Myelodysplastic SyndromePhase 2/3NCT02744742

G-CSF+Decitabine+BUCY vs BUCY Conditioning Regimen for RAEB-1, REAB-2 and AML Secondary to MDS Undergoing Allo-HSCT

A Phase 2/3 study testing Decitabine, Busulfan (BU) and Cyclophosphamide (CY) in people with Myelodysplastic Syndrome and Allogeneic Hematopoietic Stem Cell Transplantation, run by Nanfang Hospital, Southern Medical University at 1 site in China.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

Who is it for?: Ages 14 to 65 years
Conditions the study is for: Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
What is being tested?: Decitabine, Busulfan (BU) and Cyclophosphamide (CY)
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 202 participants
Where: 1 siteChina
Study dates: 18 Apr 2016to 30 Sep 2021
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 14 to 65 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT
14-65 years

Who cannot join

Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
Patients 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

Other trials in China

About this study

Official title: Granulocyte Colony-stimulating Factor+Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation

Allogeneic hematopoietic stem cell transplantation (allo-HSCT) appears to be an efficient tool to cure refractory anemia with excess blasts-1 (RAEB-1), refractory anemia with excess blasts-2 (RAEB-2) and acute myeloid leukemia (AML) secondary to myelodysplastic syndrome (MDS). At present, the best conditioning regimen for RAEB-1, RAEB-2 and AML secondary to MDS undergoing allo-HSCT remains in discussion. In this prospective randomized controlled study, the safety and efficacy of G-CSF+ Decitabine + BUCY and BUCY myeloablative conditioning regimens in patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT are evaluated.

Read the detailed description

Allo-HSCT appears to be an efficient tool to cure patients with MDS and AML secondary to MDS. At present, the best conditioning regimen for MDS and AML secondary to MDS undergoing allo-HSCT remains in discussion. BUCY conditioning regimen is the standard myeloablative regimen for MDS and AML secondary to MDS undergoing allo-HSCT. However, it appears to have higher relapse rate. To reduce the relapse rate, decitabine is added in the conditioning regimen. In this prospective randomized controlled study, the safety and efficacy of G-CSF + Decitabine + BUCY and BUCY myeloablative conditioning regimens in RAEB-1, REAB-2 and AML secondary to MDS undergoing allo-HSCT are evaluated.

Conditions: Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
Being tested: Decitabine, Busulfan (BU), Cyclophosphamide (CY), Granulocyte Colony-Stimulating Factor(G-CSF)
Medical subject headings: Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

DecitabineDrug
Decitabine was administered at 20mg/m2/day on days -14 and -10.
Busulfan (BU)Drug
Busulfan was administered at 3.2 mg/kg/day on days -7 to -4.
Cyclophosphamide (CY)Drug
Cyclophosphamide was administered at 60 mg/kg/day on days -3 to -2.
Granulocyte Colony-Stimulating Factor(G-CSF)Drug
G-CSF was administered at 5-10 ug/kg/day on days -17 and -10. When white blood cell is more than 20G/L, stop using G-CSF.

Study groups in NCT02744742
Study group	Role	What they receive
G-CSF + Decitabine + BUCY	Experimental	For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，Granulocyte Colony-Stimulating Factor(G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5-10ug/kg/day on days -17 and -10 (when white blood cell is more than 20G/L, stop using G-CSF)；Decitabine 20mg/m2/day on days -14 and -10； Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2. Receives: Drug: Decitabine; Drug: Busulfan (BU); Drug: Cyclophosphamide (CY); Drug: Granulocyte Colony-Stimulating Factor(G-CSF)
BUCY	Active comparator	For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，BUCY conditioning regimen was Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2. Receives: Drug: Busulfan (BU); Drug: Cyclophosphamide (CY)

What is being measured

The primary outcome is the main question the study is designed to answer.

relapse rate
Measured: 2 year

Secondary outcomes (3)

overall survival (OS)
Measured: 2 year
disease-free survival (DFS)
Measured: 2 year
transplant-related mortality (TRM)
Measured: 2 year

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

18 Apr 201630 Sep 2021

First posted20 Apr 2016 (estimated)
Study start18 Apr 2016
Primary completion30 Sep 2019
Study completion30 Sep 2021

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 Myelodysplastic SyndromeStudies 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 2/3
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 202 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT02744742
Sponsor's protocol ID: G-CSF+Dec+BUCYvsBUCY-MDS-2016
Status last verified: Apr 2016
First submitted: 18 Apr 2016
Last updated: 14 Feb 2022
Results posted: No
Data monitoring committee: Yes
Individual data sharing: Yes

Publications

Xuan L, Dai M, Jiang E, Wang Y, Huang F, Fan Z, Xu N, Nie D, Liang X, Chen H, Ye J, Shi P, Liu H, Jin H, Lin R, Yan C, Zhang Y, Sun J, Han M, Liu Q. The effect of granulocyte-colony stimulating factor, decitabine, and busulfan-cyclophosphamide versus busulfan-cyclophosphamide conditioning on relapse in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023 Mar;10(3):e178-e190. doi: 10.1016/S2352-3026(22)00375-1. Epub 2023 Jan 23.
PubMed 36702138

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
High Grade Myelodysplastic Syndromes
RecruitingPhase 1/2
Pacritinib, a Kinase Inhibitor of CSF1R, IRAK1, JAK2, and FLT3, in Adults and Pediatric Participants 12 Years of Age or Older With Myelodysplastic Syndromes or Myelodysplastic/Myeloproliferative Neoplasms
Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
Adult Acute Lymphoblastic Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Not yet recruitingPhase 1
ATRN-119 in Combination With Decitabine in Patients With TP53-Mutated AML or Higher-Risk MDS
Acute Myeloid Leukemia, High-risk Myelodysplastic Syndrome

