Sham ( DrugBank: - )


3 diseases
告示番号疾患名(ページ内リンク)臨床試験数
90網膜色素変性症7
211左心低形成症候群1
303アッシャー症候群4

90. 網膜色素変性症


臨床試験数 : 130 薬物数 : 180 - (DrugBank : 41) / 標的遺伝子数 : 49 - 標的パスウェイ数 : 109
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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 TherapeuticsNULLRecruiting12 YearsN/AAll120Phase 2/Phase 3United States;United Kingdom
2NCT05158296
(ClinicalTrials.gov)
December 8, 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 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 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 TherapeuticsNULLRecruiting12 YearsN/AAll81Phase 2/Phase 3United States;United Kingdom
3NCT04945772
(ClinicalTrials.gov)
July 13, 202115/6/2021Efficacy and Safety of of vMCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]A Phase 2b Randomized, Double-Masked, Sham-Controlled, Study to Evaluate the Efficacy and Safety of Intravitreal Injection of vMCO-010 Optogenetic Therapy in Adults With Retinitis Pigmentosa [RESTORE]Retinitis Pigmentosa;Retinitis;Retinal Diseases;Eye Diseases;Eye Diseases, Hereditary;Retinal Dystrophies;Retinal DegenerationBiological: Gene Therapy Product-vMCO-010;Procedure: Sham InjectionNanoscope Therapeutics Inc.NULLActive, not recruiting18 YearsN/AAll27Phase 2United States;Puerto Rico
4NCT04123626
(ClinicalTrials.gov)
October 7, 20191/10/2019A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO GeneA Prospective First-In-Human Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa (adRP) Due to the P23H Mutation in the RHO GeneAutosomal Dominant Retinitis Pigmentosa;Eye Diseases;Eye Diseases, Hereditary;Retinal Dystrophies;Retinal Disease;Retinitis;Vision Tunnel;Vision DisordersDrug: QR-1123;Other: Sham procedureProQR TherapeuticsNULLRecruiting18 YearsN/AAll35Phase 1/Phase 2United States
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 TherapeuticsNULLActive, not recruiting18 YearsN/AAll20Phase 1/Phase 2United States;Canada;France;Belgium
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
7NCT00661479
(ClinicalTrials.gov)
July 200829/10/2007An Exploratory Study to Evaluate the Safety of Brimonidine Intravitreal Implant in Patients With Retinitis PigmentosaRetinitis PigmentosaDrug: 400 µg Brimonidine Tartrate Implant;Drug: 200 µg Brimonidine Tartrate Implant;Drug: 100 µg Brimonidine Tartrate Implant;Other: Sham (no implant)AllerganNULLCompleted18 YearsN/AAll21Phase 1/Phase 2United States;France;Germany;Portugal

211. 左心低形成症候群


臨床試験数 : 21 薬物数 : 29 - (DrugBank : 9) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 14
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04181255
(ClinicalTrials.gov)
February 25, 202026/11/2019Cold Heart Study: A Randomized Pilot Trial of Surfactant TherapyThe Cold Heart Study: A Randomized Pilot Trial of Surfactant Therapy Following Deep Hypothermic Circulatory Arrest During Cardiac SurgeryCongenital Heart Disease;Hypoplastic Left Heart SyndromeDrug: Curosurf;Drug: ShamUniversity of MichiganChiesi Farmaceutici S.p.A.RecruitingN/A12 WeeksAll60Phase 1United States

303. アッシャー症候群


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.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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 TherapeuticsNULLRecruiting12 YearsN/AAll120Phase 2/Phase 3United States;United Kingdom
2NCT05158296
(ClinicalTrials.gov)
December 8, 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 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 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 TherapeuticsNULLRecruiting12 YearsN/AAll81Phase 2/Phase 3United States;United Kingdom
3NCT03780257
(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 TherapeuticsNULLActive, not recruiting18 YearsN/AAll20Phase 1/Phase 2United States;Canada;France;Belgium
4NCT01530659
(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