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Allogeneic Hematopoietic Stem Cell Transplantation

JEB RIC for Epidermolysis Bullosa

Phase 1 & 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 year and 2 years post-transplant

Awards & highlights

Study Summary

This trial is testing whether infusions of bone marrow or cord blood from a healthy donor can improve the symptoms of epidermolysis bullosa, a condition that causes very fragile skin.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 1 year and 2 years post-transplant

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~1 year and 2 years post-transplant

This trial's timeline: 3 weeks for screening, Varies for treatment, and 1 year and 2 years 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

Percentage of Participants With Event-free Survival

Secondary outcome measures

Average Biochemical Improvement

Durability of HSC Donor Engraftment in the Skin

Measure Patients Quality of Life Using a Questionnaire

+3 more

Trial Design

4Treatment groups

Experimental Treatment

Group I: RDEB RICExperimental Treatment6 Interventions

Recessive Dystrophic EB (RDEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)

Group II: RDEB MacExperimental Treatment5 Interventions

Recessive Dystrophic EB (RDEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg

Group III: JEB RICExperimental Treatment6 Interventions

Junctional EB (JEB) - Reduced Intensity Condition Participants received Fludarabine 500 mg/m2, Cyclosphosphamide 50 mg/kg, equine Anti-thymocyte globulin 90 mg/kg, and low dose total body irradiation (either 200 or 300 cGy)

Group IV: JEB MACExperimental Treatment5 Interventions

Junctional EB (JEB) - Myeloablative conditioning (MAC) Participants receive Myeloablative Busulfan (targeting AUC 1000 umol/min), Fludarabine 75 mg/m2, and Cyclophosphamide 200 mg/kg

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Myeloablative Busulfan

2010

Completed Phase 2

~40

Cyclophosphamide

1995

Completed Phase 3

~3770

Fludarabine

2012

Completed Phase 3

~1100

Anti-thymocyte globulin

2010

Completed Phase 4

~470

Mesenchymal stem cell transplantation

2010

Completed Phase 2

~40

Total body irradiation

2008

Completed Phase 3

~1080

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

2010

Completed Phase 2

~40

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

14,593 Total Patients Enrolled

3 Trials studying Epidermolysis Bullosa

58 Patients Enrolled for Epidermolysis Bullosa

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

2 Previous Clinical Trials

33 Total Patients Enrolled

2 Trials studying Epidermolysis Bullosa

33 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.

Are there vacancies for participation in this research?

"As of this moment, applications for participation in the clinical trial are not being accepted. The study was initially posted on January 1st 2010 and has since been adjusted as recently as September 28th 2022. If you wish to explore other avenues, there are currently 28 epidermolysis bullosa trials with open admissions and 989 Transplant in Epidermolysis Bullosa studies that will accept new participants."

Answered by AI

Has there been any research regarding the efficacy of Transplant in Epidermolysis Bullosa?

"Currently, there are 989 medical studies in progress regarding Transplant in Epidermolysis bullosa; 180 of these clinical trials have entered Phase 3. 29470 sites across the USA are running research into this subject, with most situated within Philadelphia, Pennsylvania."

Answered by AI

How many participants are there in this trial?

"At this time, the clinical trial is not accepting applications. The original posting was on January 1st 2010 and it was last edited September 28th 2022. For those still keen to participate in a medical study involving epidermolysis bullosa, there are presently 28 active trials plus an additional 989 transplantation studies actively looking for participants."

Answered by AI

For what medical issues is Transplant in Epidermolysis Bullosa typically applied?

"Transplantation in Epidermolysis bullosa is an oft-utilized treatment for multiple sclerosis, lupus nephritis, acute myelocytic leukemia and other related conditions."

Answered by AI

Recent research and studies

clinicaltrials.govStudy

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

2010. "Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT01033552.