Azacitidine and Homoharringtonine in JMML

JMML Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04505995
• Other study ID #: JMML-CAMS-2020
• Status: Recruiting
• Phase: N/A
• Start date: January 1, 2020
• Est. completion date: January 1, 2025

Study information

• Verified date: August 2020
• Source: Fourth Military Medical University
• Contact: Wen-Yu Yang, M.D.
• Phone: +86-22-23909138
• Email: yangwenyu[@]ihcams.ac.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 30
• Est. completion date: January 1, 2025
• Est. primary completion date: January 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Month to 18 Years

Eligibility

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.

Related Conditions & MeSH terms

• JMML

Intervention

Drug:
• Azacitidine and homoharringtonine
Concommitant use of azacitidine and homoharringtonine

Locations

Country	Name	City	State
China	Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College	Tianjin	Tianjin

Sponsors (6)

Lead Sponsor	Collaborator
Fourth Military Medical University	Children's Hospital of Hebei Province, Qilu Hospital of Shandong University, Taian City Central Hospital, The Affiliated Hospital of Qingdao University, The Second Hospital of Hebei Medical University

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 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).	Up to 4 Months