Azacitidine and Homoharringtonine in JMML

Not Applicable

Recruiting

• Conditions: JMML
• Interventions: Drug: Azacitidine and homoharringtonine

• Registration Number: NCT04505995
• Lead Sponsor: Air Force Military Medical University, China

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.

Detailed Description

Not available

Study Timeline

• Study Start: 2020-01-01
• Status Verified: 2020-08
• Study First Submitted: 2020-08-06
• Study First Submitted QC: 2020-08-06
• Last Update Submitted: 2020-08-06
• Study First Posted: 2020-08-10
• Last Update Posted: 2020-08-10
• Primary Completion: 2023-01-01
• Study Completion: 2025-01-01

Recruitment & Eligibility

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

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Intervention group	Azacitidine and homoharringtonine	-

Primary Outcome Measures

Name	Time	Method
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).

Trial Locations

Locations (1)

Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College; 🇨🇳 Tianjin, Tianjin, China