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Stem Cell Transplant for Hemoglobinopathy

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT00176852
Recruitment Status : Completed
First Posted : September 15, 2005
Results First Posted : May 9, 2017
Last Update Posted : February 27, 2020
Sponsor:
Collaborator:
National Marrow Donor Program
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type Interventional
Study Design Allocation: Non-Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Sickle Cell Disease
Thalassemia
Severe Congenital Neutropenia
Diamond-Blackfan Anemia
Shwachman-Diamond Syndrome
Interventions Drug: Busulfan, Fludarabine, ATG, TLI
Drug: Busulfan, Cyclophosphamide, ATG, GCSF
Drug: Campath, Fludarabine, Cyclophosphamide
Radiation: Total Body Irradiation
Procedure: Stem cell infusion
Enrollment 22
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2)
Hide Arm/Group Description

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Period Title: Overall Study
Started 3 5 14
Completed 3 5 14
Not Completed 0 0 0
Arm/Group Title Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2) Total
Hide Arm/Group Description

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

 Total of all reporting groups
Overall Number of Baseline Participants 3 5 14 22
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 5 participants 14 participants 22 participants
<=18 years
3
 100.0%
5
 100.0%
12
  85.7%
20
  90.9%
Between 18 and 65 years
0
   0.0%
0
   0.0%
2
  14.3%
2
   9.1%
>=65 years
0
   0.0%
0
   0.0%
0
   0.0%
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 3 participants 5 participants 14 participants 22 participants
Female
2
  66.7%
1
  20.0%
8
  57.1%
11
  50.0%
Male
1
  33.3%
4
  80.0%
6
  42.9%
11
  50.0%
1.Primary Outcome
Title Number of Patients Who Experienced Grade 3-5 Treatment Related Toxicity
Hide Description In general, grade 3 equates to moderate, grade 4 to severe and grade 5 to death.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
2
  14.3%
2.Secondary Outcome
Title The Incidence of Chimerism at 100 Days
Hide Description The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 100 days.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 2 5 13
Measure Type: Count of Participants
Unit of Measure: Participants
1
  50.0%
5
 100.0%
11
  84.6%
3.Secondary Outcome
Title The Incidence of Chimerism at 6 Months
Hide Description The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 6 months.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 2 5 13
Measure Type: Count of Participants
Unit of Measure: Participants
1
  50.0%
5
 100.0%
8
  61.5%
4.Secondary Outcome
Title The Incidence of Chimerism at 1 Year
Hide Description The number of patients whose blood and/or bone marrow contains > 10% donor cells.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
One of the 3 patients treated on Arm A was retreated at Day 40 and was not evaluable. One of the 14 patients on Arm A2 died before 1 year.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 2 5 13
Measure Type: Count of Participants
Unit of Measure: Participants
1
  50.0%
5
 100.0%
8
  61.5%
5.Secondary Outcome
Title The Incidence of Grade 2-4 Acute Graft Versus Host Disease (Acute GVHD)
Hide Description The number of patients who experienced grades 2-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Grades 2-4 equate to mild to severe disease. Symptoms typically appear within weeks after transplant.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
6.Secondary Outcome
Title The Incidence of Grade 3-4 Acute Graft Versus Host Disease (Acute GVHD)
Hide Description The number of patients who experienced grades 3-4 Acute GVHD. Acute GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. IGrades 3-4 equate to moderate to severe disease. Symptoms typically appear within weeks after transplant.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
