90. 網膜色素変性症 Retinitis pigmentosa Clinical trials / Disease details


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

  
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PhaseCountries
1NCT05282953
(ClinicalTrials.gov)
August 20228/3/2022A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patient s With Retinitis Pigmentosa (ABACUS)A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of Small Molecule KIO-301 Administered Intravitreally to Patient s With Retinitis Pigmentosa (ABACUS)Retinitis PigmentosaDrug: KIO-301Kiora Pharmaceuticals, Inc.NULLNot yet recruiting18 Years80 YearsAll6Phase 1NULL
2EUCTR2020-002873-88-NL
(EUCTR)
14/03/202222/10/2021Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 3United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Netherlands;Germany
3EUCTR2020-002255-37-NL
(EUCTR)
14/03/202222/10/2021Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-hRKp.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 3Switzerland;United Kingdom;France;United States;Spain;Ireland;Israel;Italy;Canada;Belgium;Denmark;Netherlands;Germany
4EUCTR2020-002873-88-DK
(EUCTR)
01/03/202208/11/2021Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-HRKP.RPGR
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-HRKP.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 3United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands
5EUCTR2020-002255-37-DK
(EUCTR)
01/03/202208/11/2021Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene - Long Term Follow-Up study of Gene Therapy Trial for Patients with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-HRKP.RPGR
Product Name: AAV5-hRKp.RPGR
INN or Proposed INN: botaretigene sparoparvovec
Other descriptive name: AAV5-HRKP.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
66Phase 3United States;Spain;Ireland;Israel;Italy;Switzerland;United Kingdom;France;Canada;Belgium;Denmark;Germany;Netherlands
6NCT05203939
(ClinicalTrials.gov)
January 24, 202216/11/2021The Study to Assess the Safety and Efficacy of OCU400 for Retinitis PigmentosaA Phase 1/2 Study to Assess the Safety and Efficacy of OCU400 for Retinitis Pigmentosa Associated With NR2E3 (Nuclear Receptor Subfamily 2 Group E Member 3) and RHO (Rhodopsin) MutationsRetinitis PigmentosaDrug: OCU400 Low Dose;Drug: OCU400 Mid Dose;Drug: OCU400 High DoseOcugenNULLRecruiting18 YearsN/AAll18Phase 1/Phase 2United States
7NCT05147701
(ClinicalTrials.gov)
January 202229/11/2021Safety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for Eye DiseasesSafety of Cultured Allogeneic Adult Umbilical Cord Derived Mesenchymal Stem Cells for the Treatment of Eye DiseasesEye Diseases;Retinitis Pigmentosa;Glaucoma;Diabetic Retinopathy;Macular Degeneration;Traumatic Optic Neuropathy;Optic AtrophyBiological: AlloRxThe Foundation for Orthopaedics and Regenerative MedicineNULLRecruitingN/AN/AAll20Phase 1Antigua and Barbuda
8NCT05176717
(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
9NCT05158296
(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
10NCT05085964
(ClinicalTrials.gov)
September 13, 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 TherapeuticsNULLEnrolling by invitation18 YearsN/AAll20Phase 2United States;Canada;France
11NCT04850118
(ClinicalTrials.gov)
August 20215/4/2021A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With X-linked Retinitis Pigmentosa Caused by RPGR MutationsA Phase 2/3, Randomized, Controlled, Masked, Multi-center Study to Evaluate the Efficacy, Safety and Tolerability of Two Doses of AGTC-501, a Recombinant Adeno-associated Virus Vector Expressing RPGR (rAAV2tYF-GRK1-RPGR), Compared to an Untreated Control Group in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR GeneX-Linked Retinitis PigmentosaBiological: rAAV2tYF-GRK1-hRPGRcoApplied Genetic Technologies CorpNULLNot yet recruiting8 Years50 YearsMale63Phase 2/Phase 3Israel;Netherlands;United States
12NCT04284293
(ClinicalTrials.gov)
