MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

STATUS

Recruiting
End date

Dec 25, 2024
participants needed

84
sponsor

Masonic Cancer Center, University of Minnesota

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Updated on 25 March 2022

See if I qualify

stem cell transplantation

fludarabine

mycophenolate mofetil

cyclophosphamide

ejection fraction

deficiency

thymoglobulin

mutation analysis

Summary

This is a single-institution, phase II study to determine the event-free survival at 1 year post allogeneic transplant and serial mesenchymal stem cell (MSC) infusions from a related donor (HLA identical, mismatched or haploidentical) or matched unrelated donor for the biochemical correction of severe epidermolysis bullosa (EB).

Details

Condition	Epidermolysis Bullosa
Treatment	cyclophosphamide, mycophenolate mofetil, busulfan, Fludarabine, Tacrolimus, Total body irradiation, Thymoglobulin, Bone marrow infusion, Donor mesenchymal stem cell infusions
Clinical Study Identifier	NCT02582775
Sponsor	Masonic Cancer Center, University of Minnesota
Last Modified on	25 March 2022

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Eligibility		Yes	No	Not Sure
Inclusion Criteria
	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
	Available donor per section 5: targeted MFI < 1,000 (MFI exceeding 1000 must be approved by the PI and treatment team.)
	the study period (from before the start of the preparative chemotherapy
	through 1 year post-transplant)
	Voluntary written consent - adult or parent (with information sheet for minors, if applicable) prior to any research related procedures or treatment
Exclusion Criteria
	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

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MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs