yongrong Lai, MD, +86（0771）5356510, laiyongrong@hotmail.com
About this study
The purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
Read the detailed description
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.
- Conditions
- Thalassemia Major
- Being tested
- Busulfan, Cyclophosphamide, Fludarabine, Mycophenolate mofetil, Tacrolimus
- 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）
Also called MMF
Mycophenolate mofetil（0.25g/day）
TacrolimusDrug
Also called FK506
Tacrolimus（0.03mg/kg/day）
MethotrexateDrug
Also called MTX
Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）
ThymoglobulinDrug
Also called ATG
Thymoglobulin（2.5 mg/kg/day,4 days）
BasiliximabDrug
Also called Simulect
Basiliximab（10mg on day 0 and +4）
| Study group | Role | What they receive |
|---|---|---|
| Haploidentical HSCT | Experimental | Procedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother).
Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days）
GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4） Receives: Drug: Busulfan; Drug: Cyclophosphamide; Drug: Fludarabine; Drug: Mycophenolate mofetil; Drug: Tacrolimus; Drug: Methotrexate; Drug: Thymoglobulin; Drug: Basiliximab |
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
Secondary outcomes (6)
Disease Free Survival
2-years disease free survival
Measured: 2 years
Engraftment
Myeloid engraftment at day +30
Measured: 30 days
Transplant Related Mortality
Transplant-related mortality by 1 year
Measured: 1 year
Cumulative Incidence of acute Graft Versus Host Disease
Acute graft versus host disease at day +180
Measured: 180 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 posted31 May 2017
- Study start1 Apr 2017
- Primary completion31 Dec 2021 (estimated)
- Study completion1 Apr 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- 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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical University
- Sponsor type
- University, hospital or other
- 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
- NCT03171831
- Sponsor's protocol ID
- Guangxi-Haplo-HSCT-2016
- Status last verified
- May 2017
- First submitted
- 18 Apr 2017
- Last updated
- 31 May 2017
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Haploidentical Hematopoietic Stem Cell Transplantation for Patients With Thalassemia Major
- ClinicalTrials.gov ID
- NCT03171831
- Acronym / sponsor ID
- Guangxi-Haplo-HSCT-2016
- Conditions
- Thalassemia Major
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- First Affiliated Hospital of Guangxi Medical UniversityUniversity, hospital or other
- Study start
- 1 Apr 2017 (actual)
- Primary completion
- 31 Dec 2021 (estimated)
- Study completion
- 1 Apr 2022 (estimated)
- First posted
- 31 May 2017 (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 purpose of the study is to assess the safety and efficacy of haploidentical hematopoietic stem cell transplantation for patients with thalassemia major.
Detailed description
The only curative therapy for thalassemia major remains the replacement of the defective erythropoiesis by allogeneic hematopoietic stem cell transplantation(HSCT). However, this option is unavailable to many patients as a result of a lack of compatible donors.Haploidentical transplantation has been certified as a valuable alternative for hematological malignancy patients lacking a well-matched donor, with results comparable matched HLA-identical sibling donors or unrelated donors.The objective of this study is to test the feasibility of haploidentical HSCT for patients with thalassemia major.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Haploidentical HSCT | Experimental | Drug: Busulfan, Drug: Cyclophosphamide, Drug: Fludarabine, Drug: Mycophenolate mofetil, Drug: Tacrolimus, Drug: Methotrexate, Drug: Thymoglobulin, Drug: BasiliximabProcedure: Haploidentical hematopoietic stem cell transplantation from a related donor (partially matched sibling, father or mother). Conditioning: Busulfan （4 mg/kg/day,4 days） + Cyclophosphamide （50 mg/kg/day,4 days）+ Fludarabine （50 mg/m2/day,3 days） GVHD Prophylaxis：Mycophenolate mofetil（0.25g/day）+ Tacrolimus（0.03mg/kg/day）+ Methotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11）+ Thymoglobulin（2.5 mg/kg/day,4 days）+Basiliximab（10mg on day 0 and +4） |
Who may qualify
- Ages
- 2 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (4 listed)
- Diagnosed with thalassemia major
- Lacking of a HLA-identical sibling donor or unrelated donor
- Indication of haploidentical hematopoietic stem cell transplantation
- No restrictions for transplantation
Key exclusion criteria (2 listed)
- Any restriction for transplantation
- No indication of haploidentical hematopoietic stem cell 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 | Haploidentical HSCT |
| CyclophosphamideAlso: CyCyclophosphamide（50 mg/kg/day,4 days） | Drug | Haploidentical HSCT |
| FludarabineAlso: FluFludarabine（50 mg/m2/day,3 days） | Drug | Haploidentical HSCT |
| Mycophenolate mofetilAlso: MMFMycophenolate mofetil（0.25g/day） | Drug | Haploidentical HSCT |
| TacrolimusAlso: FK506Tacrolimus（0.03mg/kg/day） | Drug | Haploidentical HSCT |
| MethotrexateAlso: MTXMethotrexate（15mg/m2 on day +1,10mg/m2 on day +3,+6,+11） | Drug | Haploidentical HSCT |
| ThymoglobulinAlso: ATGThymoglobulin（2.5 mg/kg/day,4 days） | Drug | Haploidentical HSCT |
| BasiliximabAlso: SimulectBasiliximab（10mg on day 0 and +4） | Drug | Haploidentical HSCT |
Primary outcome
Overall Survival
Time frame: 2 years
2-years overall survival
6 secondary outcomes
Disease Free Survival
Time frame: 2 years
Engraftment
Time frame: 30 days
Transplant Related Mortality
Time frame: 1 year
Cumulative Incidence of acute Graft Versus Host Disease
Time frame: 180 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
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2017.
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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 4 inclusion and 2 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: status not confirmed, as of the sponsor's last update on 31 May 2017. 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yongrong Lai, MD (+86（0771）5356510, laiyongrong@hotmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Survival (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 31 May 2017.