Severe Aplastic AnemiaPhase 4NCT06069180
The Optimization of Conditioning Regimen for HLA Matched HSCT in SAA
A Phase 4 study testing Busulfan and Flu/Cy/ATG or Cy/ATG in people with Severe Aplastic Anemia, run by Peking University People's Hospital at 1 site in China.
Trial summary
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. Read more about this study
- Who is it for?
- Ages 1 year to 50 years
- Conditions the study is for
- Severe Aplastic Anemia
- What is being tested?
- Busulfan and Flu/Cy/ATG or Cy/ATG
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 160 participants
- Where
- 1 siteChina
- Study dates
- 15 Nov 2023to 31 Dec 2025
- 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 1 year to 50 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosed as SAA/vSAA
- Indication for hematopoietic stem cell transplantation
- Available HLA matched sibling or unrelated donor
- No active infection
- No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency)
- Signed informed consent
Show all 9Show fewer
- High risk factors of mixed chimerism, at least one of the following
- Age < 18 years old
- Ferritin level ≥2500ng/ml before transplantation
Who cannot join
- Age > 50 years old
- ECOG≥3
- Active infections that were difficult to control
- Severe liver and kidney dysfunction
- Mental illness
- Not signing the informed consent
- pregnant or lactating women
- Any condition considered by the investigators to be unsuitable for enrollment
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Deparment of Hematology, Peking University People's Hospital
Beijing, Beijing Municipality, 100044
Xiaojun Huang, doctor, 8601088326666, xjrm@medmail.com.cn
About this study
Official title: To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.
- Conditions
- Severe Aplastic Anemia
- Being tested
- Busulfan, Flu/Cy/ATG or Cy/ATG
- Keywords
- HLA matched donor transplantation
- Medical subject headings
- Anemia, Aplastic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BusulfanDrug
Conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.
Flu/Cy/ATG or Cy/ATGDrug
Conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity.
| Study group | Role | What they receive |
|---|---|---|
| Busulfan included group | Experimental | The conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. Receives: Drug: Busulfan |
| Control group | Other | The conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. Receives: Drug: Flu/Cy/ATG or Cy/ATG |
What is being measured
The primary outcome is the main question the study is designed to answer.
Failure free survival
Failure free survival was defined as survival with a response to therapy.
Measured: 1 year post HSCT
Secondary outcomes (7)
The incidence of mixed chimerism
The mixed chimerism was defined as the presence of 5%-95% donor haematopoietic cells.
Measured: 1 year post HSCT
Regimen related toxicity
The regimen related toxicity (RTT) was measured according to the Seattle Toxicity Criteria (Bearman et al, 1988).
Measured: 100 days post HSCT
Myeloid and platelet engraftment
Myeloid and platelet engraftment were defined as international criteria.
Measured: 100 days post HSCT
The incidence of graft versus host disease
The severity of acute and chronic GVHD was evaluated according to standard criteria.
Measured: 100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD
The incidence of CMV and EBV reactivation
The incidence of CMV and EBV reactivation was defined as CMV and EBV viremia.
Measured: 100 days post HSCT
The incidence of Transplantation related mortality
Transplantation related mortality was defined as death without disease progression.
Measured: 1 year post HSCT
The probability of Overall survival
Overall survival was defined as the time from transplantation to death from any cause or to the last follow-up
Measured: 1 year post HSCT
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 Oct 2023
- Study start15 Nov 2023
- Primary completion31 Dec 2024 (estimated)
- Study completion31 Dec 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Severe Aplastic AnemiaStudies of the same condition, open and under way.
- More trials run by Peking University People's HospitalThe 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 160 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking University People's Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Xiao-Jun Huang, Peking University People's Hospital
- Investigators
- Xiao-Jun Huang, Principal investigator, Peking University People's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06069180
- Sponsor's protocol ID
- 2023PHB232-001
- Status last verified
- Nov 2023
- First submitted
- 1 Oct 2023
- Last updated
- 18 Nov 2023
- Results posted
- No
- 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 18 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- To Evaluate Different Conditioning Regimens for HLA Matched Donor Transplantation in Severe Aplastic Anemia: a Prospective, Multicenter, Randomized Controlled Study
- ClinicalTrials.gov ID
- NCT06069180
- Acronym / sponsor ID
- 2023PHB232-001
- Conditions
- Severe Aplastic Anemia
- Keywords
- HLA matched donor transplantation
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 160 participants (estimated)
- Lead sponsor
- Peking University People's HospitalUniversity, hospital or other
- Study start
- 15 Nov 2023 (actual)
- Primary completion
- 31 Dec 2024 (estimated)
- Study completion
- 31 Dec 2025 (estimated)
- First posted
- 5 Oct 2023 (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.
