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
| 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 | EUCTR2017-004806-17-DE (EUCTR) | 27/09/2018 | 08/02/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS | Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 INN or Proposed INN: unavailable | Wings Therapeutics Inc. | NULL | Not Recruiting | Female: yes Male: yes | 8 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Czech Republic;Spain;Germany;United Kingdom | ||
| 2 | EUCTR2017-004806-17-ES (EUCTR) | 06/09/2018 | 11/04/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS | Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 INN or Proposed INN: unavailable | ProQR Theraputics | NULL | Not Recruiting | Female: yes Male: yes | 8 | Phase 1;Phase 2 | France;United States;Czech Republic;Canada;Spain;Austria;Germany;United Kingdom | ||
| 3 | NCT03605069 (ClinicalTrials.gov) | July 2, 2018 | 25/6/2018 | A Double-blind, Randomized, Intra-subject Placebo-controlled, Multicenter, Multiple Dose Study, Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Confirmed DDEB or RDEB Diagnosis With One or More Pathogenic Mutations in Exon 73 in the COL7A1 Gene | A First in Human, Double-blind, Randomized, Intra-subject Placebo-controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With DDEB or RDEB Due to Mutation(s) in Exon 73 of the COL7A1 Gene | Epidermolysis Bullosa Dystrophica, Recessive;Epidermolysis Bullosa Dystrophica, Dominant | Drug: QR-313;Drug: Placebo | Phoenicis Therapeutics | NULL | Terminated | 4 Years | N/A | All | 2 | Phase 1/Phase 2 | United States;France;Spain |
| 4 | EUCTR2017-004806-17-GB (EUCTR) | 18/05/2018 | 24/01/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS | Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 INN or Proposed INN: unavailable | Wings Therapeutics Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 8 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | France;United States;Czech Republic;Spain;Germany;United Kingdom | ||
| 5 | EUCTR2017-004806-17-FR (EUCTR) | 05/02/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS | Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 INN or Proposed INN: unavailable | ProQR Therapeutics | NULL | Not Recruiting | Female: yes Male: yes | 14 | Phase 1;Phase 2 | United States;France;Czech Republic;Canada;Spain;Austria;Germany;United Kingdom | |||
| 6 | EUCTR2017-004806-17-CZ (EUCTR) | 21/03/2018 | Study to Evaluate QR-313 in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene | A first in human, double-blind, randomized, intra-subject placebo-controlled, multiple dose study of QR-313 evaluating safety, proof of mechanism, preliminary efficacy and systemic exposure in subjects with Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene - WINGS | Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to mutation(s) in exon 73 of the COL7A1 gene MedDRA version: 20.0;Level: PT;Classification code 10014989;Term: Epidermolysis bullosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Skin and Connective Tissue Diseases [C17] | Product Name: QR-313 Gel for Topical (Cutaneous) Administration Product Code: QR-313 INN or Proposed INN: unavailable | Wings Therapeutics Inc. | NULL | NA | Female: yes Male: yes | 8 | Phase 1;Phase 2 | France;United States;Czech Republic;Spain;Germany;United Kingdom |