36. 表皮水疱症
[臨床試験数:147,薬物数:170(DrugBank:40),標的遺伝子数:32,標的パスウェイ数:113]
Searched query = "Epidermolysis bullosa", "EBS", "JEB", "DDEB", "RDEB", "Kindler syndrome"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.
No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
1 | EUCTR2016-000095-17-AT (EUCTR) | 17/08/2017 | 07/06/2017 | Gene therapy for patients with Junctional Epidermolysis Bullosa (JEB) | 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 COL17A1 cDNA FOR RESTORATION OF EPIDERMIS IN PATIENTS WITH JUNCTIONAL EPIDERMOLYSIS BULLOSA - HOLOGENE 17 | JEB is genetically and clinically heterogeneous characterized by mutations in COL17A1,encoding for Collagen17 protein.COLXVII is a collagenous transmembrane type II protein component of the hemidesmosomes and plays a key role in the adhesion of epidermis to the basement membrane. JEB is characterized by a wide range of severity: skin blistering, which can be generalized or localized, different degrees of mucosal involvement, enamel defects, dystrophy or loss of nails, and alopecia can occur.;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: HOLOGENE17 Product Code: HOLOGENE17 INN or Proposed INN: Transduced keratinocytes suspension, containing epidermal stem cells genetically corrected with a COL17A1-encoding retroviral vector | Holostem Terapie Avanzate s.r.l. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | Phase 1;Phase 2 | Austria |