Epidermolysis Bullosa Clinical Trial
Official title:
MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
NCT number | NCT02582775 |
Other study ID # | 2015LS076 |
Secondary ID | |
Status | Completed |
Phase | Phase 2 |
First received | |
Last updated | |
Start date | March 2016 |
Est. completion date | July 26, 2023 |
Verified date | July 2023 |
Source | Masonic Cancer Center, University of Minnesota |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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).
Status | Completed |
Enrollment | 17 |
Est. completion date | July 26, 2023 |
Est. primary completion date | November 15, 2022 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A to 25 Years |
Eligibility | 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 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 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. |
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 | Event-free survival | An event defined as death or a 50% increase in a patient's IScoreEB from baseline | 1 year post-transplant | |
Secondary | Percentage change of a patient's iscorEB | iscorEB surveys are a validated, standard of care tool used to assess disease status in patients with Epidermolysis Bullosa. | 1 and 2 year post-transplant | |
Secondary | Transplant-related mortality | Cumulative incidence will be used to estimate the probability of relapse treating non-relapse death as a competing risk and transplant-related mortality conversely treating relapse as a competing risk. | 180 days post-transplant | |
Secondary | Quality of life | Measured by the Lansky or Karnofsky score (10-100) | 1 year post-transplant | |
Secondary | Quality of life | Measured by the Lansky or Karnofsky score (10-100) | 2 years post-transplant | |
Secondary | Lymphoid Chimerism | Proportion of lymphoid chimerism at various time-points. | Day 28, 60, 100, 180, and year 1 and 2 post-transplant | |
Secondary | Myeloid Chimerism | Proportion of myeloid chimerism at various time points. | Day 28, 60, 100, 180, and year 1 and 2 post-transplant |
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