Study Stopped
Competing studies
Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa
Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa
3 other identifiers
interventional
7
1 country
1
Brief Summary
RATIONALE: In animal models, stem cells have been shown to home to the skin and repair the biochemical and structural abnormalities associated with recessive dystrophic epidermolysis bullosa (RDEB) (collagen 7 deficiency). PURPOSE: To determine the safety and effectiveness of stem cell infusion in the treatment of RDEB.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for not_applicable
Started Apr 2007
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
April 1, 2007
CompletedFirst Submitted
Initial submission to the registry
May 23, 2007
CompletedFirst Posted
Study publicly available on registry
May 24, 2007
CompletedPrimary Completion
Last participant's last visit for primary outcome
August 1, 2011
CompletedStudy Completion
Last participant's last visit for all outcomes
August 1, 2011
CompletedResults Posted
Study results publicly available
February 11, 2013
CompletedDecember 28, 2017
December 1, 2017
4.3 years
May 23, 2007
July 17, 2012
December 3, 2017
Conditions
Keywords
Outcome Measures
Primary Outcomes (1)
Number of Patients With Detectable Collagen Type VII
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.
Day 100 Post Transplant
Secondary Outcomes (9)
Number of Patients With >70% Donor Chimerism
Days 21, 100, 180, 365 and 730 Post Transplant
Number of Patients With Transplant-Related Mortality
Day 180 Post Transplant
Number of Patients With Platelet Engraftment
Day 180 Post Transplant
Number of Patients With Acute Graft-Versus-Host Disease (GVHD)
Day 100 Post Transplant
Number of Patients With Chronic Graft-Versus-Host Disease (cGVHD)
Day 365 Post Transplant
- +4 more secondary outcomes
Study Arms (1)
Epidermolysis Bullosa (EB) Patients
EXPERIMENTALEpidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
Interventions
Day -9 through Day -6: 1.1 mg/kg if \< 12 kg IV every 6 hours; 0.8 mg/kg if \> 12 kg.
Day -5 through Day -2: 50 mg/kg IV over 120 min.
Day -5 through Day -3: 25 mg/m2 IV over 60 min.
allogeneic bone marrow, peripheral stem cell or umbilical cord blood transplantation
Eligibility Criteria
You may qualify if:
- Diagnosis of epidermolysis bullosa (EB)
- Documented collagen type VII deficiency by:
- Antigenic mapping (LH7.2 antibody)
- Ultrastructure analysis of anchoring fibrils
- DNA mutation analysis
- Performance status: \>50% Lansky; \>50% Karnofsky
- Adequate organ function
- Renal: glomerular filtration rate \> 60ml/min/1.73m2 patients aged ≤ 10 years
- Hepatic: bilirubin, aspartate aminotransferase/alanine aminotransferase (AST/ALT), Alkaline phosphatase (ALP) \< 5 x upper limit of normal 4.2.3 Pulmonary: oxygen saturation \>92% 4.2.4 Cardiac: left ventricular ejection fraction \> 45%.
- Healthy related hematopoietic stem cell donor available and meeting 1 of the following criteria:
- HLA-A, B, DRB1-identical sibling bone marrow and/or umbilical cord blood donor (first priority)
- HLA-A, B, DRB1-matched or partially matched related donor (second priority)
- Donor may be a carrier but must be unaffected by EB
- /8 HLA A, B, C, DRB1 allele level matched unrelated marrow donor (third priority)
- /8 HLA-A, B, C, DRB1 allele level matched unrelated marrow donor or 4/6 HLA-A, B (antigen level), DRB1 (allele level) matched unrelated cord blood donor (fourth priority)
You may not qualify if:
- Active infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
- Squamous cell carcinoma of the skin
- History of human immunodeficiency virus (HIV) infection
- Prior transplantation with donor skin
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, 55455, United States
Related Publications (1)
Wagner JE, Ishida-Yamamoto A, McGrath JA, Hordinsky M, Keene DR, Woodley DT, Chen M, Riddle MJ, Osborn MJ, Lund T, Dolan M, Blazar BR, Tolar J. Bone marrow transplantation for recessive dystrophic epidermolysis bullosa. N Engl J Med. 2010 Aug 12;363(7):629-39. doi: 10.1056/NEJMoa0910501.
PMID: 20818854DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- John E. Wagner, M.D.
- Organization
- Masonic Cancer Center, University of Minnesota
Study Officials
- STUDY CHAIR
John E. Wagner, MD
Masonic Cancer Center, University of Minnesota
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 23, 2007
First Posted
May 24, 2007
Study Start
April 1, 2007
Primary Completion
August 1, 2011
Study Completion
August 1, 2011
Last Updated
December 28, 2017
Results First Posted
February 11, 2013
Record last verified: 2017-12