NCT05796570

A Pilot Study to Evaluate the Feasibility of Post-Hematopoietic Stem Cell Transplant Prophylaxis With Decitabine Combined With Filgrastim for Children and Young Adults With AML, MDS and Related Myeloid Malignancies

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 37 (estimated)
• Sponsor: Franziska Wachter · Academic / Other
• Sex: All
• Age: 1 Year – 39 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to examine if it is feasible to administer decitabine and filgrastim after allogenic hematopoietic stem cell transplant (HCT) in children and young adults with myelodysplastic syndrome, acute myeloid leukemia and related myeloid disorders, and if the treatment is effective in preventing relapse after HCT. The names of the study drugs involved in this study are: * Decitabine (a nucleoside metabolic inhibitor) * Filgrastim (a recombinant granulocyte colony-stimulating factor (G-CSF)

Detailed description

This is a single arm, pilot study to determine if sequential cycles of maintenance therapy with decitabine and filgrastim post allogenic hematopoietic cell transplant (HCT) are feasible and effective in preventing relapse after HCT in pediatric and young adult patients with acute myeloid leukemia (AML), myelodysplastic syndrome (MDS) and treatment related myeloid malignancies (tAML/MDS) with either idiopathic disease or underlying germline disorders, including a subset that may be at higher risk for toxicity from treatment. Decitabine in combination with filgrastim after stem cell transplant has been shown to be effective in a large study that included mainly adult patients with acute myeloid leukemia. The research study procedures include screening for eligibility, study treatment including evaluations and follow up visits and blood tests. Bone marrow biopsies and aspirates will be performed as standard of care. Participants will receive study treatment for 6 months if tolerated and will be followed for 24 months after stem cell transplant. It is expected about 37 people will take part in this research study. This research study is supported by Dana-Farber Cancer Institute philanthropy and institutional grants from the Dana-Farber Cancer Institute and Boston Children's Hospital.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndromes
• Myeloid Malignancies
• MDS
• Inherited Bone Marrow Failure Syndrome
• Myeloid Neoplasm
• Aml

Interventions

Type	Name	Description
DRUG	Decitabine	Nucleoside metabolic inhibitor, via IV infusion.
DRUG	Filgrastim	Recombinant granulocyte colony-stimulating factor (G-CSF), via subcutaneous injection.

Timeline

• Start date: 2023-04-19
• Primary completion: 2026-09-01
• Completion: 2029-09-01
• First posted: 2023-04-03
• Last updated: 2026-03-19

Locations

2 sites across 1 country: United States

Regulatory

• FDA-regulated drug study