Phase 2
N=15
Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)
Juvenile Myelomonocytic Leukemia
Bottom Line
View on ClinicalTrials.gov: NCT00167219 ↗Enrolled (actual)
15
Serious AEs
33.3%
Results posted
Nov 2023
Primary outcome: Primary: Determine Probability of Long-term Disease Free Survival in JMML — 67 Percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Stem Cell Transplant (Biological); Preparative Regimen (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Jul 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Determine Probability of Long-term Disease Free Survival in JMML |
67 | — |
| SECONDARY Combined Incidence of Neutrophil Engraftment, Graft-versus-host Disease (GVHD), Regimen-related Toxicity, and Relapse. |
33 | — |
Summary
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).
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT00167219). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.