Azacitidine and Homoharringtonine in JMML

Sponsor

Air Force Military Medical University, China (Other)

Overall Status

Recruiting

CT.gov ID

NCT04505995

Collaborator

Qilu Hospital of Shandong University (Other), Taian City Central Hospital (Other), The Affiliated Hospital of Qingdao University (Other), The Second Hospital of Hebei Medical University (Other), Children's Hospital of Hebei Province (Other)

Enrollment

Location

Arm

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Juvenile myelomonocytic leukemia (JMML) has a poor prognosis in general, with hematopoietic stem cell transplant (HSCT) remaining the standard of care for cure. Azacitidine and homoharringtonine, which exhibited a anticancer mechanism and has been widely used in patients with relapse/refractory acute myeloid leukemia (AML). However, the efficacy of these agents has not been elucidated in children with JMML. The primary objective is to assess the treatment effect on response rate in this subject population.

Condition or Disease	Intervention/Treatment	Phase
JMML	Drug: Azacitidine and homoharringtonine	N/A

Study Design

Study Type:

Interventional

Anticipated Enrollment :

30 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia

Actual Study Start Date :

Jan 1, 2020

Anticipated Primary Completion Date :

Jan 1, 2023

Anticipated Study Completion Date :

Jan 1, 2025

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Intervention group	Drug: Azacitidine and homoharringtonine Concommitant use of azacitidine and homoharringtonine

Arm

Intervention/Treatment

Experimental: Intervention group

Drug: Azacitidine and homoharringtonine

Concommitant use of azacitidine and homoharringtonine

Outcome Measures

Primary Outcome Measures

Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months [Up to 4 Months]
Defined as proportion of patients with sustained clinical complete remission [cCR] or clinical partial remission [cPR] according to the International JMML response criteria in Niemeyer 2014 at 3 months (28 days cycles). Response must be sustained for at least 4 weeks either in the 4-week period preceding or succeeding 3 months (ie, sustained over the period minimum 2 months to end of 3 months, or end of 3 months to end of 4 months).

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Month to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Newly diagnosed JMML, with PB and BM confirming diagnosis prior to informed consent signature, with one of the following:

somatic mutation in PTPN11
somatic mutation in KRAS
somatic mutation in NRAS and HbF % > 5x normal value for age
clinical diagnosis of neurofibromatosis Type 1.

Exclusion Criteria:

1. Any condition that would prevent the subject from participating in the study.

Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study.
Any condition that confounds the ability to interpret data from the study.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College	Tianjin	Tianjin	China	300020

Sponsors and Collaborators

Air Force Military Medical University, China
Qilu Hospital of Shandong University
Taian City Central Hospital
The Affiliated Hospital of Qingdao University
The Second Hospital of Hebei Medical University
Children's Hospital of Hebei Province

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Lipeng Liu, Dr, Air Force Military Medical University, China

ClinicalTrials.gov Identifier:

NCT04505995

Other Study ID Numbers:

JMML-CAMS-2020

First Posted:

Aug 10, 2020

Last Update Posted:

Aug 10, 2020

Last Verified:

Aug 1, 2020

Individual Participant Data (IPD) Sharing Statement:

Undecided

Plan to Share IPD:

Undecided

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Azacitidine

Homoharringtonine

Study Results

No Results Posted as of Aug 10, 2020