Phase 2
N=17
MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
Epidermolysis Bullosa
Bottom Line
View on ClinicalTrials.gov: NCT02582775 ↗Enrolled (actual)
17
Serious AEs
17.7%
Results posted
Sep 2024
Primary outcome: Primary: Event-free Survival — 4; 2; 1 Count of Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Thymoglobulin (Drug); Cyclophosphamide (Drug); Fludarabine (Drug); Total Body Irradiation (Radiation); Bone marrow infusion (Procedure); Tacrolimus (Drug); Mycophenolate Mofetil (Drug); Donor mesenchymal stem cell infusions (Biological); Busulfan (Drug)
- Age
- Pediatric, Adult
- Sex
- All
- Sponsor
- Masonic Cancer Center, University of Minnesota
- Primary completion
- Nov 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Event-free Survival |
4; 2; 1 | — |
| SECONDARY Change of a Patient's iscorEB |
-10; -24; 110 | — |
| SECONDARY Transplant-related Mortality |
0; 0; 0 | — |
| SECONDARY Average Change in Quality of Life |
10; 0 | — |
| SECONDARY Average Change in Quality of Life |
10; 0 | — |
| SECONDARY Lymphoid Chimerism |
100; 60; NA; 99; 78; NA | — |
| SECONDARY Myeloid Chimerism |
76; 95; 23; 32; 96; 0 | — |
| SECONDARY Average Change of a Patient's iscorEB |
9; -2 | — |
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).
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT02582775). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.