7.Secondary Outcome
Title The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
Hide Description The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
8.Secondary Outcome
Title The Incidence of Chronic Graft Versus Host Disease (Chronic GVHD)
Hide Description The number of patients who experienced Chronic GVHD. Chronic GVHD is when the donated bone marrow or peripheral blood stem cells view the recipient's body as foreign, and the donated cells/bone marrow attack the body. Chronic GVHD can appear at any time after allogeneic transplant or several years after transplant.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
0
   0.0%
9.Secondary Outcome
Title Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Hide Description The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Time Frame pre-transplant
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Median (Full Range)
Unit of Measure: units on a scale
100
(100 to 100)
98
(90 to 100)
99
(90 to 100)
10.Secondary Outcome
Title Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Hide Description The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
Two of the 14 patients treated on Arm A2 died before 1 year.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 12
Median (Full Range)
Unit of Measure: units on a scale
97
(90 to 100)
100
(100 to 100)
100
(80 to 100)
11.Secondary Outcome
Title Change in the Patient's Quality of Life as Compared to the Pre-Transplant Assessment
Hide Description The measure for quality of life used in this study is the Karnofsky Performance Score. The Karnofsky Performance Score runs from 100 to 0, where 100 is "perfect" health and 0 is death.
Time Frame 2 years
Hide Outcome Measure Data
Hide Analysis Population Description
Two of the 14 patients treated on Arm A2 died before 2 years and 2 failed their 2 year clinic appointment.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 10
Median (Full Range)
Unit of Measure: units on a scale
100
(90 to 100)
100
(100 to 100)
100
(90 to 100)
12.Secondary Outcome
Title Determine Physical Characteristics and Biologic Effects of Mixed Populations of Donor and Host Red Blood Cells
Hide Description [Not Specified]
Time Frame During study
Hide Outcome Measure Data
Hide Analysis Population Description
data were not collected
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
13.Secondary Outcome
Title Determine the Concentration of Campath in the Serum
Hide Description [Not Specified]
Time Frame Day 0
Hide Outcome Measure Data
Hide Analysis Population Description
The Principal Investigator removed this as a study objective and therefore Campath concentrations were not collected.
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 0 0 0
No data displayed because Outcome Measure has zero total analyzed.
14.Secondary Outcome
Title Overall Survival
Hide Description Number of patients alive 100 days after transplant.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
5
 100.0%
12
  85.7%
15.Secondary Outcome
Title Overall Survival
Hide Description Number of patients alive 1 year after transplant.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
5
 100.0%
12
  85.7%
16.Secondary Outcome
Title Disease Free Survival
Hide Description Number of patients alive without disease 100 days after transplant.
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
5
 100.0%
11
  78.6%
17.Secondary Outcome
Title Disease Free Survival
Hide Description Number of patients alive without disease 1 year after transplant.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title RIC Bu/Flu (A) (Discontinued) MA Bu/Cy (B) RIC Cy/Flu/TBI (A2)
Hide Arm/Group Description:

