Risk-adapted treatment for children with juvenile myelomonocytic leukemia
Implementation of an interventional, risk-adapted clinical trial for children with newly diagnosed juvenile myelomonocytic leukemia.
This project tests tailoring treatment using DNA methylation and genetic tests so some children with newly diagnosed JMML may avoid transplant while others receive a drug combination (azacitidine plus trametinib) to improve outcomes.
Quick facts
| Grant type | R37 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | University of California, San Francisco NIH-funded |
| Lab location | 1 site (San Francisco, United States) |
| Project ID | NIH-11176942 on NIH RePORTER |
What this research studies
If your child is diagnosed with JMML, doctors would run DNA methylation and genetic tests to classify risk. Children with a low-risk pattern might be closely monitored and could avoid immediate stem cell transplant, while higher-risk children would be offered treatment with azacitidine plus trametinib and/or transplant as guided by the risk plan. The trial builds on prior signals that each drug can help and uses laboratory markers to decide who gets which approach. Your child would be followed over time for response, side effects, and longer-term outcomes.
Who could benefit from this research
Good fit: Children (typically infants and those up to about 11 years old) with a new diagnosis of juvenile myelomonocytic leukemia who can undergo molecular testing and receive care at participating treatment centers.
Not a fit: Children with other types of leukemia, those unable to travel to participating centers, or those whose disease lacks the specific molecular features targeted by the protocol may not benefit from this trial.
Why it matters
Potential benefit: If successful, this approach could spare low-risk children from toxic transplants and improve survival or disease control for high-risk children using a targeted drug combination.
How similar studies have performed: Early-phase trials showed azacitidine and trametinib each had promising activity in JMML and preclinical work suggests the combination is more effective, but the combination has not yet been proven in children in a risk-adapted trial.
Where this research is happening
San Francisco, United States
- University of California, San Francisco — San Francisco, United States (Active)
Researchers
- Principal investigator: Stieglitz, Elliot — University of California, San Francisco
- Study coordinator: Stieglitz, Elliot
About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page below.