Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Masonic Cancer Center, University of Minnesota
Age
0–25
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Diagnosis of severe form of EB characterized by collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis).
Adequate organ function within 4 weeks of study registration defined as:
* Renal: glomerular filtration rate within normal range for age
* Hepatic: Hepatic: bilirubin, AST/ALT, ALP \< 5 x upper limit of normal
* Pulmonary: adequate pulmonary function in the opinion of the enrolling investigator
* Cardiac: left ventricular ejection fraction ≥ 45%, normal EKG or approved by Cardiology for transplant
Sexually active participants must agree to use adequate birth control for the during the study period (from before the start of the preparative chemotherapy through 1 year post-transplant)
Available donor per section 5: targeted MFI \< 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
You may not be if
beta 3 laminin JEB mutants
Active untreated systemic infection at time of transplantation (including active infection with Aspergillus or other mold within 30 days)
History of HIV infection
Evidence of squamous cell carcinoma
Pregnant or breast feeding. Females of child-bearing potential must have a negative pregnancy test prior to study registration as the agents administered in this study are Pregnancy Category C and D.
