36. 表皮水疱症 Epidermolysis bullosa Clinical trials / Disease details


臨床試験数 : 163 薬物数 : 185 - (DrugBank : 46) / 標的遺伝子数 : 50 - 標的パスウェイ数 : 125

  
1 trial found
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1EUCTR2015-004592-74-AT
(EUCTR)
01/06/201619/01/2016Gene Therapy for patients with Recessive Dystrophic Epidermolysis Bullosa (RDEB).PROSPECTIVE, OPEN LABEL, UNCONTROLLED CLINICAL TRIAL TO ASSESS THE SAFETY AND EFFICACY OF AUTOLOGOUS CULTURED EPIDERMAL GRAFTS CONTAINING EPIDERMAL STEM CELLS GENETICALLY MODIFIED WITH A GAMMA-RETROVIRAL (RV) VECTOR CARRYING COL7A1 cDNA FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH RECESSIVE DYSTROPHIC EPIDERMOLYSIS BULLOSA. - HOLOGENE7 RDEB is characterized by generalized skin blistering, erosions, crusts, atrophic scarring, onychodystrophy and loss of nails, mutilating pseudosyndactyly of hands and feet, as well as oral cavity lesions, esophageal strictures and eye and genitourinary tract lesions, all of which can also lead to anemia, iron deficiency and growth delay. Aggressive metastasizing squamous cell carcinomas are a common complication of RDEB, which reduce patients’ average life expectancy to less than 40 years.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17]Product Name: HOLOGENE7
Product Code: HOLOGENE7
INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector
Other descriptive name: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL7A1-encoding retroviral vector, obtained from secondary culture of ex vivo expanded autologous human keratinocytes.
Holostem Terapie Avanzate s.r.l.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
12Phase 1;Phase 2Austria