Gene Transfer Study for Recessive Dystrophic EB
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Stanford is currently recruiting individuals with Recessive Dystrophic Epidermolysis Bullosa (RDEB) to participate in a gene transfer study. The researchers will genetically engineer the patient's own cells in the laboratory to express the missing type VII collagen protein. They will then make a skin graft of these corrected cells to transplant back into the patient. Click here to learn more.
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A Neurokinin-1 Receptor Antagonist for the Treatment of Itch in EB Patients
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Stanford is recruiting individuals with EB aged 13 and above to test the safety and efficacy of Serlopitant in reducing itch. This drug works by inhibiting the neurokinin-1 receptor. In EB patients, itching leads to increased disease severity by causing individuals to scratch and create new wounds.
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Stem Cell Transplant
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Researchers at the University of Minnesota are seeking participants with severe EB to determine if stem cell transplantation of bone marrow or umbilical cord blood from a healthy donor will correct protein deficiencies and reduce skin fragility. Click here for more information on this trial.
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Delivers Trial: CCP-020 Diacerein 1% Ointment for EB Simplex
FCX-007 Gene Therapy for Recessive Dystrophic EB
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Fibrocell is currently recruiting participants with Recessive Dystrophic EB for its FCX-007 gene therapy trial. The researchers take cells from the patient and genetically modify them to express the missing collagen VII protein. These cells are then injected back into the patient intradermally to treat wounds. The study will evaluate not only the safety of this therapy, but also type VII collagen expression, presence of anchoring fibrils, and evidence of wound healing. Click here to learn more about Fibrocell's study.
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Gentamicin Therapy for Recessive Dystrophic EB Nonsense Mutations
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Researchers at USC are currently recruiting participants with Recessive Dystrophic EB as a result of a nonsense mutation to participate in their intravenous gentamicin therapy trial. Gentamicin is an aminoglycoside antibiotic which can override nonsense mutations in DNA sequences, allowing full length, functional proteins to form. Previous studies of topical and intradermal gentamicin have shown positive results in Recessive Dystrophic EB patients, so the researchers are testing if intravenous intervention will provide a systemic solution. Click here to learn more about this trial.
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Amryt Pharma is currently conducting the EASE Study, a global phase III study of the topical gel Oleogel-S10 (AP101), to investigate its safety and efficacy in the treatment of EB wounds in patients with Dystrophic EB, Junctional EB, and Kindler Syndrome. The gel was authorized in the EU in 2016 as a treatment for partial thickness wounds in adults as Episalvan®. Several study sites are open for recruitement in the USA as well as Europe, Australasia, the Middle East, and Latin America. Read the most recent update from Amryt below or learn more at clinicaltrials.gov.
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