Myelodysplastic SyndromePhase 2/3NCT05453552
G-CSF+DAC+BUCY vs G-CSF+DAC+BF Conditioning Regimen for High-risk MDS Undergoing Allo-HSCT
A Phase 2/3 study testing Granulocyte Colony-Stimulating Factor(G-CSF), Decitabine (DAC) and Busulfan in people with Myelodysplastic Syndrome and Allogeneic Hematopoietic Stem Cell Transplantation, run by Nanfang Hospital, Southern Medical University at 1 site in China.
Trial summary
Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
- What is being tested?
- Granulocyte Colony-Stimulating Factor(G-CSF), Decitabine (DAC) and Busulfan
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 242 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2022to Dec 2024
- Phase
- Phase 2/3
- 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
- Had a diagnosis of RAEB-1 or RAEB-2 with IPSS-R >3
- Age 18 to 65 years old
- ECOG performance status of 0-2
- HCT-CI of 0-2
- Were willing to undergo allo-HSCT
Who cannot join
- Therapy-related MDS
- Previous allo-HSCT
- Uncontrolled infections
- Liver or renal dysfunction
- Severe concomitant conditions not suitable for the trial
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Department of Hematology,Nanfang Hospital, Southern Medical University
Guangzhou, Guangdong, 510515
Li Xuan, 15521251270, 356135708@qq.com
About this study
Official title: G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. In this prospective study, the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT are evaluated.
Read the detailed description
Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. A previous study by the investigators has showed that G-CSF +DAC+BUCY conditioning regimen could reduce the relapse and improve the survival compared with BUCY conditioning regimen, while the two conditioning regimens both have high non-relapse mortality (NRM). Several retrospective and prospective studies have demonstrated that BF conditioning regimen has a lower NRM compared with BUCY conditioning regimen, while the relapse and survival are similar in patients undergoing BF and BUCY conditioning regimens. Based on the above, the investigators design the prospective randomized controlled study to evaluate the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT.
- Conditions
- Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
- Being tested
- Granulocyte Colony-Stimulating Factor(G-CSF), Decitabine (DAC), Busulfan, Fludarabine (FLU), Cyclophosphamide (CY)
- Medical subject headings
- Myelodysplastic Syndromes
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Granulocyte Colony-Stimulating Factor(G-CSF)Drug
G-CSF was administered at 5 ug/kg/day on days-17 to -10. When white blood cell is more than 20G/L, stop using G-CSF.
Decitabine (DAC)Drug
Decitabine was administered at 20mg/m2/day on days -14 to -10.
BusulfanDrug
Busulfan was administered at 3.2 mg/kg/day on days -7 to -4 in G-CSF+DAC+BUCY group.
Fludarabine (FLU)Drug
Fludarabine was administered at 30 mg/m2/day on days -7 to -3.
Cyclophosphamide (CY)Drug
Cyclophosphamide was administered at 60 mg/kg/day on days -3,-2.
Busulfan (BU)Drug
Busulfan was administered at 3.2 mg/kg/day on days -6 to -3 in G-CSF+DAC +BF group.
| Study group | Role | What they receive |
|---|---|---|
| G-CSF+DAC+BF | Experimental | For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony-Stimulating Factor (G-CSF)+Decitabine+BF conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -6 to -3, Fludarabine (FLU) 30mg/m2/ day on days -7 to -3. Receives: Drug: Granulocyte Colony-Stimulating Factor(G-CSF); Drug: Decitabine (DAC); Drug: Fludarabine (FLU); Drug: Busulfan (BU) |
| G-CSF+DAC+BUCY | Active comparator | For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony -Stimulating Factor (G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -7 to -4, Cyclophosphamide (CY) 60 mg/kg/day on days -3, -2. Receives: Drug: Granulocyte Colony-Stimulating Factor(G-CSF); Drug: Decitabine (DAC); Drug: Busulfan; Drug: Cyclophosphamide (CY) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Non-relapse mortality (NRM)
Measured: 1 year
Secondary outcomes (4)
Overall survival (OS)
Measured: 1 year
Disease-free survival (DFS)
Measured: 1 year
Cumulative incidence of relapse
Measured: 1 year
Adverse effects
Measured: within 100 days post-transplantation
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2022
- Study start1 Jul 2022
- Primary completionDec 2023 (estimated)
- Study completionDec 2024 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myelodysplastic SyndromeStudies 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 242 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Nanfang Hospital, Southern Medical University
- Sponsor type
- University, hospital or other
- Collaborators
- Peking University People's Hospital, Institute of Hematology & Blood Diseases Hospital, China, Guangzhou First People's Hospital, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, First People's Hospital of Chenzhou, The Seventh Affiliated Hospital of Sun Yat-sen University
- Responsible party
- Principal investigator, Qifa Liu, Nanfang Hospital, Southern Medical University
- Investigators
- Qifa Liu, Principal investigator, Nanfang Hospital, Southern Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05453552
- Sponsor's protocol ID
- NFEC-2022-233
- Status last verified
- Jul 2022
- First submitted
- 5 Jul 2022
- Last updated
- 21 Mar 2023
- 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 21 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- G-CSF+DAC+BUCY vs. G-CSF+DAC+BF Conditioning Regimen for Patients With High-risk MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
- ClinicalTrials.gov ID
- NCT05453552
- Acronym / sponsor ID
- NFEC-2022-233
- Conditions
- Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 242 participants (estimated)
- Lead sponsor
- Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
- Collaborators
- Peking University People's Hospital, Institute of Hematology & Blood Diseases Hospital, China, Guangzhou First People's Hospital, Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, First People's Hospital of Chenzhou, The Seventh Affiliated Hospital of Sun Yat-sen University
- Study start
- 1 Jul 2022 (actual)
- Primary completion
- Dec 2023 (estimated)
- Study completion
- Dec 2024 (estimated)
- First posted
- 12 Jul 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.
Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. In this prospective study, the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT are evaluated.
Detailed description
Allo-HSCT is the most effective way to cure high-risk MDS patients. At present, the best conditioning regimen for high-risk MDS patients undergoing allo-HSCT remains in discussion. A previous study by the investigators has showed that G-CSF +DAC+BUCY conditioning regimen could reduce the relapse and improve the survival compared with BUCY conditioning regimen, while the two conditioning regimens both have high non-relapse mortality (NRM). Several retrospective and prospective studies have demonstrated that BF conditioning regimen has a lower NRM compared with BUCY conditioning regimen, while the relapse and survival are similar in patients undergoing BF and BUCY conditioning regimens. Based on the above, the investigators design the prospective randomized controlled study to evaluate the safety and efficacy of G-CSF+DAC+BUCY and G-CSF+DAC+BF conditioning regimens in high-risk MDS patients undergoing allo-HSCT.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| G-CSF+DAC+BF | Experimental | Drug: Granulocyte Colony-Stimulating Factor(G-CSF), Drug: Decitabine (DAC), Drug: Fludarabine (FLU), Drug: Busulfan (BU)For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony-Stimulating Factor (G-CSF)+Decitabine+BF conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -6 to -3, Fludarabine (FLU) 30mg/m2/ day on days -7 to -3. |
| G-CSF+DAC+BUCY | Active comparator | Drug: Granulocyte Colony-Stimulating Factor(G-CSF), Drug: Decitabine (DAC), Drug: Busulfan, Drug: Cyclophosphamide (CY)For patients with high-risk MDS undergoing allo- HSCT, Granulocyte Colony -Stimulating Factor (G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5ug/kg/day on days -17 to -10 (when white blood cell is more than 20G/L, stop using G-CSF), Decitabine 20mg/m2/day on days -14 to -10, Busulfan (BU) 3.2 mg/kg/day on days -7 to -4, Cyclophosphamide (CY) 60 mg/kg/day on days -3, -2. |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Had a diagnosis of RAEB-1 or RAEB-2 with IPSS-R >3
- Age 18 to 65 years old
- ECOG performance status of 0-2
- HCT-CI of 0-2
- Were willing to undergo allo-HSCT
Key exclusion criteria (5 listed)
- Therapy-related MDS
- Previous allo-HSCT
- Uncontrolled infections
- Liver or renal dysfunction
- Severe concomitant conditions not suitable for the trial
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 |
|---|---|---|
| Granulocyte Colony-Stimulating Factor(G-CSF)G-CSF was administered at 5 ug/kg/day on days-17 to -10. When white blood cell is more than 20G/L, stop using G-CSF. | Drug | G-CSF+DAC+BF, G-CSF+DAC+BUCY |
| Decitabine (DAC)Decitabine was administered at 20mg/m2/day on days -14 to -10. | Drug | G-CSF+DAC+BF, G-CSF+DAC+BUCY |
| BusulfanBusulfan was administered at 3.2 mg/kg/day on days -7 to -4 in G-CSF+DAC+BUCY group. | Drug | G-CSF+DAC+BUCY |
| Fludarabine (FLU)Fludarabine was administered at 30 mg/m2/day on days -7 to -3. | Drug | G-CSF+DAC+BF |
| Cyclophosphamide (CY)Cyclophosphamide was administered at 60 mg/kg/day on days -3,-2. | Drug | G-CSF+DAC+BUCY |
| Busulfan (BU)Busulfan was administered at 3.2 mg/kg/day on days -6 to -3 in G-CSF+DAC +BF group. | Drug | G-CSF+DAC+BF |
Primary outcome
Non-relapse mortality (NRM)
Time frame: 1 year
4 secondary outcomes
Overall survival (OS)
Time frame: 1 year
Disease-free survival (DFS)
Time frame: 1 year
Cumulative incidence of relapse
Time frame: 1 year
Adverse effects
Time frame: within 100 days post-transplantation
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Mar 2023.
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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.
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 2/3 study testing Granulocyte Colony-Stimulating Factor(G-CSF), Decitabine (DAC) and Busulfan in people with Myelodysplastic Syndrome and Allogeneic 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 5 inclusion and 5 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 21 Mar 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 combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Li Xuan (+86-020-62787883, 356135708@qq.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Non-relapse mortality (NRM) (time frame: 1 year). 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 21 Mar 2023.