Azacitidine and Homoharringtonine in JMML

NCT04505995 | Unknown

• 1 other identifier: JMML-CAMS-2020
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

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.

Conditions

JMML

Outcome Measures

Primary Outcomes (1)

• 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

Study Arms (1)

Intervention group

EXPERIMENTAL

Drug: Azacitidine and homoharringtonine

Interventions

Azacitidine and homoharringtonine DRUG

Concommitant use of azacitidine and homoharringtonine

Intervention group

Eligibility Criteria

• Age: 1 Month - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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.

You may not qualify if:

• \. 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.

Sponsors & Collaborators

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

Study Sites (1)

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

Tianjin, Tianjin Municipality, 300020, China

RECRUITING

MeSH Terms

Interventions

Azacitidine; Homoharringtonine

Intervention Hierarchy (Ancestors)

Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides; Harringtonines; Alkaloids; Benzazepines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring; Heterocyclic Compounds, 4 or More Rings

Central Study Contacts

Wen-Yu Yang, M.D.

CONTACT

+86-22-23909138; yangwenyu@ihcams.ac.cn

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Dr

Study Record Dates

• First Submitted: August 6, 2020
• First Posted: August 10, 2020
• Study Start: January 1, 2020
• Primary Completion: January 1, 2023
• Study Completion: January 1, 2025
• Last Updated: August 10, 2020

Record last verified: 2020-08

Locations

CN (1)