Peripheral Blood Stem Cell TransplantationPhase 1/2NCT04582604

Ruxolitinib and Decitabine for High Risk Hematological Malignancies

A Phase 1/2 study testing modified By/Cy conditioning regimen intensified by Ruxolitinib and Decitabine in people with Peripheral Blood Stem Cell Transplantation, run by Chinese PLA General Hospital at 1 site in China.

Unknown status

Status not confirmed. The sponsor has not verified this record for over two years, so it may be out of date.

Official record

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Trial summary

The purpose of this study is to determine the efficacy and safety of Ruxolitinib and Decitabine intensified Conditioning Regimen in Patients with High Risk hematological malignancies undergoing allogeneic peripheral blood stem cell transplantation. Read more about this study

Who is it for?: Ages 12 to 65 years
Conditions the study is for: Peripheral Blood Stem Cell Transplantation
What is being tested?: modified By/Cy conditioning regimen intensified by Ruxolitinib and Decitabine
Type of treatment: Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 60 participants
Where: 1 siteChina
Study dates: 1 Sep 2020to 30 Sep 2025
Phase: Phase 1/2
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 12 to 65 years
All sexes
No healthy volunteers
Child, Adult, Older adult

Who may be able to join

Relapsed/refractory acute leukemia with indications for allogeneic hematopoietic stem cell transplantation; High risk acute leukemia with indications for allogeneic hematopoietic stem cell transplantation;
Medium to high risk myelodysplastic syndrome, myeloproliferative disease, myelodysplastic syndrome/myeloproliferative disease, Chronic myelomonocytic leukemia;
Have matched sibling donors, ≥8/10 HLA matched unrelated donors or haploidentical donors
All patients should aged 12 to 65 years;
Liver function: ALT and AST≤2.5 times the upper limit of normal , bilirubin≤2 times the upper limit of normal;
Renal function: creatinine ≤the upper limit of normal;

Show all 9Show fewer

Patients without any uncontrolled infections , without organ dysfunction or without severe mental illness;
Eastern Cooperative Oncology Group (ECOG) performance status ≤2;
Have signed informed consent.

Who cannot join

pregnant women;
Patients with mental illness or other states unable to comply with the protocol;
AML patients with t (15;17);

Trial locations and contacts

1 site in 1 country.

Daihong Liu contact

Liping Dou contact

China1 site, 1 recruiting

Chinese PLA General Hospital
Beijing, Beijing Municipality, 100853
Jiang Cao, 01066937166
Recruiting

Other trials in China

About this study

Official title: Ruxolitinib and Decitabine Intensified Conditioning Regimen for Patients With High Risk Hematological Malignancies Underwenting Allogeneic Stem Cell Transplantation

Read the detailed description

Allogeneic hematopoietic stem cell transplantation should be offered to eligible patients with high risk hematological malignancies whenever feasible. To further improve the outcome of transplantation patients with high risk hematological malignancies, the investigators developed a modified Bu/Cy conditioning regimen intensified by Ruxolitinib and Decitabine. In this study, the investigators tested the efficacy and feasibility of the modified Bu/Cy conditioning regimen intensified by Ruxolitinib and Decitabine in Patients with high risk hematological malignancies undergoing allogeneic peripheral blood stem cell transplantation.

Conditions: Peripheral Blood Stem Cell Transplantation
Being tested: modified By/Cy conditioning regimen intensified by Ruxolitinib and Decitabine

What participants receive

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

modified By/Cy conditioning regimen intensified by Ruxolitinib and DecitabineDrug
Also called Ruxolitinib and Decitabine
Day -15 to -14 ： Decitabine 20 mg/m2/day, Ruxolitinib 70mg bid; Day-10： Cytarabine 1.6 g/m2/day CI (only for Haploidentical and unrelated donor), Ruxolitinib 60mg bid; Day- 9： Cytarabine 4g/m2/day CI, Ruxolitinib 60mg bid; Day- 8 to -7： Busulfan 0.8mg/kg Q6h iv, Ruxolitinib 50mg bid; Day-6: Busulfan 0.8mg/kg Q6h iv, Ruxolitinib 40mg bid; Day-5： Cyclophosphamide 1.8 g/m2/day CI, Ruxolitinib 30mg bid; Day-4： Cyclophosphamide 1.8 g/m2/day CI, Ruxolitinib 20mg bid; Day-3： Carmustine 250mg/m2/day iv, Ruxolitinib 10mg bid; Day-2： Ruxolitinib 5mg bid; Day-1： Ruxolitinib 5mg qd;

