Clinical trial · Interventional
Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia
NCT00167219CI-TRIAL-00071083completedPhase 1 / Phase 2Results postedClinicalTrials.gov clinicaltrialsProvenance
- 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)
The investigators hypothesize that long-term disease-free survival (DFS) in patients with JMML can be achieved with a treatment of busulfan (BU), cyclophosphamide (CY) and melphalan (L-PAM) followed by hematopoietic cell transplantation (HCT).
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 |
|---|---|---|---|
| Juvenile Myelomonocytic Leukemia | Juvenile Myelomonocytic Leukemia | ONTOLOGY_EXACT | 0.98 |
Interventions
Interventions (2)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Preparative Regimen | Drug | — | UNRESOLVED |
| Stem Cell Transplant | Biological | Hematopoietic Cell Transplantation | ALIAS |
Design
Arms and outcomes
Arms (1)
- type
- EXPERIMENTAL
- label
- Intent-to-Treat
- description
- Patients receiving study regimen.
- interventionNames
- Biological: Stem Cell Transplant
- Drug: Preparative Regimen
Primary outcomes (1)
- measure
- Determine Probability of Long-term Disease Free Survival in JMML
- timeFrame
- at 1 year after transplant
Secondary outcomes (1)
- measure
- Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse.
- timeFrame
- at 1 year after transplant
- description
- Measured by AGVHD Grades 2 - 4.
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria:
* Patients must have a diagnosis of JMML and fulfill these minimal criteria (International diagnostic criteria for JMML):
* Leukocytosis (\> 13,000) with absolute monocytosis (\> 1,000)
* The presence of immature myeloid cells in the peripheral blood
* Less than 30% marrow blasts
* Absence of t(9:22) or BCR-ABL transcript
* Adequate major organ function including:
* Cardiac: ejection fraction \> 45%
* Hepatic: no clinical evidence of hepatic failure (e.g. coagulopathy, ascites)
* Karnofsky performance status \> 70% or Lansky score \> 50%
* Creatinine must be \< 2 x normal for age
* Written informed consent.
Exclusion Criteria:
* Active uncontrolled infection within one week of HCT.References
Publications (0)
Data not yet available
No reference posted for this study.