Allogeneic Hematopoietic Stem Cell Transplant For Epidermolysis Bullosa

Stopped early · Not applicable

Conditions studied: Epidermolysis Bullosa

In brief

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.

Key facts

Study ID

NCT00478244

Run by

Masonic Cancer Center, University of Minnesota

People needed

7

Starts

2007-04-01

Expected to finish

2011-08-01

Last updated by the study team

2017-12-28

Who can join

Age: any, up to 25. Sex: any. Healthy volunteers: not accepted.

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
• 8/8 HLA A, B, C, DRB1 allele level matched unrelated marrow donor (third priority)
• 7/8 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

Where it is running

• Masonic Cancer Center, University of Minnesota — Minneapolis, Minnesota, United States

Full record on ClinicalTrials.gov

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