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Trial record 1 of 14 for:    "Dyskeratosis Congenita"
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Stem Cell Transplant (SCT) for Dyskeratosis Congenita or SAA

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ClinicalTrials.gov Identifier: NCT00455312
Recruitment Status : Completed
First Posted : April 3, 2007
Results First Posted : May 31, 2017
Last Update Posted : December 5, 2017
Sponsor:
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 Dyskeratosis Congenita
Aplastic Anemia
Interventions Drug: Campath 1H
Drug: Cyclophosphamide
Drug: Fludarabine
Procedure: Total Body Irradiation
Procedure: Stem Cell Transplantation
Drug: antithymocyte globulin
Drug: Methylprednisolone
Enrollment 36
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin (ATG), total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Period Title: Overall Study
Started 15 21
Completed 15 20
Not Completed 0 1
Reason Not Completed
Death             0             1
Arm/Group Title Patients With DC Patients With SAA Total
Hide Arm/Group Description

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Total of all reporting groups
Overall Number of Baseline Participants 15 21 36
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 21 participants 36 participants
<=18 years
6
  40.0%
15
  71.4%
21
  58.3%
Between 18 and 65 years
9
  60.0%
5
  23.8%
14
  38.9%
>=65 years
0
   0.0%
1
   4.8%
1
   2.8%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 15 participants 21 participants 36 participants
Female
5
  33.3%
11
  52.4%
16
  44.4%
Male
10
  66.7%
10
  47.6%
20
  55.6%
1.Primary Outcome
Title Neutrophil Engraftment
Hide Description Defined as an absolute neutrophil count (ANC) >5 x 10^8/L (first of three consecutive laboratory measurements on different days) with at least 10% donor cells by day 100. Demonstrate sustained engraftment after a fludarabine based preparative regimen in patients with dyskeratosis congenita followed by hematopoietic cell transplantation.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 20
Measure Type: Count of Participants
Unit of Measure: Participants
15
 100.0%
20
 100.0%
2.Secondary Outcome
Title Incidence of Regimen Related Mortality at 100 Days
Hide Description all deaths without previous relapse or progression
Time Frame 100 days
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   6.7%
2
   9.5%
3.Secondary Outcome
Title Incidence of Chronic GVHD
Hide Description Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Time Frame 6 months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
4.Secondary Outcome
Title Incidence of Chronic GVHD
Hide Description Chronic Graft-Versus-Host Disease is a severe long-term complication created by infusion of donor cells into a foreign host.
Time Frame 1 year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   6.7%
2
   9.5%
5.Secondary Outcome
Title Incidence of Late Secondary Malignancies
Hide Description Defined as patients who have a secondary malignancy (cancer) occurring.
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
4
  19.0%
6.Secondary Outcome
Title Incidence of Grade 2-4 Acute Graft Versus Host Disease (GVHD)
Hide Description Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
1
   6.7%
0
   0.0%
7.Secondary Outcome
Title Incidence of Grade 3-4 Acute Graft Versus Host Disease (GVHD)
Hide Description Acute Graft-Versus-Host Disease is a severe short-term complication created by infusion of donor cells into a foreign host.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
0
   0.0%
0
   0.0%
8.Secondary Outcome
Title Overall Survival
Hide Description Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Time Frame Day 100
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
14
  93.3%
19
  90.5%
9.Secondary Outcome
Title Overall Survival
Hide Description Overall survival is defined as time from date of transplant to date of death or censored at the date of last documented contact for patients still alive.
Time Frame 1 Year
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
12
  80.0%
19
  90.5%
10.Secondary Outcome
Title Incidence of Pulmonary Complications
Hide Description Defined as patients who exhibit a pulmonary (lung) adverse event.
Time Frame 6 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description:

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

Overall Number of Participants Analyzed 15 21
Measure Type: Count of Participants
Unit of Measure: Participants
3
  20.0%
3
  14.3%
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Patients With DC Patients With SAA
Hide Arm/Group Description

Patients with dyskeratosis congenita (DC). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, total body irradiation and stem cell transplantation.

Campath 1H: 10, 9, 8, 7, and 6 days before transplant subjects will be given 1 dose of campath 1H given via catheter (0.2 mg/kg over 2 hours).

