Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

This study has been terminated.
(Competing studies)
Sponsor:
Information provided by (Responsible Party):
Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier:
NCT00478244
First received: May 23, 2007
Last updated: January 4, 2013
Last verified: January 2013
Results First Received: July 17, 2012  
Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Epidermolysis Bullosa
Interventions: Drug: busulfan
Drug: cyclophosphamide
Drug: fludarabine phosphate
Procedure: hematopoietic bone marrow transplantation

  Participant Flow
  Hide Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
All subjects were registered with the BMT Biostatistical Support Group at the University of Minnesota.

Reporting Groups
  Description
Epidermolysis Bullosa (EB) Patients Epidermolysis bullosa patients enrolled for treatment with chemotherapy (Busulfan 0.8 or 1.1 mg/kg Days 6-9 before transplant; Fludarabine 25 mg/m^2 Days 3-5 before transplant; Cyclophosphamide 50 mg/kg Days 2-5 before transplant) and stem cell infusion (Day 0) followed by donor epidermal transplant .

Participant Flow:   Overall Study
    Epidermolysis Bullosa (EB) Patients  
STARTED     7  
COMPLETED     6  
NOT COMPLETED     1  
Did not receive transplant                 1  



  Baseline Characteristics
  Hide Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Epidermolysis Bullosa (EB) Patients Epidermolysis bullosa patients enrolled for treatment with chemotherapy (Busulfan 0.8 or 1.1 mg/kg Days 6-9 before transplant; Fludarabine 25 mg/m^2 Days 3-5 before transplant; Cyclophosphamide 50 mg/kg Days 2-5 before transplant) and stem cell infusion (Day 0) followed by donor epidermal transplant .

Baseline Measures
    Epidermolysis Bullosa (EB) Patients  
Number of Participants  
[units: participants]
  7  
Age  
[units: participants]
 
<=18 years     7  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  6.0  ± 4.5  
Gender  
[units: participants]
 
Female     4  
Male     3  
Region of Enrollment  
[units: participants]
 
United States     7  



  Outcome Measures
  Show All Outcome Measures

1.  Primary:   Number of Patients With Detectable Collagen Type VII   [ Time Frame: Day 100 Post Transplant ]

2.  Secondary:   Number of Patients With >70% Donor Chimerism   [ Time Frame: Days 21, 100, 180, 365 and 730 Post Transplant ]

3.  Secondary:   Number of Patients With Transplant-Related Mortality   [ Time Frame: Day 180 Post Transplant ]

4.  Secondary:   Number of Patients With Platelet Engraftment   [ Time Frame: Day 180 Post Transplant ]

5.  Secondary:   Number of Patients With Acute Graft-Versus-Host Disease (GVHD)   [ Time Frame: Day 100 Post Transplant ]

6.  Secondary:   Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)   [ Time Frame: Day 365 Post Transplant ]

7.  Secondary:   Overall Survival   [ Time Frame: 1 year and 2 years Post Transplant ]

8.  Secondary:   Number of Patients With Donor Derived Cells in Skin   [ Time Frame: Day 90 Post Transplant ]

9.  Secondary:   Number of Patients With Resistance to Blister Formation   [ Time Frame: Month 1 through Month 24 Inclusive ]

10.  Secondary:   Number of Patients With Neutrophil Engraftment   [ Time Frame: Day 42 Post Transplant ]


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats
  Hide Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information
  Hide More Information

Certain Agreements:  
All Principal Investigators ARE employed by the organization sponsoring the study.


Results Point of Contact:  
Name/Title: John E. Wagner, M.D.
Organization: Masonic Cancer Center, University of Minnesota
phone: 612-626-2961
e-mail: wagne002@umn.edu


No publications provided by Masonic Cancer Center, University of Minnesota

Publications automatically indexed to this study:

Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00478244     History of Changes
Other Study ID Numbers: MT2006-15, CDR0000546620, UMN-0702M01504
Study First Received: May 23, 2007
Results First Received: July 17, 2012
Last Updated: January 4, 2013
Health Authority: United States: Institutional Review Board