Study groups in NCT04582604
Study group	Role	What they receive
Ruxolitinib combined with Decitabine	Experimental	Ruxolitinib and Decitabine conditioning regimen All recipients in this arm received the modified Bu/Cy conditioning regimen intensified by Ruxolitinib and Decitabine. The conditioning regimen for allogeneic hematopoietic stem cell transplantation consist of ruxolitinib (35 mg bid [p.o.], days -15 to -10, diminishing to day -1), decitabine (20 mg/m2/day, days -15 to -10), cytarabine (4 g/m2/day, days -10 to -9 (for unrelated donors or haploidentical donors; and 4 g/m2/day, days -9 for sibling donors)), busulfan (0.8mg/kg, Q6h, days -8 to -6), cyclophosphamide (1.8 g/m2/day, days -5 to -4);carmustine(BCNU)(250mg/m2/day, day -3), Receives: Drug: modified By/Cy conditioning regimen intensified by Ruxolitinib and Decitabine

What is being measured

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

Number of participants relapse as assessed by NCCN (National Comprehensive Cancer Network )criteria
Defined as the proportion of participants whose underlying malignancy relapsed.
Measured: 365 days after transplantation

Secondary outcomes (7)

DFS(disease-free survival )
DFS was defined as survival with no evidence of relapse or progression.
Measured: 365 days after transplantation
TRM(treatment-related mortality )
Defined as the proportion of subjects who died due to causes other than malignancy relapse.
Measured: 365 days after transplantation
Number of participants with aGVHD as assessed by acute graft versus host disease grading criteria (refer to Glucksberg criteria)
Defined as the proportion of participants who developed acute GVHD.
Measured: 100 days after transplantation
Number of participants with cGVHD as assessed by chronic graft versus host disease grading criteria (refer to NIH criteria)
Defined as the proportion of participants who developed chronic GVHD.
Measured: 365 days after transplantation
OS(overall survival )
OS was defined as the time from transplantation to death due to any cause.
Measured: 365 days after transplantation
GRFS (GVHD free, relapse free survival)
GVHD-free, relapse-free survival (GRFS) was defined as survival with no evidence of grade III-IV acute GVHD or cGVHD requiring immunosuppressive treatment, and without disease recurrence or death from any cause during the first year after transplantation.
Measured: 365 days after transplantation
infection rate
Defined as the proportion of participants who developed all kinds of infection.
Measured: 365 days after transplantation

Timeline

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

1 Sep 202030 Sep 2025

First posted9 Oct 2020
Study start1 Sep 2020
Primary completion1 Sep 2023 (estimated)
Study completion30 Sep 2025 (estimated)

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (7)Papers the sponsor has linked to this study.
Other trials for Peripheral Blood Stem Cell TransplantationStudies of the same condition, open and under way.
More trials run by Chinese PLA General 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 1/2
Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 60 participants (estimated)

IDs, dates and publications

ClinicalTrials.gov ID: NCT04582604
Sponsor's protocol ID: S2020-297-01
Status last verified: Oct 2020
First submitted: 3 Oct 2020
Last updated: 20 Oct 2020
Results posted: No
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Publications