July 22, 202118/2/2020CNS10-NPC for the Treatment of RPClinical Study to Assess Safety and Efficacy of Subretinal Injection of Human Neural Progenitor Cells for Treatment of Retinitis PigmentosaRetinitis PigmentosaBiological: CNS10-NPC implantationCedars-Sinai Medical CenterCalifornia Institute for Regenerative Medicine (CIRM)Recruiting18 YearsN/AAll16Phase 1United States
13NCT04945772
(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
14NCT04925687
(ClinicalTrials.gov)
June 1, 20212/6/2021Pilot Study of Intravitreal Autologous CD34+ Stem Cell Therapy for Retinitis PigmentosaA Pilot Study of the Feasibility and Safety of Intravitreal Autologous Bone Marrow CD34+ Stem Cell Therapy for Eyes With Vision Loss From Retinitis PigmentosaRetinitis PigmentosaBiological: Intravitreal autologous CD34+ cellsUniversity of California, DavisCures Within ReachRecruiting18 YearsN/AAll4Phase 1United States
15NCT04864496
(ClinicalTrials.gov)
April 17, 202121/4/2021Effects of Treatment With N- Acetylcysteine on Visual Outcomes in Patients With Retinitis PigmentosaEffects of Oral N- Acetylcysteine on Macular Function in Retinitis Pigmentosa; a Phase 2 Randomized Controlled TrialRetinitis PigmentosaDrug: Prescribe N-acetylcysteine tablets;Drug: Prescribe placebo tabletsShahid Beheshti University of Medical SciencesNULLActive, not recruiting18 Years40 YearsAll30Phase 2Iran, Islamic Republic of
16NCT04794101
(ClinicalTrials.gov)
March 16, 202115/12/2020Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneFollow-up Phase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneX-Linked Retinitis PigmentosaBiological: Genetic: AAV5-RPGR 4e11;Biological: Genetic: AAV5-RPGR 2e11MeiraGTx UK II LtdJanssen Research & Development, LLCRecruiting3 YearsN/AAll66Phase 3United States
17NCT04671433
(ClinicalTrials.gov)
March 16, 20215/11/2020Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GenePhase 3 Randomized, Controlled Study of AAV5-RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR GeneX-Linked Retinitis PigmentosaBiological: Genetic: AAV5-RPGRMeiraGTx UK II LtdJanssen Research & Development, LLCRecruiting3 YearsN/AAll66Phase 3United States
18NCT02018692
(ClinicalTrials.gov)
March 1, 202117/12/2013The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis PigmentosaThe Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Adolescent Patients With Retinitis PigmentosaRetinitis PigmentosaDietary Supplement: Alga Dunaliella Bardawil powder;Dietary Supplement: Placebo:StarchSheba Medical CenterNULLNot yet recruiting12 Years18 YearsAll30Phase 1/Phase 2Israel
19EUCTR2017-003104-42-DK
(EUCTR)
08/02/202101/07/2020A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2- REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent studyA Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)
MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2-REP1
Product Code: AAV2-REP1
INN or Proposed INN: timrepigene emparvovec
Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
INN or Proposed INN: Cotoretigene Toliparvovec
Other descriptive name: AAV8-RPGR
NightstaRx Ltd (A Biogen Company)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3Netherlands;Germany;United Kingdom;France;United States;Canada;Finland;Brazil;Denmark
20NCT04763369
(ClinicalTrials.gov)
February 20216/2/2021Investigation of Therapeutic Efficacy and Safety of UMSCs for the Management of Retinitis Pigmentosa (RP)Investigation of Therapeutic Efficacy and Safety of Umbilical Cord Derived Mesenchymal Stem Cells (UMSCs) for the Management of Retinitis Pigmentosa (RP)Retinitis Pigmentosa (RP)Biological: Injection of stem cells in sub-tenon space of eye for the management of retinitis pigmentosa;Biological: Injection of stem cells in suprachoroidal space of eye for the management of Retinitis PigmentosaJinnah Burn and Reconstructive Surgery Centre, LahoreThe Layton Rahmatullah Benevolent Trust (LRBT) Free Eye Hospital, Township Lahore.;Centre of Excellence in Molecular Biology (CEMB), University of the Punjab, Lahore.Recruiting18 Years70 YearsAll50Phase 2Pakistan
