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

Brief Title

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

Official Title

MT2015-20: Biochemical Correction of Severe Epidermolysis Bullosa by Allogeneic Cell Transplantation and Serial Donor Mesenchymal Cell Infusions

Brief 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).
    

Study Phase

Phase 2

Study Type

Interventional

Primary Outcome

Event-free survival

Secondary Outcome

 Percentage change of a patient's iscorEB

Condition

Epidermolysis Bullosa

Intervention

Thymoglobulin

Study Arms / Comparison Groups

 CLOSED TO ACCRUAL Arm A: HCT with 300 cGy of TBI

Description:  Epidermolysis bullosa patients treated per study regimen with chemotherapy and stem cell transplant without mesenchymal stem cell infusions.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Status

Drug

Estimated Enrollment

84

Start Date

March 2016

Completion Date

December 2024

Primary Completion Date

December 2024

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.
      

Gender

All

Ages

N/A - 25 Years

Accepts Healthy Volunteers

No

Contacts

Jakub Tolar, MD, PhD, 612-273-8482, [email protected]

Location Countries

United States

Location Countries

United States

NCT ID

NCT02582775

Organization ID

2015LS076

Responsible Party

Sponsor

Study Sponsor

Masonic Cancer Center, University of Minnesota

Study Sponsor

Jakub Tolar, MD, PhD, Principal Investigator, Masonic Cancer Center, University of Minnesota

Verification Date

January 2022