Karjalainen R, Pemovska T, Popa M, Liu M, Javarappa KK, Majumder MM, Yadav B, Tamborero D, Tang J, Bychkov D, Kontro M, Parsons A, Suvela M, Mayoral Safont M, Porkka K, Aittokallio T, Kallioniemi O, McCormack E, Gjertsen BT, Wennerberg K, Knowles J, Heckman CA. JAK1/2 and BCL2 inhibitors synergize to counteract bone marrow stromal cell-induced protection of AML. Blood. 2017 Aug 10;130(6):789-802. doi: 10.1182/blood-2016-02-699363. Epub 2017 Jun 15.
PubMed 28619982
Rampal R, Ahn J, Abdel-Wahab O, Nahas M, Wang K, Lipson D, Otto GA, Yelensky R, Hricik T, McKenney AS, Chiosis G, Chung YR, Pandey S, van den Brink MR, Armstrong SA, Dogan A, Intlekofer A, Manshouri T, Park CY, Verstovsek S, Rapaport F, Stephens PJ, Miller VA, Levine RL. Genomic and functional analysis of leukemic transformation of myeloproliferative neoplasms. Proc Natl Acad Sci U S A. 2014 Dec 16;111(50):E5401-10. doi: 10.1073/pnas.1407792111. Epub 2014 Dec 2.
PubMed 25516983
Delgado-Martin C, Meyer LK, Huang BJ, Shimano KA, Zinter MS, Nguyen JV, Smith GA, Taunton J, Winter SS, Roderick JR, Kelliher MA, Horton TM, Wood BL, Teachey DT, Hermiston ML. JAK/STAT pathway inhibition overcomes IL7-induced glucocorticoid resistance in a subset of human T-cell acute lymphoblastic leukemias. Leukemia. 2017 Dec;31(12):2568-2576. doi: 10.1038/leu.2017.136. Epub 2017 May 9.
PubMed 28484265
Venugopal S, Bar-Natan M, Mascarenhas JO. JAKs to STATs: A tantalizing therapeutic target in acute myeloid leukemia. Blood Rev. 2020 Mar;40:100634. doi: 10.1016/j.blre.2019.100634. Epub 2019 Oct 25.
PubMed 31677846
Ding YY, Stern JW, Jubelirer TF, Wertheim GB, Lin F, Chang F, Gu Z, Mullighan CG, Li Y, Harvey RC, Chen IM, Willman CL, Hunger SP, Li MM, Tasian SK. Clinical efficacy of ruxolitinib and chemotherapy in a child with Philadelphia chromosome-like acute lymphoblastic leukemia with GOLGA5-JAK2 fusion and induction failure. Haematologica. 2018 Sep;103(9):e427-e431. doi: 10.3324/haematol.2018.192088. Epub 2018 May 17. No abstract available.
PubMed 29773603
Wei Y, Luan S, Wang L, Wang L, Li F, Jin X, Yang R, Qian K, Peng B, Tang J, Zhang H, Dou L, Liu D. Ruxolitinib and decitabine plus a busulfan-cyclophosphamide conditioning regimen for relapse prophylaxis in patients with high-risk acute myeloid leukemia or myelodysplastic syndromes. Front Immunol. 2025 Aug 18;16:1586512. doi: 10.3389/fimmu.2025.1586512. eCollection 2025.
PubMed 40901473
Wei Y, Qian K, Le N, Wang L, Li F, Luan S, Wang L, Jin X, Peng B, Wang N, Dou L, Liu D. Addition of ruxolitinib and decitabine to modified busulfan/cyclophosphamide conditioning regimen for prophylaxis relapse in high-risk acute myeloid leukemia: the phase 2 prospective study. Ann Hematol. 2024 Nov;103(11):4707-4719. doi: 10.1007/s00277-024-05972-w. Epub 2024 Sep 7.
PubMed 39243311

