NCT04582604

Brief 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.

Trial Health

43
At Risk

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Trial has exceeded expected completion date
Enrollment
60

participants targeted

Target at P75+ for phase_1

Timeline
Completed

Started Sep 2020

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
unknown

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

September 1, 2020

Completed
1 month until next milestone

First Submitted

Initial submission to the registry

October 3, 2020

Completed
6 days until next milestone

First Posted

Study publicly available on registry

October 9, 2020

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2023

Completed
2.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

September 30, 2025

Completed
Last Updated

October 20, 2020

Status Verified

October 1, 2020

Enrollment Period

3 years

First QC Date

October 3, 2020

Last Update Submit

October 19, 2020

Conditions

Peripheral Blood Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

  • Number of participants relapse as assessed by NCCN (National Comprehensive Cancer Network )criteria

    Defined as the proportion of participants whose underlying malignancy relapsed.

    365 days after transplantation

Secondary Outcomes (7)

  • DFS(disease-free survival )

    365 days after transplantation

  • TRM(treatment-related mortality )

    365 days after transplantation

  • Number of participants with aGVHD as assessed by acute graft versus host disease grading criteria (refer to Glucksberg criteria)

    100 days after transplantation

  • Number of participants with cGVHD as assessed by chronic graft versus host disease grading criteria (refer to NIH criteria)

    365 days after transplantation

  • OS(overall survival )

    365 days after transplantation

  • +2 more secondary outcomes

Study Arms (1)

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),

Drug: modified By/Cy conditioning regimen intensified by Ruxolitinib and Decitabine

Interventions

modified By/Cy conditioning regimen intensified by Ruxolitinib and DecitabineDRUG

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;

Also known as: Ruxolitinib and Decitabine
Ruxolitinib combined with Decitabine

Eligibility Criteria

Age12 Years - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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;
  • 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.

You may not qualify if:

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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Chinese PLA General Hospital

Beijing, Beijing Municipality, 100853, China

RECRUITING

Related Publications (7)

  • 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.

    PMID: 28619982BACKGROUND

  • 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.

    PMID: 25516983BACKGROUND

  • 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.

    PMID: 28484265BACKGROUND

  • 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.

    PMID: 31677846BACKGROUND

  • 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.

    PMID: 29773603BACKGROUND

  • 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.

    PMID: 40901473DERIVED

  • 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.

    PMID: 39243311DERIVED

MeSH Terms

Interventions

Decitabineruxolitinib

Intervention Hierarchy (Ancestors)

AzacitidineAza CompoundsOrganic ChemicalsCytidinePyrimidine NucleosidesPyrimidinesHeterocyclic Compounds, 1-RingHeterocyclic CompoundsNucleosidesNucleic Acids, Nucleotides, and NucleosidesRibonucleosides

Study Officials

  • Daihong Liu

    Chinese PLA General Hospital

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Daihong Liu

CONTACT

86-13681171597daihongrm@163.com

Liping Dou

CONTACT

96-13681207138lipingruirui@163.com

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director

Study Record Dates

First Submitted

October 3, 2020

First Posted

October 9, 2020

Study Start

September 1, 2020

Primary Completion

September 1, 2023

Study Completion

September 30, 2025

Last Updated

October 20, 2020

Record last verified: 2020-10

Data Sharing

IPD Sharing
Will not share

Locations

CN(1)