MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
Status and phase
Completed
Phase 2
Conditions
Epidermolysis Bullosa
Treatments
Radiation: Total Body Irradiation
Drug: Thymoglobulin
Biological: Donor mesenchymal stem cell infusions
Procedure: Bone marrow infusion
Drug: Mycophenolate Mofetil
Drug: Cyclophosphamide
Drug: Busulfan
Drug: Fludarabine
Drug: Tacrolimus
Details and patient eligibility
About
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).
Enrollment
17 patients
Sex
All
Ages
Under 25 years old
Volunteers
No Healthy Volunteers
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.
Trial design
Primary purpose
Treatment
Allocation
Non-Randomized
Interventional model
Parallel Assignment
Masking
None (Open label)
17 participants in 8 patient groups
RDEB: HCT plus MSC Arm B
Experimental group
Description:
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
JEB: HCT plus MSC Arm B
Experimental group
Description:
Junctional epidermolysis bullosa (JEB) patient treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (300 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
RDEB: HCT Plus MSC Arm E
Experimental group
Description:
Recessive dominant epidermolysis bullosa (RDEB) patients treated per study regimen on a post-transplant cyclophosphamide allogeneic hematopoietic cell transplant (HCT) platform conditioned with reduced intensity chemotherapy and low dose total body irradiation (400 cGy), followed post-HCT by serial infusions of donor-derived mesenchymal stromal cells (MSC).
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
HCT with 300 cGy of TBI Arm A
Experimental group
Description:
Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant without mesenchymal stem cell infusions.
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
Re-Transplant Arm C
Experimental group
Description:
Epidermolysis bullosa patients treated regardless of original transplant arm with re-transplant using 300 cGy of TBI.
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
HCT Arm D
Experimental group
Description:
HLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGY BID of TBI (400 cGy total).
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
HCT Alone Arm F
Experimental group
Description:
HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
HCT plus MSC Arm G
Experimental group
Description:
HLA-mismatched epidermolysis bullosa patients treated with hematopoietic cell transplant plus serial MSC infusions using 200 cGy BID of TBI (400 cGy total) + addition of low dose busulfan for recipients of HLA-mismatched bone marrow
Treatment:
Drug: Tacrolimus
Drug: Fludarabine
Drug: Busulfan
Drug: Cyclophosphamide
Drug: Mycophenolate Mofetil
Procedure: Bone marrow infusion
Biological: Donor mesenchymal stem cell infusions
Drug: Thymoglobulin
Radiation: Total Body Irradiation
Trial contacts and locations
1
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Data sourced from clinicaltrials.gov
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