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Bone marrow infusion for Epidermolysis Bullosa

Phase 2

Waitlist Available

Led By Jakub Tolar, MD, PhD

Research Sponsored by Masonic Cancer Center, University of Minnesota

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Be younger than 65 years old

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 1 and 2 year post-transplant

Awards & highlights

Study Summary

This trial is testing whether a transplant of stem cells can improve the event-free survival of people with severe epidermolysis bullosa.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 and 2 year post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 and 2 year post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 and 2 year post-transplant for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Event-free survival

Secondary outcome measures

Lymphoid Chimerism

Myeloid Chimerism

Percentage change of a patient's iscorEB

+2 more

Trial Design

7Treatment groups

Experimental Treatment

Group I: CLOSED TO ACCRUAL Arm B: HCT plus MSC, 300 cGy of TBIExperimental Treatment8 Interventions

Epidermolysis bullosa patients treated per study regimen with chemotherapy and hematopoietic stem cell transplant with mesenchymal stem cell infusions using 300 cGY of TBI.

Group II: CLOSED TO ACCRUAL Arm A: HCT with 300 cGy of TBIExperimental Treatment7 Interventions

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

Group III: Arm G: HCT plus MSC, 200 cGyExperimental Treatment9 Interventions

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

Group IV: Arm F: HCT Alone, 200 cGy BID of TBIExperimental Treatment8 Interventions

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

Group V: Arm E: HCT plus MSC, 200 cGy BID of TBIExperimental Treatment7 Interventions

HLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant plus serial MSC infusions using 200 cGY BID of TBI (400 cGy total)

Group VI: Arm D: HCT with 200 cGy BID of TBIExperimental Treatment6 Interventions

HLA-matched epidermolysis bullosa patients treated with hematopoietic cell transplant alone using 200 cGY BID of TBI (400 cGy total).

Group VII: Arm C: Re-Transplant with 300 cGy of TBIExperimental Treatment8 Interventions

Epidermolysis bullosa patients treated regardless of original transplant arm with re-transplant using 300 cGy of TBI.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fludarabine

2012

Completed Phase 3

~1100

Busulfan

2008

Completed Phase 3

~1120

Cyclophosphamide

1995

Completed Phase 3

~3780

Mycophenolate Mofetil

1997

Completed Phase 4

~2380

Bone marrow infusion

2016

Completed Phase 2

~20

Donor mesenchymal stem cell infusions

2016

Completed Phase 2

~20

Tacrolimus

2011

Completed Phase 4

~4740

Thymoglobulin

2005

Completed Phase 4

~1500

Total Body Irradiation

2006

Completed Phase 3

~820

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

Masonic Cancer Center, University of MinnesotaLead Sponsor

271 Previous Clinical Trials

14,608 Total Patients Enrolled

3 Trials studying Epidermolysis Bullosa

73 Patients Enrolled for Epidermolysis Bullosa

Jakub Tolar, MD, PhDPrincipal InvestigatorMasonic Cancer Center, University of Minnesota

2 Previous Clinical Trials

48 Total Patients Enrolled

2 Trials studying Epidermolysis Bullosa

48 Patients Enrolled for Epidermolysis Bullosa

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is this research initiative currently welcoming participants?

"According to information present on clinicaltrials.gov,this medical experiment is actively gathering participants. It was initially posted back in March 2016 and most recently updated in January 2022."

Answered by AI

Are there any preceding experiments involving Donor mesenchymal stem cell infusions?

"Currently there are 1,032 active trials for Donor mesenchymal stem cell infusions with 185 of them in the concluding phase 3. Of these studies, many can be found within Philadelphia, however 29625 sites across the globe host clinical research involving this type of therapy."

Answered by AI

How many people have taken part in this clinical research so far?

"Correct. According to clinicaltrials.gov, this scientific experiment is presently enlisting participants; it was initially published on March 1st 2016 and the latest update being made available on January 20th 2022. 84 individuals are needed from a single location for the trial's completion."

Answered by AI

What therapeutic applications are donor mesenchymal stem cell infusions most commonly utilized for?

"Donor mesenchymal stem cell infusions are known to effectively manage the symptoms of leukemia, lung cancer, and immune system suppression."

Answered by AI

How risky is it to receive mesenchymal stem cell infusions from a donor?

"Our safety rating for donor mesenchymal stem cell infusions is a 2, as these Phase 2 trials have only provided evidence of their security but not yet proven efficacy."

Answered by AI

Recent research and studies

British Journal of DermatologyJournal

Bone Marrow Transplant with Post‐transplant Cyclophosphamide for Recessive Dystrophic Epidermolysis Bullosa Expands the Related Donor Pool and Permits Tolerance of Nonhaematopoietic Cellular Grafts

Ebens, C.L., J.A. McGrath, K. Tamai, A. Hovnanian, J.E. Wagner, M.J. Riddle, D.R. Keene, et al.. 2019. “Bone Marrow Transplant with Post‐transplant Cyclophosphamide for Recessive Dystrophic Epidermolysis Bullosa Expands the Related Donor Pool and Permits Tolerance of Nonhaematopoietic Cellular Grafts”. British Journal of Dermatology. Wiley. doi:10.1111/bjd.17858.

clinicaltrials.govStudy

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

2016. "MT2015-20: Biochemical Correction of Severe EB by Allo HSCT and Serial Donor MSCs". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT02582775.