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
Overall Number of Participants Analyzed 3 5 14
Measure Type: Count of Participants
Unit of Measure: Participants
3
 100.0%
5
 100.0%
11
  78.6%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2)
Hide Arm/Group Description

Full Preparative Regimen for subjects with matched donors using Busulfan on Day -8 and -7, Fludarabine on Day -6 through -2, antithymocyte globulin (ATG) on Day -2 through -1, total lymphoid radiation (TLI) on Day -1, stem cell infusion on Day 0.

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

Myeloablative Preparative Regimen for subjects with HLA identical sibling donors consists of Busulfan on day -9 through -6, Cyclophosphamide on day -5 through -2, ATG on day -3 through -1, stem cell infusion on Day 0 and Granulocyte Colony Stimulating Factor on day -3 until ANC >2500 x 2 days.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0

Patients with sickle cell disease or thalassemia who do not have an HLA-identical sibling donor or who has pre-existing organ dysfunction making myeloablative condition ineligible will receive Campath on day -10 through -6, Cyclophosphamide on day -7, Fludarabine on day -6 through -2, total body irradiation (TBI) on day -1, stem cell infusion on Day 0.

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.

Total Body Irradiation: 300 cGY Day -1

Stem cell infusion: Given Day 0
All-Cause Mortality
Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/--   --/-- 
Hide Serious Adverse Events
Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   2/3 (66.67%)   0/5 (0.00%)   0/14 (0.00%) 
General disorders       
Primary Graft Failure  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Secondary Graft Failure  2/3 (66.67%)  0/5 (0.00%)  0/14 (0.00%) 
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Full Conditioning (Discontinued / A) Busulfan Conditioning (B) Campath and TBI Conditioning (A2)
Affected / at Risk (%) Affected / at Risk (%) Affected / at Risk (%)
Total   3/3 (100.00%)   5/5 (100.00%)   13/14 (92.86%) 
Blood and lymphatic system disorders       
Polycythemia  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Polymorphic Post-Transplant Lymphoproliferative Disorder  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Cardiac disorders       
Pericardial Effusion  0/3 (0.00%)  3/5 (60.00%)  3/14 (21.43%) 
Intraventricular Septal Hypertrophy  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Endocrine disorders       
Hypothyroidism  3/3 (100.00%)  1/5 (20.00%)  1/14 (7.14%) 
Hypogonadism  1/3 (33.33%)  0/5 (0.00%)  1/14 (7.14%) 
Eye disorders       
Conjunctival Hemorrhage  0/3 (0.00%)  0/5 (0.00%)  2/14 (14.29%) 
Gastrointestinal disorders       
Esophageal Varices  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Hepatobiliary disorders       
Veno-Occlusive Disease  0/3 (0.00%)  0/5 (0.00%)  2/14 (14.29%) 
Immune system disorders       
Engraftment Syndrome  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Infections and infestations       
Bacterial Infection, Blood  2/3 (66.67%)  2/5 (40.00%)  5/14 (35.71%) 
Bacterial Infection, Pulmonary  0/3 (0.00%)  1/5 (20.00%)  2/14 (14.29%) 
Fungal Infection, Gastrointestinal  2/3 (66.67%)  1/5 (20.00%)  1/14 (7.14%) 
Pneumonia  1/3 (33.33%)  3/5 (60.00%)  5/14 (35.71%) 
Viral Infection, Blood  1/3 (33.33%)  0/5 (0.00%)  5/14 (35.71%) 
Viral Infection, Pulmonary  0/3 (0.00%)  2/5 (40.00%)  6/14 (42.86%) 
Fungal Infection, Blood  1/3 (33.33%)  0/5 (0.00%)  1/14 (7.14%) 
Fungal Infection, Genitourinary  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Fungal Infection, Pulmonary  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Otitis Media  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Paronychia of Bilateral Great Toes  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Sepsis  0/3 (0.00%)  1/5 (20.00%)  0/14 (0.00%) 
Viral Infection, Gastrointestinal  0/3 (0.00%)  2/5 (40.00%)  6/14 (42.86%) 
Investigations       
Elevated Liver Function Tests  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Hepatitis  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Metabolism and nutrition disorders       
Hemochromatosis  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Musculoskeletal and connective tissue disorders       
Delayed Growth/Stature  3/3 (100.00%)  2/5 (40.00%)  0/14 (0.00%) 
Joint Dysfunction  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)       
Second Malignancy  0/3 (0.00%)  0/5 (0.00%)  2/14 (14.29%) 
Nervous system disorders       
Seizure  1/3 (33.33%)  0/5 (0.00%)  2/14 (14.29%) 
Neuro Toxicity, NOS  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Neuropathy  0/3 (0.00%)  1/5 (20.00%)  1/14 (7.14%) 
Subarachnoid Hemorrhage  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
Renal and urinary disorders       
Renal Failure  0/3 (0.00%)  0/5 (0.00%)  2/14 (14.29%) 
Cystitis  1/3 (33.33%)  1/5 (20.00%)  1/14 (7.14%) 
Reproductive system and breast disorders       
Upper Respiratory Stridor  1/3 (33.33%)  0/5 (0.00%)  0/14 (0.00%) 
Respiratory, thoracic and mediastinal disorders       
Respiratory Failure  0/3 (0.00%)  0/5 (0.00%)  2/14 (14.29%) 
Vascular disorders       
Hypertension  1/3 (33.33%)  1/5 (20.00%)  8/14 (57.14%) 
Thrombus, Right Atrium  0/3 (0.00%)  0/5 (0.00%)  1/14 (7.14%) 
[Not Specified]
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
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Name/Title: Dr. Angela Smith
Organization: Masonic Cancer Center, University of Minnesota
Phone: 612-626-2778
EMail: smith719@umn.edu
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Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00176852    
Obsolete Identifiers: NCT00005897
Other Study ID Numbers: MT2002-07
0206M26241 ( Other Identifier: IRB, University of Minnesota )
First Submitted: September 12, 2005
First Posted: September 15, 2005
Results First Submitted: March 28, 2017
Results First Posted: May 9, 2017
Last Update Posted: February 27, 2020