303. アッシャー症候群 Usher syndrome Clinical trials / Disease details


臨床試験数 : 10 薬物数 : 11 - (DrugBank : 0) / 標的遺伝子数 : 0 - 標的パスウェイ数 : 0

  
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1NCT05176717
(ClinicalTrials.gov)
December 15, 202123/11/2021Study 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 LossRetinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: QR-421a;Other: Sham-procedureProQR TherapeuticsNULLActive, not recruiting12 YearsN/AAll5Phase 2/Phase 3United States;United Kingdom
2NCT05158296
(ClinicalTrials.gov)
December 8, 202123/11/2021Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With 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 Tolerability of Ultevursen in Subjects With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene (Sirius)Retinitis Pigmentosa;Usher Syndrome Type 2;Deaf Blind;Retinal Disease;Eye Diseases;Eye Diseases, Hereditary;Eye Disorders Congenital;Vision DisordersDrug: Ultevursen;Other: Sham-procedureProQR TherapeuticsNULLActive, not recruiting12 YearsN/AAll81Phase 2/Phase 3United States;Germany;Netherlands;United Kingdom
3NCT05085964
(ClinicalTrials.gov)
September 16, 20217/10/2021An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis PigmentosaAn 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 2Drug: RNA antisense oligonucleotide for intravitreal injectionProQR TherapeuticsNULLTerminated12 YearsN/AAll21Phase 2United States;Canada;France
4NCT04355689
(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
5NCT03780257
(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 TherapeuticsNULLCompleted18 YearsN/AAll20Phase 1/Phase 2United States;Canada;France;Belgium
6EUCTR2017-001139-38-PT
(EUCTR)
12/02/201815/11/2017MK-3475 (SCH 9000475) as neoadjuvant and adjuvant therapy in Stage III-IVA resectable LA HNSCC (Locoregionally Advanced Head and Neck Squamous Cell Carcinoma)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 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
Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.NULLNot RecruitingFemale: yes
Male: yes
704Phase 1;Phase 2United States;France
7EUCTR2012-002574-31-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]
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 & développementNULLNot RecruitingFemale: yes
Male: yes
27Phase 1;Phase 2United States;France
8NCT02065011
(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
9NCT01505062
(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
10NCT01530659
(ClinicalTrials.gov)
January 201219/1/2012Retinal Imaging in CNTF -Releasing Encapsulated Cell Implant Treated Patients 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 FranciscoCompleted18 Years55 YearsAll22Phase 2United States