Phase 1/2 Trial of PTR-01 in Adult Patients With RDEB
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Phoenix Tissue Repair is currently recruiting adults with RDEB for their Phase 1/2 trial of PTR-01, a protein replacement therapy that uses recombinant type VII collagen, to assess the therapy's safety and efficacy. Preclinical studies of PTR-01 have demonstrated anchoring fibril formation and improved survival in RDEB models. The US FDA and European EMA have gratned Orphan Drug Designation to this therapy. The 3 sites include Stanford University, Children's Hospital Colorado, and Thomas Jefferson University.
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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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FCX-007 Gene Therapy for Recessive Dystrophic EB
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In July 2019, Fibrocell initiated DEFI-RDEB, their Phase 3 Clinical Trial of FCX-007, their cell therapy for RDEB. The therapy involves taking cells from a patient and genetically modify them to express collagen VII protein, which is lacking in patients with RDEB. These corrected cells are then injected back into the patient intradermally. FCX-007 has received several designations from the US FDA, including Orphan Drug, Rare Pediatric Disease, Fast Track, and RMAT.
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Amryt Pharma is currently conducting the EASE Study, a global Phase III Clinical Trial 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.
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VIITAL Study: Phase 3 Clinical Trial for EB-101
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The U.S. Food and Drug Administration cleared Abeona Therapeutics Inc to proceed with their VIITAL Study, the pivotal Phase 3 clinical trial for EB-101, a gene therapy for treatment of RDEB. Abeona, who provided the FDA with supplemental evidence of EB-101's stability when being transported to clinical sites, expects to initiate VIITAL in Q1 2020. This trial is not currently recruiting.
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USC's Gentamicin Studies
Researchers at USC are studying Gentamicin as a therapy for RDEB adn JEB causes by nonsense mutations. Gentamicin is an aminoglycoside antibiotic which can override nonsense mutations in DNA sequences, allowing full length, functional proteins to form. USC is currently recruiting for all 3 trials below.
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Intravenous Gentamicin Therapy for RDEB
Individuals with a nonsense mutation in the Collagen VII gene may be eligibel for this Phase 1/2 Clinical Trial. 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.
Gentamicin for JEB
Individuals with nonsense mutations in the LAMB3 gene may be eligible for this clinical trial. 80% of Herlitz JEB (H-JEB) have LAMB3 mutations, 95% of which are nonsense mutations. The investigators have already demonstrated that Gentamicin induced nonsense readthrough, producing full length protein, in several nonsense mutations. In this trial, Gentamicin will be delivered to 3 subjects intravenously and 3 subjects topically.
Optimizing IV Gentamicin in JEB
Individuals with nonsense mutations in the LAMB3 gene or LAM3A gene may be eligible for this clinical trial. The investigators aim to optimize the intravenous gentamicin regimen for enhanced therapeutic results. The study will measure expression of Lamnin 332 in the skin as well as the safety of the therapy.
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Stem Cell Transplant
Delivers Trial: CCP-020 Diacerein 1% Ointment for EB Simplex