All Myelodysplastic Syndrome trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Feb 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Granulocyte Colony-stimulating Factor+Decitabine+Busulfan+Cyclophosphamide vs Busulfan+Cyclophosphamide Conditioning Regimen for Patients With RAEB-1, RAEB-2 and AML Secondary to MDS Undergoing Allogeneic Hematopoietic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT02744742
Acronym / sponsor ID: G-CSF+Dec+BUCYvsBUCY-MDS-2016
Conditions: Myelodysplastic Syndrome, Allogeneic Hematopoietic Stem Cell Transplantation, Conditioning
Phase: Phase 2/3
Study type: Interventional
Recruitment status: Completed
Enrolment: 202 participants (actual)
Lead sponsor: Nanfang Hospital, Southern Medical UniversityUniversity, hospital or other
Collaborators: Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University, Institute of Hematology & Blood Diseases Hospital, China, Peking University People's Hospital, First People's Hospital of Chenzhou, Liuzhou Workers' Hospital
Study start: 18 Apr 2016 (actual)
Primary completion: 30 Sep 2019 (actual)
Study completion: 30 Sep 2021 (actual)
First posted: 20 Apr 2016 (estimated)
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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
G-CSF + Decitabine + BUCY	Experimental	Drug: Decitabine, Drug: Busulfan (BU), Drug: Cyclophosphamide (CY), Drug: Granulocyte Colony-Stimulating Factor(G-CSF)For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，Granulocyte Colony-Stimulating Factor(G-CSF)+Decitabine+BUCY conditioning regimen was G-CSF 5-10ug/kg/day on days -17 and -10 (when white blood cell is more than 20G/L, stop using G-CSF)；Decitabine 20mg/m2/day on days -14 and -10； Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2.
BUCY	Active comparator	Drug: Busulfan (BU), Drug: Cyclophosphamide (CY)For patients with RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT ，BUCY conditioning regimen was Busulfan (BU) 3.2 mg/kg/day on days -7 and -4；Cyclophosphamide (CY) 60 mg/kg/day on days -3 and -2.

Who may qualify

Ages: 14 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (2 listed)

RAEB-1, REAB-2 and AML Secondary to MDS undergoing allo-HSCT
14-65 years

Key exclusion criteria (2 listed)

Any abnormality in a vital sign (e.g., heart rate, respiratory rate, or blood pressure)
Patients with any conditions not suitable for the trial (investigators' decision)

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
DecitabineDecitabine was administered at 20mg/m2/day on days -14 and -10.	Drug	G-CSF + Decitabine + BUCY
Busulfan (BU)Busulfan was administered at 3.2 mg/kg/day on days -7 to -4.	Drug	BUCY, G-CSF + Decitabine + BUCY
Cyclophosphamide (CY)Cyclophosphamide was administered at 60 mg/kg/day on days -3 to -2.	Drug	BUCY, G-CSF + Decitabine + BUCY
Granulocyte Colony-Stimulating Factor(G-CSF)G-CSF was administered at 5-10 ug/kg/day on days -17 and -10. When white blood cell is more than 20G/L, stop using G-CSF.	Drug	G-CSF + Decitabine + BUCY

Primary outcome

relapse rate
Time frame: 2 year

3 secondary outcomes

overall survival (OS)
Time frame: 2 year
disease-free survival (DFS)
Time frame: 2 year
transplant-related mortality (TRM)
Time frame: 2 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 14 Feb 2022.
Derived from this study: PubMed 36702138Xuan L, Dai M, Jiang E, Wang Y, Huang F, Fan Z, Xu N, Nie D, Liang X, Chen H, Ye J, Shi P, Liu H, Jin H, Lin R, Yan C, Zhang Y, Sun J, Han M, Liu Q. The effect of granulocyte-colony stimulating factor, decitabine, and busulfan-cyclophosphamide versus busulfan-cyclophosphamide conditioning on relapse in patients with myelodysplastic syndrome or secondary acute myeloid leukaemia evolving from myelodysplastic syndrome undergoing allogeneic haematopoietic stem-cell transplantation: an open-label, multicentre, randomised, phase 3 trial. Lancet Haematol. 2023 Mar;10(3):e178-e190. doi: 10.1016/S2352-3026(22)00375-1. Epub 2023 Jan 23.
Individual participant dataSharing planned: Yes

Related trials for Myelodysplastic Syndrome

RecruitingPhase 2
Testing New Therapies for Patients With Acute Myeloid Leukemia Who Are Newly Diagnosed and Have Not Yet Started Treatment (A MyeloMATCH Treatment Trial)
Acute Myeloid Leukemia, Acute Myeloid Leukemia Arising From Previous Myelodysplastic/Myeloproliferative Neoplasm
Active, not recruitingPhase 1/2
Omacetaxine + Azacitidine in Untreated Patients With High Grade MDS
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RecruitingPhase 1/2
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Myelodysplastic Syndromes
Active, not recruitingPhase 2
Clofarabine and Melphalan Before Donor Stem Cell Transplant in Treating Patients With Myelodysplasia, Acute Leukemia in Remission, or Chronic Myelomonocytic Leukemia
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Not yet recruitingPhase 1
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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?

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 2 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: finished, as of the sponsor's last update on 14 Feb 2022. 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?

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?

relapse rate (time frame: 2 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 NCT02744742, 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 14 Feb 2022.