MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs

• Register: ClinicalTrials.gov
• Last refreshed on: 21 August 2023
• Main ID: NCT02582775
• Date of registration: 16/10/2015
• Prospective Registration: Yes
• Primary sponsor: Masonic Cancer Center, University of Minnesota
• Public title: MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs
• Scientific title: MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions
• Date of first enrolment: March 2016
• Target sample size: 17
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02582775
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Jakub Tolar, MD, PhD
• Affiliation: Masonic Cancer Center, University of Minnesota

Key inclusion & exclusion 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.



Age minimum: N/A
Age maximum: 25 Years
Gender: All

Health Condition(s) or Problem(s) studied

Epidermolysis Bullosa

Intervention(s)

Biological: Donor mesenchymal stem cell infusions

Drug: Cyclophosphamide

Drug: Busulfan

Drug: Tacrolimus

Procedure: Bone marrow infusion

Radiation: Total Body Irradiation

Drug: Fludarabine

Drug: Mycophenolate Mofetil

Drug: Thymoglobulin

Primary Outcome(s)

Event-free survival [Time Frame: 1 year post-transplant]

Secondary Outcome(s)

Myeloid Chimerism [Time Frame: Day 28, 60, 100, 180, and year 1 and 2 post-transplant]

Percentage change of a patient's iscorEB [Time Frame: 1 and 2 year post-transplant]

Lymphoid Chimerism [Time Frame: Day 28, 60, 100, 180, and year 1 and 2 post-transplant]

Quality of life [Time Frame: 1 year post-transplant]

Quality of life [Time Frame: 2 years post-transplant]

Transplant-related mortality [Time Frame: 180 days post-transplant]

Secondary ID(s)

2015LS076

Source(s) of Monetary Support

Please refer to primary and secondary sponsors