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

Patients with severe aplastic anemia (SAA). Patients are treated with alemtuzumab (Campath 1H), Cyclophosphamide, Fludarabine, antithymocyte globulin, total body irradiation and stem cell transplantation.

Cyclophosphamide: 7 days before the transplant, 1 dose of cyclophosphamide is given via catheter (50mg/kg IV over 2 hours).

Fludarabine: 6, 5, 4, 3, and 2 days before the transplant, 1 dose fludarabine is given via catheter (40 mg/kg IV over 1 hour)

Total Body Irradiation: 1 day before the transplant one dose (200 cGy) of total body irradiation is given

Stem Cell Transplantation: Infusion of stem cells on Day 0.

antithymocyte globulin: ATG (rabbit) 3 mg/kg for 3 days.

Methylprednisolone: 2mg/kg IV is given before each dose of ATG.

All-Cause Mortality
Patients With DC Patients With SAA
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Patients With DC Patients With SAA
Affected / at Risk (%) Affected / at Risk (%)
Total   2/15 (13.33%)   1/21 (4.76%) 
Nervous system disorders     
Intracranial Hemorrhage  1/15 (6.67%)  0/21 (0.00%) 
Renal and urinary disorders     
Renal Failure  1/15 (6.67%)  0/21 (0.00%) 
Respiratory, thoracic and mediastinal disorders     
Adult Respiratory Distress Syndrome  0/15 (0.00%)  1/21 (4.76%) 
Pulmonary Failure  1/15 (6.67%)  0/21 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Patients With DC Patients With SAA
Affected / at Risk (%) Affected / at Risk (%)
Total   15/15 (100.00%)   20/21 (95.24%) 
Blood and lymphatic system disorders     
Cytopenias  0/15 (0.00%)  1/21 (4.76%) 
Erythroid Aplasia  0/15 (0.00%)  1/21 (4.76%) 
Low Reticulocyte Count  0/15 (0.00%)  1/21 (4.76%) 
Post Transplant Lymphoproliferative Disease  0/15 (0.00%)  1/21 (4.76%) 
Cardiac disorders     
Atrial Fibrillation  0/15 (0.00%)  1/21 (4.76%) 
Congestive Heart Failure  1/15 (6.67%)  0/21 (0.00%) 
Pericardial Effusion  1/15 (6.67%)  1/21 (4.76%) 
Pulmonary Hypertension  0/15 (0.00%)  1/21 (4.76%) 
Ear and labyrinth disorders     
Otomastoiditis  0/15 (0.00%)  1/21 (4.76%) 
Endocrine disorders     
Hypogonadism  1/15 (6.67%)  0/21 (0.00%) 
Eye disorders     
Retinal Hemorrhage  1/15 (6.67%)  0/21 (0.00%) 
Gastrointestinal disorders     
Acute Pancreatitis  0/15 (0.00%)  1/21 (4.76%) 
GI Bleed  3/15 (20.00%)  2/21 (9.52%) 
Hemorrhagic Pancreatitis  0/15 (0.00%)  1/21 (4.76%) 
Nausea  0/15 (0.00%)  1/21 (4.76%) 
Vomiting  0/15 (0.00%)  1/21 (4.76%) 
Anorexia  0/15 (0.00%)  1/21 (4.76%) 
General disorders     
Early Small Bowel Obstruction vs. Ileus  1/15 (6.67%)  0/21 (0.00%) 
Fever  0/15 (0.00%)  1/21 (4.76%) 
Fever with Chills  0/15 (0.00%)  1/21 (4.76%) 
Multi Organ Failure  1/15 (6.67%)  0/21 (0.00%) 
Hepatobiliary disorders     
Gallstones  0/15 (0.00%)  1/21 (4.76%) 
Veno-Occlusive Disease  0/15 (0.00%)  1/21 (4.76%) 
Infections and infestations     
Blood Infection  10/15 (66.67%)  14/21 (66.67%) 
Bone Marrow Biopsy Site Infection  1/15 (6.67%)  0/21 (0.00%) 
