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Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders

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ClinicalTrials.gov Identifier: NCT01043640
Recruitment Status : Completed
First Posted : January 7, 2010
Results First Posted : May 12, 2017
Last Update Posted : February 5, 2018
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota

Study Type Interventional
Study Design Intervention Model: Single Group Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Conditions Mucopolysaccharidosis
Hurler Syndrome
Hunter Syndrome
Maroteaux-Lamy Syndrome
Sly Syndrome
Alpha Mannosidosis
Fucosidosis
Aspartylglucosaminuria
Adrenoleukodystrophy (ALD)
Krabbe Disease
Metachromatic Leukodystrophy (MLD)
Sphingolipidoses
Peroxisomal Disorders
Interventions Drug: Campath-1H
Drug: Cyclophosphamide
Drug: Busulfan
Procedure: Allogeneic stem cell transplantation
Drug: Cyclosporine A
Drug: Mycophenolate Mofetil
Enrollment 46
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Transplant Patients
Hide Arm/Group Description

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Period Title: Overall Study
Started 46
Completed 46
Not Completed 0
Arm/Group Title Transplant Patients
Hide Arm/Group Description

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Baseline Participants 46
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 46 participants
<=18 years
46
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 46 participants
Female
11
  23.9%
Male
35
  76.1%
1.Primary Outcome
Title Number of Patients With Donor Derived Engraftment
Hide Description Donor derived engraftment is defined as 80 percent or greater donor cells in the recipient's bone marrow and blood cells.
Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
42
  91.3%
2.Secondary Outcome
Title Number of Patients With Grade 0 Graft-Versus-Host Disease (GVHD)
Hide Description

GVHD grading is performed using modified Glucksberg criteria and is as follows:

grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4

Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
32
  69.6%
3.Secondary Outcome
Title Number of Patients With Grade 1 Graft-Versus-Host Disease (GVHD)
Hide Description

GVHD grading is performed using modified Glucksberg criteria and is as follows:

grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4

Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
2
   4.3%
4.Secondary Outcome
Title Number of Patients With Grade 2 Graft-Versus-Host Disease (GVHD)
Hide Description

GVHD grading is performed using modified Glucksberg criteria and is as follows:

grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4

Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
5
  10.9%
5.Secondary Outcome
Title Number of Patients With Grade 3 Graft-Versus-Host Disease (GVHD)
Hide Description

GVHD grading is performed using modified Glucksberg criteria and is as follows:

grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4

Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
2
   4.3%
6.Secondary Outcome
Title Number of Patients With Grade 4 Graft-Versus-Host Disease (GVHD)
Hide Description

GVHD grading is performed using modified Glucksberg criteria and is as follows:

grade 0: absence of any skin, liver and/or gastrointestinal (GI) involvement grade 1: skin stage 1 or 2 only grade 2: skin stage 3 or liver stage 1 or lower GI stage 1 or upper GI involvement grade 3: skin stage 0 - 3 plus liver stage 2-4 or lower GI stage 2-3 grade 4: skin stage 4 or lower GI stage 4

Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
5
  10.9%
7.Secondary Outcome
Title Number of Patients Who Died Peri-Transplant
Hide Description Peri-transplant is defined as within 100 days of transplant.
Time Frame By Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Measure Type: Count of Participants
Unit of Measure: Participants
2
   4.3%
8.Secondary Outcome
Title Donor Cell Chimerism Following Transplant
Hide Description Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Time Frame Day 28
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: percentage of donor cells
95  (9)
9.Secondary Outcome
Title Donor Cell Chimerism Following Transplant
Hide Description Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Time Frame Day 42
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: percentage of donor cells
93  (15)
10.Secondary Outcome
Title Donor Cell Chimerism Following Transplant
Hide Description Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Time Frame Day 100
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: percentage of donor cells
90  (26)
11.Secondary Outcome
Title Donor Cell Chimerism Following Transplant
Hide Description Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Time Frame 6 months
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: percentage of donor cells
94  (22)
12.Secondary Outcome
Title Donor Cell Chimerism Following Transplant
Hide Description Donor cell chimerism is defined as the percentage of bone marrow and blood cells in the recipient that are of donor origin.
Time Frame One year
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Transplant Patients
Hide Arm/Group Description:

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

Overall Number of Participants Analyzed 46
Mean (Standard Deviation)
Unit of Measure: percentage of donor cells
99  (4)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Transplant Patients
Hide Arm/Group Description

Includes patients who received allogeneic stem cell transplantation following treatment plan of Campath-1H, cyclophosphamide, cyclosporine A, mycophenolate mofetil, and busulfan.

