NCT03687463

Brief Summary

Hematopoietic stem cell transplantation (HSCT) is the only curative option for most of juvenile myelomonocytic leukemia (JMML). However, relapse after HSCT severely influence the long-term overall survival (OS). Researches demonstrate that these malignant myeloid disorders is a particular responsiveness to epigenetic therapy with the DNA-hypomethylating agents decitabine. However, hypomethylating therapy does not eradicate the malignant clone in JMML and an emerging concept with intriguing potential is the combination of hypomethylating therapy and HSCT. Graft-versus-host disease (GVHD) is major complication after HSCT as a threshold of the quality of patient life. Many data indicate that post -transplant cyclophosphamide (PT/Cy) is an effective method to control the occurrence of GVHD.

Trial Health

35
At Risk

Trial Health Score

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

Trial has exceeded expected completion date
Enrollment
6

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Apr 2015

Longer than P75 for all trials

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

April 10, 2015

Completed
3.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 10, 2018

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

September 25, 2018

Completed
2 days until next milestone

First Posted

Study publicly available on registry

September 27, 2018

Completed
1.5 years until next milestone

Study Completion

Last participant's last visit for all outcomes

April 10, 2020

Completed
Last Updated

October 2, 2018

Status Verified

September 1, 2018

Enrollment Period

3.3 years

First QC Date

September 25, 2018

Last Update Submit

September 30, 2018

Conditions

Hematopoietic System--Cancer

Keywords

HSCTPT/CyDecitabineJMML

Outcome Measures

Primary Outcomes (1)

  • disease statue

    Disease status can be measured by test the of (minimal residual disease) MRD, MRD\<0,01% as negative. The quantitative of gene mutation is "0" as negative.

    one months

Interventions

DecitabineDRUG

Decitabine for injection

Also known as: Dacogen

Eligibility Criteria

Age1 Year - 18 Years
Sexall(Gender-based eligibility)
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

JMML patients are urgent to transplant, but this group of patients without suitable donor.

You may qualify if:

  • JMML patients diagnosed in our center and with the indications of transplant without the suitable donor.

You may not qualify if:

  • JMML patients do not need to transplant.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (4)

  • Dvorak CC, Satwani P, Stieglitz E, Cairo MS, Dang H, Pei Q, Gao Y, Wall D, Mazor T, Olshen AB, Parker JS, Kahwash S, Hirsch B, Raimondi S, Patel N, Skeens M, Cooper T, Mehta PA, Grupp SA, Loh ML. Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatr Blood Cancer. 2018 Jul;65(7):e27034. doi: 10.1002/pbc.27034. Epub 2018 Mar 12.

    PMID: 29528181RESULT

  • Zaucha-Prazmo A, Gozdzik J, Debski R, Drabko K, Sadurska E, Kowalczyk JR. Transplant-related mortality and survival in children with malignancies treated with allogeneic hematopoietic stem cell transplantation. A multicenter analysis. Pediatr Transplant. 2018 May;22(3):e13158. doi: 10.1111/petr.13158. Epub 2018 Feb 3.

    PMID: 29396905RESULT

  • Flotho C, Sommer S, Lubbert M. DNA-hypomethylating agents as epigenetic therapy before and after allogeneic hematopoietic stem cell transplantation in myelodysplastic syndromes and juvenile myelomonocytic leukemia. Semin Cancer Biol. 2018 Aug;51:68-79. doi: 10.1016/j.semcancer.2017.10.011. Epub 2017 Nov 9.

    PMID: 29129488RESULT

  • Locatelli F, Niemeyer CM. How I treat juvenile myelomonocytic leukemia. Blood. 2015 Feb 12;125(7):1083-90. doi: 10.1182/blood-2014-08-550483. Epub 2015 Jan 6.

    PMID: 25564399RESULT

MeSH Terms

Interventions

Decitabine

Intervention Hierarchy (Ancestors)

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

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Target Duration
5 Years
Sponsor Type
OTHER GOV
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Clinical Professor

Study Record Dates

First Submitted

September 25, 2018

First Posted

September 27, 2018

Study Start

April 10, 2015

Primary Completion

July 10, 2018

Study Completion

April 10, 2020

Last Updated

October 2, 2018

Record last verified: 2018-09