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Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

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ClinicalTrials.gov Identifier: NCT00478244
Recruitment Status : Terminated (Competing studies)
First Posted : May 24, 2007
Results First Posted : February 11, 2013
Last Update Posted : December 28, 2017
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
Condition Epidermolysis Bullosa
Interventions Drug: busulfan
Drug: cyclophosphamide
Drug: fludarabine phosphate
Procedure: hematopoietic bone marrow transplantation
Enrollment 7

Recruitment Details  
Pre-assignment Details All subjects were registered with the BMT Biostatistical Support Group at the University of Minnesota.
Arm/Group Title Epidermolysis Bullosa (EB) Patients
Hide Arm/Group Description 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 .
Period Title: Overall Study
Started 7
Completed 6
Not Completed 1
Reason Not Completed
Did not receive transplant             1
Arm/Group Title Epidermolysis Bullosa (EB) Patients
Hide Arm/Group Description 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 .
Overall Number of Baseline Participants 7
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
<=18 years
7
 100.0%
Between 18 and 65 years
0
   0.0%
>=65 years
0
   0.0%
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 7 participants
6.0  (4.5)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 7 participants
Female
4
  57.1%
Male
3
  42.9%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 7 participants
7
1.Primary Outcome
Title Number of Patients With Detectable Collagen Type VII
Hide Description Number of patients with epidermolysis bullosa who had collagen type VII. Type VII collagen defects cause recessive dystrophic epidermolysis bullosa (RDEB), a blistering skin disorder often accompanied by epidermal cancers.
Time Frame Day 100 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
5
2.Secondary Outcome
Title Number of Patients With >70% Donor Chimerism
Hide Description Number of patients with donor chimerism - percentage of donor cells in the patient via the peripheral blood or bone marrow.
Time Frame Days 21, 100, 180, 365 and 730 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
Day 21 6
Day 100 5
Day 180 5
Day 365 5
Day 730 5
3.Secondary Outcome
Title Number of Patients With Transplant-Related Mortality
Hide Description Number of patients who died due to complications of the transplant (includes all deaths without previous relapse or progression).
Time Frame Day 180 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
0
4.Secondary Outcome
Title Number of Patients With Platelet Engraftment
Hide Description Number of patients with a platelet count >5 x 10^10 cells/liter for 3 consecutive measurements.
Time Frame Day 180 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
5
5.Secondary Outcome
Title Number of Patients With Acute Graft-Versus-Host Disease (GVHD)
Hide Description Number of patients with GVHD. 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 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
1
6.Secondary Outcome
Title Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)
Hide Description Number of patients with cGVHD; a severe long-term complication created by infusion of donor cells into a foreign host.
Time Frame Day 365 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
0
7.Secondary Outcome
Title Overall Survival
Hide Description Survival is defined as the number of patients that were alive post transplant.
Time Frame 1 year and 2 years Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
1 Year Post Transplant 5
2 Years Post Transplant 5
8.Secondary Outcome
Title Number of Patients With Donor Derived Cells in Skin
Hide Description Number of patients who had donor skin chimerism - donor cells in the patient's epidermis (a state in bone marrow transplantation in which bone marrow and host cells exist compatibly without signs of graft-versus-host rejection disease).
Time Frame Day 90 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
6
9.Secondary Outcome
Title Number of Patients With Resistance to Blister Formation
Hide Description Resistance to Blister Formation demonstrated by response to negative pressure.
Time Frame Month 1 through Month 24 Inclusive
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
Added blister formation testing later in study; only 2 patients had pre-transplant test.
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 2
Measure Type: Number
Unit of Measure: participants
2
10.Secondary Outcome
Title Number of Patients With Neutrophil Engraftment
Hide Description Number of patients with an absolute neutrophil count >5 x 10^8 cells/liter for 3 consecutive days.
Time Frame Day 42 Post Transplant
Show Outcome Measure DataHide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Evaluable Patients
Hide Arm/Group Description:
Includes only patients that received transplant (one patient died prior to transplant).
Overall Number of Participants Analyzed 6
Measure Type: Number
Unit of Measure: participants
6
Time Frame All subjects were monitored continuously and only serious unexpected and selected serious adverse experiences were collected during the first 100 days after cell infusion.
Adverse Event Reporting Description Serious adverse events include: marrow engraftment failure, severe acute graft-versus-host (GVHD) (Grades III and IV), and death. Death was collected on 6 of 7 patients enrolled.
 
Arm/Group Title Epidermolysis Bullosa (EB) Patients
Hide Arm/Group Description 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 .
All-Cause Mortality
Epidermolysis Bullosa (EB) Patients
Affected / at Risk (%)
Total   --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Epidermolysis Bullosa (EB) Patients
Affected / at Risk (%) # Events
Total   6/7 (85.71%)    
Blood and lymphatic system disorders   
Graft failure † 1  1/7 (14.29%)  1
General disorders   
Death † 1  1/7 (14.29%)  1
Infections and infestations   
Bacterial infection † 1  6/7 (85.71%)  6
Fungal infection † 1 [1]  2/7 (28.57%)  2
Viral infection † 1  1/7 (14.29%)  4
Metabolism and nutrition disorders   
Hyperbilirubinemia † 1  4/7 (57.14%)  4
Renal and urinary disorders   
Hemorrhagic cystitis † 1  1/7 (14.29%)  1
Renal insufficiency † 1  2/7 (28.57%)  2
Respiratory, thoracic and mediastinal disorders   
Acute respiratory distress syndrome † 1  1/7 (14.29%)  1
Skin and subcutaneous tissue disorders   
Grade IV skin toxicity † 1  1/7 (14.29%)  1
†
Indicates events were collected by systematic assessment
1
Term from vocabulary, CTCAE (4.0)
[1]
Candida sepsis
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Epidermolysis Bullosa (EB) Patients
Affected / at Risk (%) # Events
Total   0/7 (0.00%)    
[Not Specified]
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
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov Identifier: NCT00478244     History of Changes
Other Study ID Numbers: MT2006-15
CDR0000546620 ( Other Identifier: PDQ )
UMN-0702M01504 ( Other Identifier: IRB, University of Minnesota )
First Submitted: May 23, 2007
First Posted: May 24, 2007
Results First Submitted: July 17, 2012
Results First Posted: February 11, 2013
Last Update Posted: December 28, 2017