303. アッシャー症候群 ［臨床試験数：3，薬物数：3（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 of the data. Export date: 11/20/2019, 11/21/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03780257 | March 6, 2019 | 4 November 2019 | 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 | ProQR Therapeutics | Recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | United States;Belgium;Canada;France | |
| 2 | NCT02065011 | September 12, 2013 | 14 October 2019 | A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B | An Open-Label Study to Determine the Long-Term Safety, Tolerability and Biological Activity of UshStat® in Patients With Usher Syndrome Type 1B | Usher Syndrome | Drug: UshStat | Sanofi | Not recruiting | 18 Years | N/A | All | 18 | Phase 1/Phase 2 | United States;France | |
| 3 | NCT01530659 | January 2012 | 10 December 2018 | 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 | Not recruiting | 18 Years | 55 Years | All | 30 | Phase 2 | United States |