NCT00167219

Stem Cell Transplant for Juvenile Myelomonocytic Leukemia (JMML)

Hematopoietic Cell Transplantation in Children With Juvenile Myelomonocytic Leukemia

• Status: Completed
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 15 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Prior to transplantation, subjects will receive BUSULFAN via the central venous line, six times a day for four days, CYCLOPHOSPHAMIDE via the central venous line once a day for two days, and MELPHALAN via the central venous line for one day. Busulfan, cyclophosphamide, and melphalan are given to destroy the subject's leukemia. As well, these drugs will destroy the subject's own immune system to help ensure the new bone marrow takes and grows after transplantation. On the day of transplantation, bone marrow or umbilical cord blood from the donor will arrive to the bone marrow transplant unit and be transfused via venous line. These new cells will replace the subject's bone marrow.

Conditions

• Juvenile Myelomonocytic Leukemia

Interventions

Type	Name	Description
BIOLOGICAL	Stem Cell Transplant	Transplantation on Day 0.
DRUG	Preparative Regimen	* Busulfan * Cyclophosphamide * Mesna * Melphalan * Anti-thymocyte Globulin (ATG)

Timeline

• Start date: 1999-11-18
• Primary completion: 2022-07-12
• Completion: 2023-08-09
• First posted: 2005-09-14
• Last updated: 2023-11-07
• Results posted: 2023-11-07

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study