NCT00176852

Stem Cell Transplant for Hemoglobinopathy

Allogeneic Hematopoietic Stem Cell Transplant for Patients With High Risk Hemoglobinopathy Using a Preparative Regimen to Achieve Stable Mixed Chimerism

• Status: Completed
• Phase: Phase 2 / Phase 3
• Study type: Interventional
• Enrollment: 22 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: 50 Years
• Healthy volunteers: Not accepted

Summary

This study tests the clinical outcomes of one of two preparative regimens (determined by available donor source) in patients with non-malignant hemoglobinopathies. The researchers hypothesize that these regimens will have a positive effect on post transplant engraftment and the incidence of graft-versus-host-disease. Regimen A2 has replaced Regimen A in this study. Two patients were treated on Regimen A but did not have evidence of initial engraftment thus triggering the stopping rule for that arm of this study.

Detailed description

Prior to transplantation, subjects will receive either: Cyclophosphamide, Fludarabine, Campath, Total body irradiation (TBI) Or Busulfan, Cyclophosphamide, antithymocyte globulin (ATG), granulocyte colony-stimulating factor (GSCF) These drugs (and the radiation) are being given 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 cyclosporine-A and mycophenolate (MMF)/or Methylprednisone/or Methotrexate to reduce the risk of graft-versus-host disease, the complication that occurs when the donor's stem cells react against the patient.

Conditions

• Sickle Cell Disease
• Thalassemia
• Severe Congenital Neutropenia
• Diamond-Blackfan Anemia
• Shwachman-Diamond Syndrome

Interventions

Type	Name	Description
DRUG	Busulfan, Fludarabine, ATG, TLI	Busulfan 0.8 mg/kg/dose intravenous (IV) Days -8 and -7 Fludarabine 35 mg/m2 IV Days -6 through -2 Antithymocyte globulin (ATG) 30 mg/kg IV Days -2 and -1 Total lymphoid radiation 300 cGy
DRUG	Busulfan, Cyclophosphamide, ATG, GCSF	Busulfan 0.8 mg/kg/dose intravenous (IV) Days -9 through -6 Cyclophosphamide 50 mg/kg IV Days -5 through -2 ATG 30 mg/kg IV Day -1 GCSF 5 mcg/kg/day IV until ANC \>2500 x 2 days.
DRUG	Campath, Fludarabine, Cyclophosphamide	Receives Campath-1H 0.2 mg/kg Days -10 through -6, Fludarabine 35 mg/m2 intravenous (IV) Days -6 through -2, total body irradiation (TBI) 300 cGy Day -1.
RADIATION	Total Body Irradiation	300 cGY Day -1
PROCEDURE	Stem cell infusion	Given Day 0

Timeline

• Start date: 2002-06-01
• Primary completion: 2014-03-01
• Completion: 2020-01-01
• First posted: 2005-09-15
• Last updated: 2020-02-27
• Results posted: 2017-05-09

Locations

1 site across 1 country: United States