Current Research Trials

EB researchers from countries around the globe are investigating new therapies. Stem cell transplantation, protein replacement, and gene therapies are moving forward and various wound care therapies are being tested. All of these efforts are aimed at improving the lives of those with EB. While it has yet to be determined which of these varied modalities will ultimately be the most successful, all are contributing to what is known about EB. This assists not only today’s researchers, but provides a knowledge base for future efforts.  If you are interested in participating in a study, please explore the list below or visit

Current U.S. studies that are recruiting include:

Fibrocell Science Inc.'s Phase I/II Clinical Trial of FCX-007 for Treatment of Recessive Dystrophic Epidermolysis Bullosa
Fibrocell Science, Inc. is currently recruiting for a Phase I/II Clinical Trial for treatment of Recessive Dystrophic EB. A primary objective of this open-label trial is to evaluate the safety of FCX-007, a novel gene-therapy product candidate for the treatment of RDEB. Additionally, the trial will evaluate type VII collagen expression and the presence of anchoring fibrils resulting from FCX-007, as well as evidence of wound healing.

Click here to read more.

Stanford Medicine's Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa
Conditons: Recessive Dystrophic Epidermolysis Bullosa
Intervention: Genetic: LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)

Stanford is currently looking for RDEB subjects ages 13 and older for their study entitled "A Phase 1 Single Center Trial of Gene Transfer for Recessive Dystrophic Epidermolysis Bullosa (RDEB) using the drug LZRSE-Col7A1 Engineered Autologous Epidermal Sheets (LEAES)".

Click here to read more.

Amicus Therapeutics’ Phase 3 EB Clinical Trial
The ESSENCE Study, a Phase 3 clinical trial of SD-101, is an investigational topical cream being developed by Amicus, together with Scioderm, to treat skin blistering and lesions associated with Epidermolysis Bullosa (EB).

Click here to read more.

University of Miami and Berg Health’s Phase I Study of Topical BPM31510 3.0% Cream in Patients with Epidermolysis Bullosa
The University of Miami is currently recruiting patients 12 years and older (children, adults, seniors) with any form of Epidermolysis Bullosa (EB) for a phase I study. Patients participating will need to have at least 1 active EB wound between 2.5 and 50 cm2 for evaluation. The main objective of the study is to evaluate the safety and tolerability of topical BPM31510 3.0% cream. A travel stipend may be provided.

Click here to read more.

Stanford’s Study of Topical Sirolimus 2% for the Treatment of Painful Keratoderma in Epidermolysis Bullosa Simplex
Stanford is currently conducting a pilot study enrolling individuals above 4 years of age with epidermolysis bullosa simplex (EBS). The study focuses on the treatment of painful keratoderma (thickened skin & blisters) of the feet using topical sirolimus. The goal of the study is to discover new treatment for individuals affected by EBS and have unmet medical needs.

Click here to read more.

Stem Cell Transplant for Epidermolysis Bullosa
Condition: Epidermolysis Bullosa
Drug: Cyclophosphamide;   Drug: Fludarabine;  
Drug: Anti-thymocyte globulin;   Drug: Cyclosporine A;  
Drug: Mycophenolate mofetil;  
Procedure: Hematopoietic stem cell transplant;  
Procedure: Mesenchymal stem cell transplant;  
Radiation: Total body irradiation

Characteristics of Patients With Dystrophic Epidermolysis Bullosa
Conditon: Epidermolysis Bullosa Dystrophica

Completed Trials:

Study of Inflammatory Mechanisms in Epidermolysis Bullosa Simplex- Dowling Meara

Conditon: Epidermolysis Bullosa Simplex Dowling Meara
Intervention: Procedure: Taking of liquid and top of blisters

This trial is now closed.

Treatment of Chronic and Non-Chronic Wounds in Patients With Recessive Dystrophic Epidermolysis Bullosa Using Helicoll Collagen Dressings Versus Standard of Care
Conditons: Epidermolysis Bullosa Dystrophica; Epidermolysis Bullosa
Interventions: Device: Helicoll; Device: Standard of Care Dressings

This trial is now closed.

Characteristics of Adult Patients With Recessive Dystrophic Epidermolysis Bullosa
Conditon: Epidermolysis Bullosa Dystrophica

This trial is now closed.

Efficacy of Granulocyte Colony Stimulating Factor (GCSF) In Patients With Dystrophic Epidermolysis Bullosa
Conditon: Dystrophic Epidermolysis Bullosa
Intervention: Drug: Granulocyte Colony Stimulating Factor (GCSF)

This trial is now closed.

Treatment of Epidermolysis Bullosa Dystrophica by Polyphenon E (Epigallocatechin 3 Gallate)
Conditon: Epidermolysis Bullosa Dystrophica
Intervention: Drug: Polyphenon E before Placebo; Drug: placebo before treatment

This trial is now closed.

As you all know, Scioderm has been recruiting people with EB to participate in the SD-003 trial. In this trial, some participants will get a placebo which means there should be no treatment effect. Scioderm has reached an agreement with the FDA that will allow everyone completing the current study, SD-003, to participate in what they call an open label study (Study SD-004). This means that everyone who has completed the 3 month study will be eligible to receive the cream with the active ingredient.

This trial is now closed.

Phase IIb Study of SD-101 for Epidermolysis Bullosa (Study SD-003)
Scioderm conducted a 3-month study. SD-101 (or a placebo) will be added to patients’ current standard of care. SD-101 is a topical cream being developed to address the following key issues of patients with EB: wound healing, reducing blisters, decreasing pain, and decreasing itching. Patients must be 6 months or older, and may have the follow types of EB: Simplex, Dystrophic (Recessive), Junctional (non-Herlitz).

This trial is now closed.

Allogeneic Hematopoietic Stem Cell Transplant for Epidermolysis Bullosa (BMT for EB)
John E. Wagner, MD
Masonic Cancer Center, University of Minnesota
Contact: Timothy Krepski 612-273-2800 tkrepsk1 [at] fairview [dot] org
Click here for Results

Study of Alwextin® Cream in Treating Epidermolysis Bullosa
Dr. Amy S. Paller and Dr. Anne Mahoney: Northwestern University
Contact: Marcy Urbanich 312-695-6829 m-urbanich [at] northwestern [dot] edu
Click here for Results

Closed Trials:

A Prospective Single Center Within Subject Controlled Observational Study to Evaluate Apligraf(R) in Nonhealing Wounds of Subjects With Epidermolysis Bullosa
Conditons: Epidermolysis Bullosa; Skin Equivalent
Intervention: Device: Apligraf application

This study has been terminated.

Treatment of Dowling Maera Type of Epidermolysis Bullosa Simplex by Oral Erythromycin
Conditon: Epidermolysis Bullosa
Intervention: Drug: Oral erythromycin

Status of recruitment last updated October 2011.

Grafting of Epidermolysis Bullosa Wounds Using Cultured Revertant Autologous Keratinocytes
Conditon: Epidermolysis Bullosa
Intervention: Procedure: Grafting of Autologous Cultured Revertant Keratinocytes

This study has been withdrawn prior to enrollment.