Azacitidine and Homoharringtonine in JMML

NCT ID: NCT04505995

Last Updated: 2020-08-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

NA

Total Enrollment

30 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-01-01

Study Completion Date

2025-01-01

Brief Summary

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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.

Detailed Description

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Conditions

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JMML

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Intervention group

Group Type EXPERIMENTAL

Azacitidine and homoharringtonine

Intervention Type DRUG

Concommitant use of azacitidine and homoharringtonine

Interventions

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Azacitidine and homoharringtonine

Concommitant use of azacitidine and homoharringtonine

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

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

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

Exclusion Criteria

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

2\. Any condition including the presence of laboratory abnormalities, which places the subject at unacceptable risk if participated in the study.

3\. Any condition that confounds the ability to interpret data from the study.
Minimum Eligible Age

1 Month

Maximum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Qilu Hospital of Shandong University

OTHER

Sponsor Role collaborator

Taian City Central Hospital

OTHER

Sponsor Role collaborator

The Affiliated Hospital of Qingdao University

OTHER

Sponsor Role collaborator

The Second Hospital of Hebei Medical University

OTHER

Sponsor Role collaborator

Children's Hospital of Hebei Province

OTHER

Sponsor Role collaborator

Air Force Military Medical University, China

OTHER

Sponsor Role lead

Responsible Party

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Lipeng Liu

Dr

Responsibility Role PRINCIPAL_INVESTIGATOR

Locations

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Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College

Tianjin, Tianjin Municipality, China

Site Status RECRUITING

Countries

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China

Central Contacts

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Wen-Yu Yang, M.D.

Role: CONTACT

+86-22-23909138

Facility Contacts

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Xiao-Fan Zhu, M.D.

Role: primary

+86-22-23909001

Other Identifiers

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JMML-CAMS-2020

Identifier Type: -

Identifier Source: org_study_id

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