303. アッシャー症候群
［臨床試験数：6，薬物数：11（DrugBank：0），標的遺伝子数：0，標的パスウェイ数：0］
Searched query = "Usher 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 | 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 | 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 |
| 2 | 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 | Recruiting | 18 Years | N/A | All | 18 | Phase 1;Phase 2 | United States;Belgium;Canada;France |
| 3 | 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 | 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] | sanofi-aventis recherche & développement | NULL | Not Recruiting | Female: yes Male: yes | 27 | Phase 1;Phase 2 | United States;France | |||
| 4 | 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 | An Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1B | 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 |
| 5 | 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 | Usher Syndrome;Retinitis Pigmentosa | Drug: SAR421869 | Sanofi | NULL | Terminated | 6 Years | N/A | All | 9 | Phase 1;Phase 2 | United States;France |
| 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 |
| 6 | NCT01530659 (ClinicalTrials.gov) | January 2012 | 19/1/2012 | Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis Pigmentosa | 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 | Retinitis Pigmentosa;Usher Syndrome Type 2;Usher Syndrome Type 3 | Drug: NT-501;Procedure: Sham | Neurotech Pharmaceuticals | University of California, San Francisco;FDA Office of Orphan Products Development | Unknown status | 18 Years | 55 Years | All | 30 | Phase 2 | United States |