Hematopoietic stem cell transplantation (HSCT) from a human leukocyte antigen (HLA) -matched donor is an effective option for severe aplastic anemia (SAA), but there is no standardized and recommended conditioning regimen. The occurrence of mixed chimerism after transplantation is associated with secondary graft failure and poor failure-free survival. Previous studies have shown that Fludarabine (Flu)/ Cyclophosphamide (Cy)/ antithymocyte globulin (antithymocyte globulin), ATG) and Cy/ATG conditioning regimens had higher rates of mixed chimerism and poorer failure-free survival. A small cohort study has suggested that adding busulfan to Flu/Cy/ATG or Cy/ATG can reduce the incidence of mixed chimerism and improve failure-free survival. This study was a prospective, multicenter, randomized controlled trial to compare the efficacy and safety of different conditioning regimens in the treatment of severe aplastic anemia (SAA) after hematopoietic stem cell transplantation (HSCT) from HLA-identical sibling or unrelated donor.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Busulfan included group | Experimental | Drug: BusulfanThe conditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. |
| Control group | Other | Drug: Flu/Cy/ATG or Cy/ATGThe conditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. |
Who may qualify
- Ages
- 1 year to 50 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (9 listed)
- Diagnosed as SAA/vSAA
- Indication for hematopoietic stem cell transplantation
- Available HLA matched sibling or unrelated donor
- No active infection
- No serious organ damage: liver and kidney function (ALT and AST < 2.5 times normal value, normal renal function, no cardiac insufficiency)
Key exclusion criteria (8 listed)
- Age > 50 years old
- ECOG≥3
- Active infections that were difficult to control
- Severe liver and kidney dysfunction
- Mental illness
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 |
|---|---|---|
| BusulfanConditioning regimens were Bu/Flu/Cy/ATG or Bu/Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. | Drug | Busulfan included group |
| Flu/Cy/ATG or Cy/ATGConditioning regimens were Flu/Cy/ATG or Cy/ATG, depending on the patient's risk factors of regimen related cardiotoxicity. | Drug | Control group |
Primary outcome
Failure free survival
Time frame: 1 year post HSCT
Failure free survival was defined as survival with a response to therapy.
7 secondary outcomes
The incidence of mixed chimerism
Time frame: 1 year post HSCT
Regimen related toxicity
Time frame: 100 days post HSCT
Myeloid and platelet engraftment
Time frame: 100 days post HSCT
The incidence of graft versus host disease
Time frame: 100 days post HSCT for aGvHD and 1 year post HSCT for cGvHD
The incidence of CMV and EBV reactivation
Time frame: 100 days post HSCT
The incidence of Transplantation related mortality
Time frame: 1 year post HSCT
The probability of Overall survival
Time frame: 1 year post HSCT
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2023.
- Individual participant dataSharing planned: No
Related trials for Severe Aplastic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 2
Allogeneic Hematopoietic Stem Cell Transplantation for Severe Aplastic Anemia and Other Bone Marrow Failure Syndromes Using G-CSF Mobilized CD34+ Selected Hematopoietic Precursor Cells Co-Infused With a Reduced Dose of Non-Mobilized Donor T-cells
Severe Aplastic Anemia, MDS (Myelodysplastic Syndrome)
RecruitingPhase 1/2
A Phase 1/2 Study to Evaluate the Safety and Efficacy of Dibotatug (DR-01) in Adults With Bone Marrow Failure Syndromes
Bone Marrow Failure Syndromes, Aplastic Anemias
Active, not recruitingPhase 2
Standard Immunosuppressive Therapy Combined With Romiplostim N01 as First-line Treatment for Severe Aplastic Anemia
Aplastic Anemia, TPO Receptor Agonists
RecruitingObservational study
Natural History of Acquired and Inherited Bone Marrow Failure Syndromes
Severe Aplastic Anemia, Telomere Biology Disorders
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.
Xiao-Jun Huang principal investigator
Peking University People's Hospital
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 and Flu/Cy/ATG or Cy/ATG in people with Severe Aplastic Anemia, run by Peking University People's Hospital 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 9 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: status not confirmed, as of the sponsor's last update on 18 Nov 2023. 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 Zheng-Li Xu, M.D. (+8613501338951, xuzhengli0202@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Failure free survival (time frame: 1 year post HSCT). 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 18 Nov 2023.