Stem Cell Transplant for Hemoglobinopathy

PHASE2/PHASE3CompletedINTERVENTIONAL
Enrollment

22

Participants

Timeline

Start Date

June 30, 2002

Primary Completion Date

March 31, 2014

Study Completion Date

January 31, 2020

Conditions
Sickle Cell DiseaseThalassemiaSevere Congenital NeutropeniaDiamond-Blackfan AnemiaShwachman-Diamond Syndrome
Interventions
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

Trial Locations (1)

55455

Masonic Cancer Center, University of Minnesota, Minneapolis

All Listed Sponsors
collaborator

National Marrow Donor Program

OTHER

lead

Masonic Cancer Center, University of Minnesota

OTHER

NCT00176852 - Stem Cell Transplant for Hemoglobinopathy | Biotech Hunter | Biotech Hunter