CompletedPhase 1/2

Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs

United States32 participantsStarted 2010-01

Plain-language summary

This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering.

Who can participate

Age range

25 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Diagnosis of severe form of epidermolysis bullosa (EB) characterized by collagen, laminin, integrin, keratin or plakin deficiency. Assessment criteria for severe EB: * Documented collagen, laminin, integrin, keratin or plakin deficiency (by immunofluorescence staining with protein specific antibodies or Western blotting and by mutation analysis) * Adequate Organ Function Criteria * Renal: glomerular filtration rate within normal range for age * Hepatic: bilirubin, aspartate aminotransferase/alanine aminotransferase (AST/ALT), Alkaline phosphatase (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 electrocardiogram (EKG) or approved by Cardiology for transplant. * Available Healthy HSC Donor (order of preference) * Related Donor (marrow or UCB) * HLA-A, B, C, DRB1 genotypic identical (sibling) donor * HLA-A, B, C, DRB1 phenotypic identical donor * 7/8 HLA matched donor at HLA-A, B, C, DRB1 * Unrelated Donor * Marrow * HLA-A, B, C, DRB1 phenotypic identical donor * 7/8 HLA matched donor at HLA-A, B, C, DRB1 * UCB * HLA-A, B (antigen level) and DRB1 (allele level) matched donor * 5/6 HLA matched donor at HLA-A, B, DRB1 * 4/6 HLA matched donor at HLA-A, B, DRB1 * Voluntary written consent Absence of Exclusion Criteria: * Active systemic infection at time of …

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Percentage of Participants With Event-free Survival

Timeframe: 1 year and 2 Years Post-transplant

Trial details

NCT IDNCT01033552

SponsorMasonic Cancer Center, University of Minnesota

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2021-08-12

Results submitted2023-12-05

View on ClinicalTrials.gov More Epidermolysis Bullosa trials