303. アッシャー症候群
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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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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gender
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size
PhaseCountries
1NCT04355689
(ClinicalTrials.gov)
September 3, 202017/4/2020Safety and Efficacy of NPI-001 Tablets for RP Associated With Usher SyndromeSafety and Efficacy of NPI-001 Tablets Versus Placebo for Treatment of Retinitis Pigmentosa Associated With Usher SyndromeUsher SyndromesDrug: NPI-001;Other: PlaceboNacuity Pharmaceuticals, Inc.Foundation Fighting BlindnessRecruiting18 YearsN/AAll48Phase 1;Phase 2Australia
2NCT03780257
(ClinicalTrials.gov)
March 6, 201917/12/2018Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A GeneA 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 GeneRetinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: QR-421a;Other: Sham-procedure (dose cohort 1&2 only)ProQR TherapeuticsNULLRecruiting18 YearsN/AAll18Phase 1;Phase 2United States;Belgium;Canada;France
3EUCTR2012-002574-31-FR
(EUCTR)
25/10/201315/05/2014Study of SAR421869 in Patients With Retinitis Pigmentosa associated with Usher Syndrome Type 1BA 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 & développementNULLNot Recruiting Female: yes
Male: yes
27Phase 1;Phase 2United States;France
4NCT02065011
(ClinicalTrials.gov)
September 12, 201314/2/2014A Study to Determine the Long-Term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1BAn Open-label Study to Determine the Long-term Safety, Tolerability and Biological Activity of SAR421869 in Patients With Usher Syndrome Type 1BUsher's SyndromeDrug: Blood draw for the laboratory assessmentSanofiNULLActive, not recruiting18 YearsN/AAll9Phase 1;Phase 2United States;France
5NCT01505062
(ClinicalTrials.gov)
March 26, 20124/1/2012Study of SAR421869 in Participants With Retinitis Pigmentosa Associated With Usher Syndrome Type 1BA Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR421869, Administered to Patients With Retinitis Pigmentosa Associated With Usher Syndrome Type 1BUsher Syndrome;Retinitis PigmentosaDrug: SAR421869SanofiNULLTerminated6 YearsN/AAll9Phase 1;Phase 2United States;France
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT01530659
(ClinicalTrials.gov)
January 201219/1/2012Retinal Imaging of Subjects Implanted With Ciliary Neurotrophic Factor (CNTF)-Releasing Encapsulated Cell Implant for Early-stage Retinitis PigmentosaPhotoreceptor 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 DensityRetinitis Pigmentosa;Usher Syndrome Type 2;Usher Syndrome Type 3Drug: NT-501;Procedure: ShamNeurotech PharmaceuticalsUniversity of California, San Francisco;FDA Office of Orphan Products DevelopmentUnknown status18 Years55 YearsAll30Phase 2United States