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

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).

Conditions

• Epidermolysis Bullosa

Interventions

DRUG

Thymoglobulin

0.5 mg/kg IV over 6 hours on day -9 and 2 mg/kg IV over 4 hours on day -8 and day -7 with premeds and solumedrol through day -2

DRUG

Cyclophosphamide

14.5 mg/kg IV over 1 hour day -6 and -5 50 mg/kg IV over 2 hours with mesna 40 mg/kg IV day +2 and +3

DRUG

Fludarabine

30 mg/m2 IV over 60 minutes days -6 through day -2

RADIATION

Total Body Irradiation

See arm description for dosing.

PROCEDURE

Bone marrow infusion

Bone marrow infusion on Day 0

DRUG

Tacrolimus

Day +5 through day +100 with goals of 5-10 ug/L (not used for HLA-identical related donors). When used in non-MSD recipients, tapered over 6-8 weeks starting at day +100.

DRUG

Mycophenolate Mofetil

15 mg/kg IV q8h (based upon actual body weight) with the maximum total daily dose not to exceed 3 grams. Day +5 through day 35

BIOLOGICAL

Donor mesenchymal stem cell infusions

Day 60, 100 and 180 (collected during donor BM harvest for graft)

DRUG

Busulfan

busulfan IV over 3 hours on days -3 and -2 for HLA-mismatched BM recipients only (Arms F and G)

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

2016-03-31

Primary Completion

2022-11-15

Completion

2023-07-26

FDA Drug

Yes

Countries

• United States

Study Locations