Campath-1H: Days -21, -20 and -19, 0.3 mg/kg SQ or IV Cyclophosphamide: Days -10 through -6, 50 mg/kg/day IV with Mesna Busulfan: Days -5 through Day -2, 1.1 mg/kg/dose IV if ≤ 12 kg; 0.8 mg/kg/dose IV if > 12 kg Allogeneic stem cell transplantation: > 24 hours after last dose of busulfan Cyclosporine A: 2.5 mg/kg/dose IV beginning on day –3. Dosing will be 3 times daily if body weight is ≤ 40 kg and 2 times daily if body weight is > 40 kg

Mycophenolate Mofetil: 15 mg/kg/dose (max dose of 1gram) IV three times a day beginning on Day -3 at a dose based on body weight:

Stop MMF at Day +42 or 7 days after engraftment achieved (ANC>500 x 10^6 neutrophils/L x 3 days and chimerism >90%), whichever is later.

All-Cause Mortality
Transplant Patients
Affected / at Risk (%)
Total   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Transplant Patients
Affected / at Risk (%)
Total   8/46 (17.39%) 
Cardiac disorders   
Pericardial Effusion  2/46 (4.35%) 
Cardiac Arrest  1/46 (2.17%) 
Gastrointestinal disorders   
Diarrhea  2/46 (4.35%) 
Nervous system disorders   
Intracerebral Hemorrhage  1/46 (2.17%) 
Renal and urinary disorders   
Renal Failure  1/46 (2.17%) 
Respiratory, thoracic and mediastinal disorders   
Respiratory Failure  1/46 (2.17%) 
Pulmonary Veno-Occlusive Disease  1/46 (2.17%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Transplant Patients
Affected / at Risk (%)
Total   40/46 (86.96%) 
Cardiac disorders   
Pericardial Effusion  7/46 (15.22%) 
Tachycardia  3/46 (6.52%) 
Gastrointestinal disorders   
Diarrhea  4/46 (8.70%) 
Hemorrhage, Gastrointestinal  8/46 (17.39%) 
Mucositis  11/46 (23.91%) 
Nausea  6/46 (13.04%) 
Vomiting  4/46 (8.70%) 
General disorders   
Fever  11/46 (23.91%) 
Graft Versus Host Disease  9/46 (19.57%) 
Infections and infestations   
Infection, NOS  35/46 (76.09%) 
Infection, Respiratory  9/46 (19.57%) 
Infection, Gastrointestinal  7/46 (15.22%) 
Investigations   
Bilirubin Increased  16/46 (34.78%) 
Metabolism and nutrition disorders   
Anorexia  7/46 (15.22%) 
Nervous system disorders   
Headache  3/46 (6.52%) 
Renal and urinary disorders   
Hemorrhage, Genitourinary  10/46 (21.74%) 
Respiratory, thoracic and mediastinal disorders   
Dyspnea  14/46 (30.43%) 
Hypoxia  14/46 (30.43%) 
Skin and subcutaneous tissue disorders   
Hives  3/46 (6.52%) 
Rash  10/46 (21.74%) 
Vascular disorders   
Hypertension  21/46 (45.65%) 
Hypotension  3/46 (6.52%) 
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
Name/Title: Dr. Paul Orchard
Organization: Masonic Cancer Center, University of Minnesota
Phone: 612-626-2313
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT01043640     History of Changes
Other Study ID Numbers: 2009LS088
MT2009-19 ( Other Identifier: Blood and Marrow Transplantation Program )
First Submitted: January 5, 2010
First Posted: January 7, 2010
Results First Submitted: March 31, 2017
Results First Posted: May 12, 2017
Last Update Posted: February 5, 2018