There are several clinical trials that are recruiting and/or underway. We have listed some below, but you can click here for a complete list of clinical trials.
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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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Itch Treatment in EB Patients
Stanford is recruiting individuals for a trial to test the safety and efficacy of VPD-737, or Serlopitant, in reducing itch in adolescents and adults with EB. This drug, which has been successful in reducing severe itch in adults with chronic pruritis, 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. Click here to learn more about this trial.
Please visit Stanford's EB Research page for updates on all ongoing research. |
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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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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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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®. Read the announcement from Amryt below or see the study record on clinicaltrials.gov for more information on EASE.
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