NCT00176865

Stem Cell Transplant for Immunologic or Histiocytic Disorders

Allogeneic Hematopoietic Stem Cell Transplant for Patients With Immunologic or Histiocytic Disorders Using a Non-Myeloablative Preparative Regimen to Achieve Stable Mixed Chimerism

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 19 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 35 Years
• Healthy volunteers: Not accepted

Summary

This study tests the clinical outcomes of a preparative regimen of fludarabine (FLU), anti-thymocyte globulin (ATG)/or Campath, and melphalan; followed by hematopoietic stem cell transplant, and a post transplant regimen of Cyclosporin A (CsA) in patients with immunologic or histiocytic disorders. The researchers hypothesize that this regimen will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease (GVHD). Patients will be randomized biologically into one of 3 arms based upon donor availability: (a) human leukocyte antigen (HLA) genotypic matched sibling donor, (b) HLA phenotypic matched unrelated peripheral blood stem cell (PBSC) donor, (c) two HLA 0-2 antigen mismatched unrelated cord blood donors (double cord).

Detailed description

Prior to transplantation, subjects will receive Melphalan, Fludarabine and Anti-Thymocyte Globulin (ATG) or Campath. These three drugs are being given to subjects to help the new stem cells take and grow. On the day of transplantation, subjects will receive stem cells transfused via intravenous (IV) catheter. After stem cell transplantation, subjects will be given Cyclosporin A (CsA) and mycophenolate mofetil (MMF) to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.

Conditions

• Hemophagocytic Lymphohistiocytosis
• X-Linked Lymphoproliferative Disorders
• Chediak-Higashi Syndrome
• Griscelli Syndrome
• Immunologic Deficiency Syndromes
• Langerhans-Cell Histiocytosis

Interventions

Type	Name	Description
PROCEDURE	Stem Cell Transplant	IV on Day 0
DRUG	Fludarabine	30mg/m\^2 IV Day -7 through Day -3
DRUG	Melphalan	140 mg/m\^2 IV Day -1
DRUG	Anti-thymocyte globulin (ATG)	30 mg/kg IV Day -5 through Day -1
DRUG	Campath 1H	0.2 mg/kg IV X 5 days (used as an alternative to Anti-thymocyte globulin (ATG) if unable to tolerate ATG) Day -10 through Day -6
DRUG	Cyclosporin A	2.5 mg/kg IV every 12 hours (adults) or every 8 hours (children \<40 kg) maintaining a level of \>200mg/L Day -3 until Day +180 when, if no GVHD, the dose will be tapered 10% per week beginning on day 181
DRUG	Mycophenolate mofetil	15 mg/kg IV or orally bid and discontinued on Day +45 unless GVHD is present
DRUG	Intravenous immunoglobulin (IVIG)	500 mg/kg IV weekly beginning on Day +7 until Day +100

Timeline

• Start date: 2002-08-01
• Primary completion: 2012-08-01
• Completion: 2014-08-01
• First posted: 2005-09-15
• Last updated: 2017-12-28
• Results posted: 2017-04-14

Locations

1 site across 1 country: United States