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
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Tracking Information | ||||
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First Submitted Date ICMJE | May 23, 2007 | |||
First Posted Date ICMJE | May 24, 2007 | |||
Results First Submitted Date ICMJE | July 17, 2012 | |||
Results First Posted Date ICMJE | February 11, 2013 | |||
Last Update Posted Date | December 28, 2017 | |||
Study Start Date ICMJE | April 2007 | |||
Actual Primary Completion Date | August 2011 (Final data collection date for primary outcome measure) | |||
Current Primary Outcome Measures ICMJE |
Number of Patients With Detectable Collagen Type VII [ Time Frame: Day 100 Post Transplant ] 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.
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Original Primary Outcome Measures ICMJE |
Incidence of detectable donor-derived collagen type VII by day 100 | |||
Change History | ||||
Current Secondary Outcome Measures ICMJE |
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Original Secondary Outcome Measures ICMJE |
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Current Other Pre-specified Outcome Measures | Not Provided | |||
Original Other Pre-specified Outcome Measures | Not Provided | |||
Descriptive Information | ||||
Brief Title ICMJE | Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa | |||
Official Title ICMJE | Allogeneic Hematopoietic Cell Transplantation to Correct the Biochemical Defect and Create Tolerance to Donor Tissue in Subjects With Epidermolysis Bullosa | |||
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. |
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Detailed Description | OBJECTIVES: Primary
Secondary
After completion of study treatment, patients are followed periodically for at least 5 years. PROJECTED ACCRUAL: 30 patients |
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Study Type ICMJE | Interventional | |||
Study Phase ICMJE | Not Applicable | |||
Study Design ICMJE | Allocation: N/A Intervention Model: Single Group Assignment Masking: None (Open Label) Primary Purpose: Treatment |
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Condition ICMJE | Epidermolysis Bullosa | |||
Intervention ICMJE |
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Study Arms ICMJE | Experimental: Epidermolysis Bullosa (EB) Patients
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant.
Interventions:
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Publications * | 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. Erratum in: N Engl J Med. 2010 Sep 30;363(14):1383. Woodley, David T [added]; Chen, Mei [added]. | |||
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline. |
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Recruitment Information | ||||
Recruitment Status ICMJE | Terminated | |||
Actual Enrollment ICMJE |
7 | |||
Original Enrollment ICMJE |
10 | |||
Actual Study Completion Date ICMJE | August 2011 | |||
Actual Primary Completion Date | August 2011 (Final data collection date for primary outcome measure) | |||
Eligibility Criteria ICMJE | Inclusion criteria:
Exclusion criteria:
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Sex/Gender ICMJE |
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Ages ICMJE | up to 25 Years (Child, Adult) | |||
Accepts Healthy Volunteers ICMJE | No | |||
Contacts ICMJE | Contact information is only displayed when the study is recruiting subjects | |||
Listed Location Countries ICMJE | United States | |||
Removed Location Countries | ||||
Administrative Information | ||||
NCT Number ICMJE | NCT00478244 | |||
Other Study ID Numbers ICMJE | MT2006-15 CDR0000546620 ( Other Identifier: PDQ ) UMN-0702M01504 ( Other Identifier: IRB, University of Minnesota ) |
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Has Data Monitoring Committee | Yes | |||
U.S. FDA-regulated Product | Not Provided | |||
IPD Sharing Statement ICMJE | Not Provided | |||
Current Responsible Party | Masonic Cancer Center, University of Minnesota | |||
Original Responsible Party | Not Provided | |||
Current Study Sponsor ICMJE | Masonic Cancer Center, University of Minnesota | |||
Original Study Sponsor ICMJE | University of Minnesota | |||
Collaborators ICMJE | Not Provided | |||
Investigators ICMJE |
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PRS Account | Masonic Cancer Center, University of Minnesota | |||
Verification Date | December 2017 | |||
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP |