QR-421a ( DrugBank: - )
2 diseases
| 告示番号 | 疾患名（ページ内リンク） | 臨床試験数 |
|---|---|---|
| 90 | 網膜色素変性症 | 11 |
| 303 | アッシャー症候群 | 4 |
90. 網膜色素変性症
臨床試験数 : 130 / 薬物数 : 180 - (DrugBank : 41) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 109
| 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 | NCT05176717 (ClinicalTrials.gov) | December 15, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 120 | Phase 2/Phase 3 | United States;United Kingdom |
| 2 | NCT05158296 (ClinicalTrials.gov) | December 8, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Advanced Vision Loss (Sirius) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Advanced Vision Loss | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 81 | Phase 2/Phase 3 | United States;United Kingdom |
| 3 | NCT05085964 (ClinicalTrials.gov) | September 13, 2021 | 7/10/2021 | An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa | An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia) | Retinitis Pigmentosa;Usher Syndrome Type 2 | Drug: RNA antisense oligonucleotide for intravitreal injection | ProQR Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | All | 20 | Phase 2 | United States;Canada;France |
| 4 | NCT03780257 (ClinicalTrials.gov) | March 6, 2019 | 17/12/2018 | Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only) | ProQR Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States;Canada;France;Belgium |
| 5 | EUCTR2018-002433-38-FR (EUCTR) | 15/02/2019 | 16/11/2018 | Study to evaluate QR-421a in subjects with retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A Gene | A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene - STELLAR | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 18 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Canada;Belgium | ||
| 6 | EUCTR2021-002728-19-DK (EUCTR) | 26/11/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | France;United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom | |||
| 7 | EUCTR2021-002729-74-DK (EUCTR) | 26/11/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom | |||
| 8 | EUCTR2021-002728-19-FR (EUCTR) | 23/09/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;France;European Union;Canada;Brazil;Netherlands;Norway;Germany;United Kingdom | |||
| 9 | EUCTR2021-002728-19-NL (EUCTR) | 15/01/2022 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision Loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Early to Moderate Vision Loss - Celeste | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 120 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom | |||
| 10 | EUCTR2021-002729-74-NL (EUCTR) | 15/01/2022 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom | |||
| 11 | EUCTR2021-002729-74-NO (EUCTR) | 14/10/2021 | A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision loss | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss - Sirius | Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11] | Product Name: QR-421a Product Code: QR-421a INN or Proposed INN: Not available Other descriptive name: QR-421a | ProQR Therapeutics IV B.V. | NULL | NA | Female: yes Male: yes | 81 | Phase 2;Phase 3 | United States;European Union;Canada;Brazil;Germany;Norway;United Kingdom |
303. アッシャー症候群
臨床試験数 : 9 / 薬物数 : 13 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0
| 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 | NCT05176717 (ClinicalTrials.gov) | December 15, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss (Celeste) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Early to Moderate Vision Loss | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 120 | Phase 2/Phase 3 | United States;United Kingdom |
| 2 | NCT05158296 (ClinicalTrials.gov) | December 8, 2021 | 23/11/2021 | Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene With Advanced Vision Loss (Sirius) | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene With Advanced Vision Loss | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Recruiting | 12 Years | N/A | All | 81 | Phase 2/Phase 3 | United States;United Kingdom |
| 3 | NCT05085964 (ClinicalTrials.gov) | September 13, 2021 | 7/10/2021 | An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa | An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Helia) | Retinitis Pigmentosa;Usher Syndrome Type 2 | Drug: RNA antisense oligonucleotide for intravitreal injection | ProQR Therapeutics | NULL | Enrolling by invitation | 18 Years | N/A | All | 20 | Phase 2 | United States;Canada;France |
| 4 | NCT03780257 (ClinicalTrials.gov) | March 6, 2019 | 17/12/2018 | Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders | Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only) | ProQR Therapeutics | NULL | Active, not recruiting | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States;Canada;France;Belgium |