Evaluation of Fludarabine, Busulfan and Alemtuzumab as a Reduced Toxicity Ablative Bone Marrow Stem Cell Transplant Regimen for Children With Stem Cell Defects, Marrow Failure Syndromes, or Myelodysplastic Syndrome (MDS)/Leukemia

NCT00301834 Phase 2 COMPLETED Results posted

RATIONALE: Monoclonal antibodies, such as alemtuzumab, can block cancer growth in different ways. Some block the ability of cancer cells to grow and spread. Others find cancer cells and help kill them or carry cancer-killing substances to them. Drugs used in chemotherapy, such as fludarabine and busulfan, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing. A peripheral stem cell, bone marrow , or umbilical cord blood transplant may be able to replace blood-forming cells that were destroyed by chemotherapy. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine together with methotrexate and methylprednisolone may stop this from happening. PURPOSE: This phase II trial is studying how well giving alemtuzumab together with fludarabine and busulfan works when given before donor stem cell transplant in treating young patients with hematologic disorders.

Details

Lead sponsor	University of California, San Francisco
Phase	Phase 2
Status	COMPLETED
Enrolment	35
Start date	2005-01
Completion	2011-09

Conditions

• Congenital Amegakaryocytic Thrombocytopenia
• Diamond-blackfan Anemia
• Leukemia
• Myelodysplastic Syndromes
• Severe Congenital Neutropenia

Interventions

• alemtuzumab
• busulfan
• cyclosporine
• fludarabine phosphate
• methotrexate
• methylprednisolone
• allogeneic bone marrow transplantation
• allogeneic hematopoietic stem cell transplantation
• peripheral blood stem cell transplantation
• umbilical cord blood transplantation

Primary outcomes

• Number of Participants Achieving Durable Engraftment (Presence of Donor Cells) at 6 Weeks Post Transplantation — 6 weeks post-transplant
  Peripheral blood chimerism studies were performed by quantitative real time polymerase chain reaction (qPCR) evaluation of differential short tandem repeat DNA sequences

Countries

United States