Clinical trial · Interventional
Azacitidine and Homoharringtonine in JMML
Efficacy of Concomitant Use of Azacitidine and Homoharringtonine in Children With Juvenile Myelomonocytic Leukemia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| JMML | Juvenile Myelomonocytic Leukemia | ALIAS | 0.90 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Azacitidine and homoharringtonine | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Intervention group
- interventionNames
- Drug: Azacitidine and homoharringtonine
Primary outcomes (1)
- measure
- Juvenile Myelomonocytic Leukemia (JMML) response rate at end of 4 Months
- timeFrame
- Up to 4 Months
- description
- 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).
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 1 Month
- Maximum age
- 18 Years
Show eligibility criteria text
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.
References
Publications (0)
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