Bone Marrow Infection  0/15 (0.00%)  1/21 (4.76%) 
Cellulitis  2/15 (13.33%)  2/21 (9.52%) 
Colitis  0/15 (0.00%)  1/21 (4.76%) 
Ear Infection  1/15 (6.67%)  0/21 (0.00%) 
Epstein Barr Virus Infection  0/15 (0.00%)  1/21 (4.76%) 
Eye Infection  1/15 (6.67%)  0/21 (0.00%) 
GI Infection  1/15 (6.67%)  4/21 (19.05%) 
Herpes Zoster  1/15 (6.67%)  1/21 (4.76%) 
Infection at Central Line Site  0/15 (0.00%)  1/21 (4.76%) 
Labia Infection  1/15 (6.67%)  0/21 (0.00%) 
Intra-Abdominal Infection, Unconfirmed  0/15 (0.00%)  1/21 (4.76%) 
Respiratory Infection  1/15 (6.67%)  3/21 (14.29%) 
Sepsis  1/15 (6.67%)  1/21 (4.76%) 
Skin Infection  2/15 (13.33%)  3/21 (14.29%) 
Tonsilar Infection  0/15 (0.00%)  1/21 (4.76%) 
Urinary Tract Infection  2/15 (13.33%)  0/21 (0.00%) 
Vaginal Infection  0/15 (0.00%)  1/21 (4.76%) 
Investigations     
Coagulopathy  0/15 (0.00%)  1/21 (4.76%) 
Transaminitis  0/15 (0.00%)  2/21 (9.52%) 
Metabolism and nutrition disorders     
Hyperglycemia  0/15 (0.00%)  1/21 (4.76%) 
Musculoskeletal and connective tissue disorders     
Aseptic/Avascular Necrosis  2/15 (13.33%)  1/21 (4.76%) 
Nervous system disorders     
Acute Brain Infarction  0/15 (0.00%)  1/21 (4.76%) 
Meningitis  1/15 (6.67%)  0/21 (0.00%) 
Neuropathy  1/15 (6.67%)  1/21 (4.76%) 
Brain Lesion, Nonspecific  0/15 (0.00%)  1/21 (4.76%) 
Seizure  0/15 (0.00%)  1/21 (4.76%) 
Renal and urinary disorders     
Acute Kidney Injury  0/15 (0.00%)  1/21 (4.76%) 
Cystitis  2/15 (13.33%)  3/21 (14.29%) 
Pyelocaliectasis  0/15 (0.00%)  1/21 (4.76%) 
Renal Injury Requiring Dialysis  0/15 (0.00%)  1/21 (4.76%) 
Respiratory, thoracic and mediastinal disorders     
Acute Respiratory Distress Syndrome  3/15 (20.00%)  2/21 (9.52%) 
Hypoxia  0/15 (0.00%)  1/21 (4.76%) 
Lung Collapse  0/15 (0.00%)  1/21 (4.76%) 
Nasal Infection  1/15 (6.67%)  0/21 (0.00%) 
Nose Bleeds  0/15 (0.00%)  1/21 (4.76%) 
Pneumonia  4/15 (26.67%)  10/21 (47.62%) 
Pulmonary Hemorrhage  1/15 (6.67%)  1/21 (4.76%) 
Sinusitis  1/15 (6.67%)  1/21 (4.76%) 
Vocal Fold Erythema and Leukoplakia  1/15 (6.67%)  0/21 (0.00%) 
Skin and subcutaneous tissue disorders     
Erythema at Central Line Site  0/15 (0.00%)  1/21 (4.76%) 
Folliculitis  1/15 (6.67%)  0/21 (0.00%) 
Pustular Rash  1/15 (6.67%)  0/21 (0.00%) 
Spongiotic Dermatitis  1/15 (6.67%)  0/21 (0.00%) 
Vascular disorders     
Capillary Leak Syndrome  0/15 (0.00%)  1/21 (4.76%) 
Deep Vein Thrombosis  1/15 (6.67%)  1/21 (4.76%) 
Hypertension  4/15 (26.67%)  8/21 (38.10%) 
Hypotension  0/15 (0.00%)  1/21 (4.76%) 
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. Jakub Tolar, MD
Organization: Masonic Cancer Center, University of Minnesota
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00455312     History of Changes
Other Study ID Numbers: MT2006-06
0612M98727 ( Other Identifier: Institutional Review Board, University of Minnesota )
First Submitted: March 30, 2007
First Posted: April 3, 2007
Results First Submitted: February 6, 2017
Results First Posted: May 31, 2017
Last Update Posted: December 5, 2017