303. Usher syndrome Clinical trials / Disease details
Clinical trials : 10 / Drugs : 11 - (DrugBank : 0) / Drug target gene : 0 - Drug target pathway : 0
Showing 1 to 10 of 10 diseases
| 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) Study to Evaluate the Efficacy Safety and Tolerability of QR-421a in Subjects With RP Due to Mutatio ... | 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 A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and To ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders Retinitis Pigmentosa;Usher SyndromeType 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Here ... | Drug: QR-421a;Other: Sham-procedure | ProQR Therapeutics | NULL | Active, not recruiting | 12 Years | N/A | All | 5 | 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 Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius) Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Muta ... | A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius) A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and To ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders Retinitis Pigmentosa;Usher SyndromeType 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Here ... | Drug: Ultevursen;Other: Sham-procedure | ProQR Therapeutics | NULL | Active, not recruiting | 12 Years | N/A | All | 81 | Phase 2/Phase 3 | United States;Germany;Netherlands;United Kingdom |
| 3 | NCT05085964 (ClinicalTrials.gov) | September 16, 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 Safety & Tolerability of QR-421a in Subjects With Retiniti ... | 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) An Open-Label Extension Study to Evaluate the Safety and Tolerability of QR 421a in Subjects With Re ... | Retinitis Pigmentosa;Usher Syndrome Type 2 | Drug: RNA antisense oligonucleotide for intravitreal injection | ProQR Therapeutics | NULL | Terminated | 12 Years | N/A | All | 21 | Phase 2 | United States;Canada;France |
| 4 | NCT04355689 (ClinicalTrials.gov) | September 3, 2020 | 17/4/2020 | Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher Syndrome | Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher Syndrome Safety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associat ... | Usher Syndromes | Drug: NPI-001;Other: Placebo | Nacuity Pharmaceuticals, Inc. | Foundation Fighting Blindness | Recruiting | 18 Years | N/A | All | 48 | Phase 1/Phase 2 | Australia |
| 5 | 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 Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 ... | 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 A First-in-Human Study to Evaluate the Safety and Tolerability of QR-421a in Subjects With Retinitis ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision Disorders Retinitis Pigmentosa;Usher SyndromeType 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Here ... | Drug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only) | ProQR Therapeutics | NULL | Completed | 18 Years | N/A | All | 20 | Phase 1/Phase 2 | United States;Canada;France;Belgium |
| 6 | EUCTR2017-001139-38-PT (EUCTR) | 12/02/2018 | 15/11/2017 | MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC (Locoregionally Advanced Head and Neck Squamous Cell Carcinoma) MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC (Loco ... | A Phase III, Randomized, Open-label Study to Evaluate Pembrolizumab as Neoadjuvant Therapy and in Combination With Standard of Care as Adjuvant Therapy for StageIII-IVA Resectable Locoregionally Advanced Head and Neck Squamous Cell Carcinoma (LA HNSCC) - MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC A Phase III, Randomized, Open-label Study to Evaluate Pembrolizumab as Neoadjuvant Therapy and in Co ... | Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B. MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10063396;Term: Usher's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Retinitis Pigmentosa, associated with Ushers SyndromeType 1B. MedDRA version: 20.0;Level: PT;Classif ... | Product Name: UshStat (SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed INN: Lentiviral vector containing the human MY07A gene Other descriptive name: UshStat Product Name: UshStat(SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed ... | Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc. | NULL | Not Recruiting | Female: yes Male: yes | 704 | Phase 1;Phase 2 | United States;France | ||
| 7 | EUCTR2012-002574-31-FR (EUCTR) | 25/10/2013 | 15/05/2014 | Study of SAR421869 in Patients With Retinitis Pigmentosa associated with Usher Syndrome Type 1B | A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients with Retinitis Pigmentosa Associated with Usher Syndrome Type 1B A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patie ... | Retinitis Pigmentosa, associated with Ushers Syndrome Type 1B. MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders MedDRA version: 20.0;Classification code 10063396;Term: Usher's syndrome;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] Retinitis Pigmentosa, associated with Ushers SyndromeType 1B. MedDRA version: 20.0;Level: PT;Classif ... | Product Name: UshStat (SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed INN: Lentiviral vector containing the human MY07A gene Other descriptive name: UshStat Product Name: UshStat(SAR421869) Product Code: Lentiviral vector containing MYO7A gene INN or Proposed ... | sanofi-aventis recherche & développement | NULL | Not Recruiting | Female: yes Male: yes | 27 | Phase 1;Phase 2 | United States;France | ||
| 8 | NCT02065011 (ClinicalTrials.gov) | September 12, 2013 | 14/2/2014 | A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Pati ... | An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR42 ... | Usher's Syndrome | Drug: Blood draw for the laboratory assessment | Sanofi | NULL | Active, not recruiting | 18 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;France |
| 9 | NCT01505062 (ClinicalTrials.gov) | March 26, 2012 | 4/1/2012 | Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B | A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1B A Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patie ... | Usher Syndrome;Retinitis Pigmentosa | Drug: SAR421869 | Sanofi | NULL | Terminated | 6 Years | N/A | All | 9 | Phase 1/Phase 2 | United States;France |
| 10 | NCT01530659 (ClinicalTrials.gov) | January 2012 | 19/1/2012 | Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retinitis Pigmentosa Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients for Early-stage Retini ... | Photoreceptor Structure in A Phase 2 Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Rates of Change in Cone Spacing and Density Photoreceptor Structure in A Phase 2 Study of Encapsulated Human NTC-201 Cell Implants Releasing Cil ... | Retinitis Pigmentosa;Usher Syndrome Type 2;Usher Syndrome Type 3 | Drug: NT-501;Procedure: Sham | Neurotech Pharmaceuticals | University of California, San Francisco | Completed | 18 Years | 55 Years | All | 22 | Phase 2 | United States |