Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs

Summary

This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will correct the collagen, laminin, integrin, or plakin deficiency and reduce the skin fragility characteristic of severe forms of EB. A secondary hypothesis is that mesenchymal stem cells from a healthy donor will enhance the safety and efficacy of the allogeneic hematopoietic stem cell transplant as well as serve as a source of renewable cells for the treatment of focal areas of residual blistering.

Conditions

• Epidermolysis Bullosa

Interventions

DRUG

Cyclophosphamide

Cyclophosphamide 50 mg/kg/day IV over 2 hours x 1 day, total dose 50 mg/kg will be administered on Day -6.

DRUG

Fludarabine

40 mg/m\^2/day intravenously on Days -6, -5, -4, -3 and -2.

DRUG

Anti-thymocyte globulin

30 mg/kg on Days -4, -3 and -2.

DRUG

Myeloablative Busulfan

Targeting AUC 1000 umol/min

PROCEDURE

Mesenchymal stem cell transplantation

infused via intravenous drip on Day 0

RADIATION

Total body irradiation

300 cGY on Day -1 administered in a single fraction at a dose rate of 10-19 cGy/minute prescribed to the midplane of the patient at the level of the umbilicus.

PROCEDURE

Bone marrow or umbilical cord blood (UCG) stem cell transplantation

Bone marrow or UCB products will be infused as soon as the product arrives and within 30 minutes. The product is infused via IV drip.

Sponsors & Collaborators

• Masonic Cancer Center, University of Minnesota

  lead OTHER

Principal Investigators

• Jakub Tolar, MD, PhD · Masonic Cancer Center, University of Minnesota

Study Design

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Model

PARALLEL

Eligibility

Max Age

25 Years

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2010-01-31

Primary Completion

2021-08-12

Completion

2021-08-12

FDA Drug

Yes

Countries

• United States

Study Locations