Multicenter, open-label, uncontrolled study of azacitidine therapy for juvenile myelomonocytic leukemia

Phase 2

Recruiting

• Conditions: Juvenile myelomonocytic leukemia; JMML; JMML, DNA methylation, Azacitidine; Leukemia, Myelomonocytic, Juvenile

• Registration Number: JPRN-jRCTs041210044
• Lead Sponsor: Muramatsu Hideki

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-07-26
• Last Update Posted: 17 October 2023

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

(1) Cases with a clinical diagnosis of JMML based on the JMML diagnostic criteria.
(2) Have submitted or are able to submit samples for central testing.
(3) Consent for CHM-14 was obtained prior to obtaining consent to participate in this study.
(4) Age at diagnosis is less than 10 years old.
(5) The patient has sufficient organ function to meet the following three criteria at the same time. Laboratory values within 7 days before the date of enrollment.
a) D-Bil level: less than 1.5 mg/dL
b) Creatinine: less than 3 times the upper limit of the age-specific test reference value
c) Left ventricular ejection fraction (EF) >=50% by echocardiography
(6) Adequate explanations and written consent have been obtained from the proxy.

Exclusion Criteria

(1) Noonan syndrome (a case of PTPN11 germ cell mutation)
(2) Associated with difficult-to-control infections (including active tuberculosis and HIV antibody-positive cases).
(3) Relapse cases with a history of hematopoietic cell transplantation.
(4) Any other case deemed inappropriate by the doctor in charge.

Study & Design

• Study Type: Interventional
• Study Design: Not specified