Study at a glance

From the registry record, last updated by the sponsor on 20 Oct 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Ruxolitinib and Decitabine Intensified Conditioning Regimen for Patients With High Risk Hematological Malignancies Underwenting Allogeneic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT04582604
Acronym / sponsor ID: S2020-297-01
Conditions: Peripheral Blood Stem Cell Transplantation
Phase: Phase 1/2
Study type: Interventional
Recruitment status: Unknown status
Enrolment: 60 participants (estimated)
Lead sponsor: Chinese PLA General HospitalUniversity, hospital or other
Study start: 1 Sep 2020 (actual)
Primary completion: 1 Sep 2023 (estimated)
Study completion: 30 Sep 2025 (estimated)
First posted: 9 Oct 2020 (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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Ruxolitinib combined with Decitabine	Experimental	Drug: modified By/Cy conditioning regimen intensified by Ruxolitinib and DecitabineRuxolitinib and Decitabine conditioning regimen All recipients in this arm received the modified Bu/Cy conditioning regimen intensified by Ruxolitinib and Decitabine. The conditioning regimen for allogeneic hematopoietic stem cell transplantation consist of ruxolitinib (35 mg bid [p.o.], days -15 to -10, diminishing to day -1), decitabine (20 mg/m2/day, days -15 to -10), cytarabine (4 g/m2/day, days -10 to -9 (for unrelated donors or haploidentical donors; and 4 g/m2/day, days -9 for sibling donors)), busulfan (0.8mg/kg, Q6h, days -8 to -6), cyclophosphamide (1.8 g/m2/day, days -5 to -4);carmustine(BCNU)(250mg/m2/day, day -3),

Who may qualify

Ages: 12 to 65 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child, Adult, Older adult

Key inclusion criteria (9 listed)

Relapsed/refractory acute leukemia with indications for allogeneic hematopoietic stem cell transplantation; High risk acute leukemia with indications for allogeneic hematopoietic stem cell transplantation;
Medium to high risk myelodysplastic syndrome, myeloproliferative disease, myelodysplastic syndrome/myeloproliferative disease, Chronic myelomonocytic leukemia;
Have matched sibling donors, ≥8/10 HLA matched unrelated donors or haploidentical donors
All patients should aged 12 to 65 years;
Liver function: ALT and AST≤2.5 times the upper limit of normal , bilirubin≤2 times the upper limit of normal;

Key exclusion criteria (3 listed)

pregnant women;
Patients with mental illness or other states unable to comply with the protocol;
AML patients with t (15;17);

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
modified By/Cy conditioning regimen intensified by Ruxolitinib and DecitabineAlso: Ruxolitinib and DecitabineDay -15 to -14 ： Decitabine 20 mg/m2/day, Ruxolitinib 70mg bid; Day-10： Cytarabine 1.6 g/m2/day CI (only for Haploidentical and unrelated donor), Ruxolitinib 60mg bid; Day- 9： Cytarabine 4g/m2/day CI, Ruxolitinib 60mg bid; Day- 8 to -7： Busulfan 0.8mg/kg Q6h iv, Ruxolitinib 50mg bid; Day-6: Busulfan 0.8mg/kg Q6h iv, Ruxolitinib 40mg bid; Day-5： Cyclophosphamide 1.8 g/m2/day CI, Ruxolitinib 30mg bid; Day-4： Cyclophosphamide 1.8 g/m2/day CI, Ruxolitinib 20mg bid; Day-3： Carmustine 250mg/m2/day iv, Ruxolitinib 10mg bid; Day-2： Ruxolitinib 5mg bid; Day-1： Ruxolitinib 5mg qd;	Drug	Ruxolitinib combined with Decitabine

Primary outcome

Number of participants relapse as assessed by NCCN (National Comprehensive Cancer Network )criteria
Time frame: 365 days after transplantation
Defined as the proportion of participants whose underlying malignancy relapsed.

7 secondary outcomes

DFS(disease-free survival )
Time frame: 365 days after transplantation
TRM(treatment-related mortality )
Time frame: 365 days after transplantation
Number of participants with aGVHD as assessed by acute graft versus host disease grading criteria (refer to Glucksberg criteria)
Time frame: 100 days after transplantation
Number of participants with cGVHD as assessed by chronic graft versus host disease grading criteria (refer to NIH criteria)
Time frame: 365 days after transplantation
OS(overall survival )
Time frame: 365 days after transplantation
GRFS (GVHD free, relapse free survival)
Time frame: 365 days after transplantation
infection rate
Time frame: 365 days after transplantation

Results and safety

Results are not available in the source record.

