303. アッシャー症候群 Usher syndrome Clinical trials / Disease details
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 | 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 |
| 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 | 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 |
| 6 | 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] | 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 | sanofi-aventis recherche & développement | NULL | Not Recruiting | Female: yes Male: yes | 27 | Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France | ||
| 7 | 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 |
| 8 | 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 |
| 9 | 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 |