Epidermolysis Bullosa Clinical Trial
Official title:
MT2009-09: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Stem Cell Transplantation and "Off-the-shelf" Mesenchymal Stem Cells
Verified date | March 2024 |
Source | Masonic Cancer Center, University of Minnesota |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 32 |
Est. completion date | August 12, 2021 |
Est. primary completion date | August 12, 2021 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 25 Years |
Eligibility | 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 transplantation (including active infection with Aspergillus or other mold within 30 days). - History of human immunodeficiency virus (HIV) infection - Evidence of squamous cell carcinoma - Donor has EB - Pregnancy females of child-bearing age must have a documented negative pregnancy test and agree to use contraception as a condition for enrollment. |
Country | Name | City | State |
---|---|---|---|
United States | University of Minnesota Masonic Cancer Center and Medical Center | Minneapolis | Minnesota |
Lead Sponsor | Collaborator |
---|---|
Masonic Cancer Center, University of Minnesota |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Percentage of Participants With Event-free Survival | Event-free survival rate, with an event defined as death or failure to have a demonstrable increase in collagen, laminin, intergrin, keratin or plakin deposition. Assessed at follow up appointments through questionnaire and patient samples. | 1 year and 2 Years Post-transplant | |
Secondary | Percentage of Participants Transplant-related Mortality (TRM) | Incidence of transplant-related mortality (TRM) | 180 Days Post Transplant | |
Secondary | Average Biochemical Improvement | Pattern of biochemical improvement measured by cumulative increase in protein expression and related structural and physical changes | 1 Year Post-Transplant | |
Secondary | Measure Patients Quality of Life Using a Questionnaire | Health quality of life questionnaire as compared to pretreatment results. Scores can range from 0 to 100. The QOLS scores are summed so that a higher score indicates higher quality of life. | Pretreatment and 1 year | |
Secondary | Durability of HSC Donor Engraftment in the Skin | Incidence of HSC donor engraftment in the skin | 100 Days | |
Secondary | Probability of Survival | Surviving patients one year after engraftment | 1 Year | |
Secondary | Percentage of Participants Who Experienced Acute GVHD | Incidence of acute GCHD | 100 Days |
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