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 20 Oct 2020.
Background: PubMed 28619982Karjalainen R, Pemovska T, Popa M, Liu M, Javarappa KK, Majumder MM, Yadav B, Tamborero D, Tang J, Bychkov D, Kontro M, Parsons A, Suvela M, Mayoral Safont M, Porkka K, Aittokallio T, Kallioniemi O, McCormack E, Gjertsen BT, Wennerberg K, Knowles J, Heckman CA. JAK1/2 and BCL2 inhibitors synergize to counteract bone marrow stromal cell-induced protection of AML. Blood. 2017 Aug 10;130(6):789-802. doi: 10.1182/blood-2016-02-699363. Epub 2017 Jun 15.
Background: PubMed 25516983Rampal R, Ahn J, Abdel-Wahab O, Nahas M, Wang K, Lipson D, Otto GA, Yelensky R, Hricik T, McKenney AS, Chiosis G, Chung YR, Pandey S, van den Brink MR, Armstrong SA, Dogan A, Intlekofer A, Manshouri T, Park CY, Verstovsek S, Rapaport F, Stephens PJ, Miller VA, Levine RL. Genomic and functional analysis of leukemic transformation of myeloproliferative neoplasms. Proc Natl Acad Sci U S A. 2014 Dec 16;111(50):E5401-10. doi: 10.1073/pnas.1407792111. Epub 2014 Dec 2.
Background: PubMed 28484265Delgado-Martin C, Meyer LK, Huang BJ, Shimano KA, Zinter MS, Nguyen JV, Smith GA, Taunton J, Winter SS, Roderick JR, Kelliher MA, Horton TM, Wood BL, Teachey DT, Hermiston ML. JAK/STAT pathway inhibition overcomes IL7-induced glucocorticoid resistance in a subset of human T-cell acute lymphoblastic leukemias. Leukemia. 2017 Dec;31(12):2568-2576. doi: 10.1038/leu.2017.136. Epub 2017 May 9.
Background: PubMed 31677846Venugopal S, Bar-Natan M, Mascarenhas JO. JAKs to STATs: A tantalizing therapeutic target in acute myeloid leukemia. Blood Rev. 2020 Mar;40:100634. doi: 10.1016/j.blre.2019.100634. Epub 2019 Oct 25.
Background: PubMed 29773603Ding YY, Stern JW, Jubelirer TF, Wertheim GB, Lin F, Chang F, Gu Z, Mullighan CG, Li Y, Harvey RC, Chen IM, Willman CL, Hunger SP, Li MM, Tasian SK. Clinical efficacy of ruxolitinib and chemotherapy in a child with Philadelphia chromosome-like acute lymphoblastic leukemia with GOLGA5-JAK2 fusion and induction failure. Haematologica. 2018 Sep;103(9):e427-e431. doi: 10.3324/haematol.2018.192088. Epub 2018 May 17. No abstract available.
Derived from this study: PubMed 40901473Wei Y, Luan S, Wang L, Wang L, Li F, Jin X, Yang R, Qian K, Peng B, Tang J, Zhang H, Dou L, Liu D. Ruxolitinib and decitabine plus a busulfan-cyclophosphamide conditioning regimen for relapse prophylaxis in patients with high-risk acute myeloid leukemia or myelodysplastic syndromes. Front Immunol. 2025 Aug 18;16:1586512. doi: 10.3389/fimmu.2025.1586512. eCollection 2025.
Derived from this study: PubMed 39243311Wei Y, Qian K, Le N, Wang L, Li F, Luan S, Wang L, Jin X, Peng B, Wang N, Dou L, Liu D. Addition of ruxolitinib and decitabine to modified busulfan/cyclophosphamide conditioning regimen for prophylaxis relapse in high-risk acute myeloid leukemia: the phase 2 prospective study. Ann Hematol. 2024 Nov;103(11):4707-4719. doi: 10.1007/s00277-024-05972-w. Epub 2024 Sep 7.
Individual participant dataSharing planned: No

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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.

Daihong Liu contact

Liping Dou contact

Daihong Liu principal investigator

Chinese PLA General Hospital

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 9 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: status not confirmed, as of the sponsor's last update on 20 Oct 2020. 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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 Daihong Liu (86-13681171597, daihongrm@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Number of participants relapse as assessed by NCCN (National Comprehensive Cancer Network )criteria (time frame: 365 days after transplantation). 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 NCT04582604, which also lists 7 linked publications.

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 20 Oct 2020.