21NCT04636853
(ClinicalTrials.gov)
December 23, 202014/11/2020CB-PRP in Retinitis PigmentosaCord Blood Platelet-rich Plasma (CB-PRP) in Retinitis PigmentosaRetinitis PigmentosaBiological: Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP)Fondazione Policlinico Universitario Agostino Gemelli IRCCSNULLCompleted18 Years70 YearsAll20Phase 3Italy
22NCT04604899
(ClinicalTrials.gov)
December 1, 202016/10/2020Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis PigmentosaA Phase 2 Study of the Safety of Repeat Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)Retinitis PigmentosaBiological: human retinal progenitor cellsjCyte, IncCalifornia Institute for Regenerative Medicine (CIRM)Active, not recruiting18 YearsN/AAll30Phase 2United States
23NCT04355689
(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
24NCT04517149
(ClinicalTrials.gov)
June 9, 202014/8/20204D-125 in Patients With X-Linked Retinitis Pigmentosa (XLRP)An Open-label, Phase 1/2 Trial of Gene Therapy 4D-125 in Males With X-linked Retinitis Pigmentosa (XLRP) Caused by Mutations in the RPGR GeneX-Linked Retinitis PigmentosaBiological: 4D-125 IVT Injection;Other: Observational4D Molecular TherapeuticsNULLRecruiting18 YearsN/AMale43Phase 1/Phase 2United States
25EUCTR2019-004547-77-GB
(EUCTR)
09/06/202026/02/2020Safety and Tolerability of hRPC in Retinitis PigmentosaFirst-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Subjects with Retinitis Pigmentosa (RP) Retinitis Pigmentosa
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: human Retinal Progenitor Cells Drug Product
Product Code: hRPC DP
INN or Proposed INN: ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED
Other descriptive name: ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED
ReNeuron LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
33Phase 2United States;United Kingdom
26NCT03944239
(ClinicalTrials.gov)
May 202027/12/2018Safety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis PigmentosaSafety and Efficacy of Subretinal Transplantation of Clinical Human Embryonic Stem Cell Derived Retinal Pigment Epitheliums in Treatment of Retinitis PigmentosaRetinitis PigmentosaBiological: Retinal pigment epitheliums transplantationQi ZhouBeijing Tongren HospitalRecruiting18 Years80 YearsAll10Phase 1China
27NCT04120883
(ClinicalTrials.gov)
February 25, 20208/10/2019Oral Hydroxychloroquine (HCQ) for Retinitis Pigmentosa Caused by P23H- Rhodopsin (RHO)Oral Hydroxychloroquine for Retinitis Pigmentosa Caused by P23H-RHO (Substitution of Proline to Histidine at Codon 23 of the Rhodopsin Protein)Retinitis PigmentosaDrug: Hydroxychloroquine lower dose;Drug: Hydroxychloroquine higher doseUniversity of MichiganCures Within ReachRecruiting18 YearsN/AAll12Phase 1/Phase 2United States
28NCT04278131
(ClinicalTrials.gov)
February 6, 202013/2/2020BS01 in Patients With Retinitis PigmentosaPhase 1/2, Safety and Efficacy Trial of BS01, a Recombinant Adeno-Associated Virus Vector Expressing ChronosFP in Patients With Retinitis PigmentosaRetinitis PigmentosaDrug: BS01Bionic Sight LLCNULLRecruiting18 YearsN/AAll20Phase 1/Phase 2United States
29NCT04919473
(ClinicalTrials.gov)
October 23, 201925/5/2021Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis PigmentosaA Phase I/IIa Open Label, Dose-Escalation Study to Evaluate the Safety and Tolerability of Intravitreal vMCO-I in Patients With Advanced Retinitis PigmentosaRetinitis Pigmentosa;Retinal Diseases;Retinal DegenerationBiological: Gene Therapy product:vMCO-INanoscope Therapeutics Inc.NULLCompleted18 YearsN/AAll11Phase 1/Phase 2India
30NCT04123626
(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
31NCT04611503
(ClinicalTrials.gov)
September 24, 201920/5/2020PDE6A Gene Therapy for Retinitis PigmentosaPIGMENT - PDE6A Gene Therapy for Retinitis PigmentosaRetinitis PigmentosaDrug: subretinal injection of rAAV.hPDE6ASTZ eyetrialNULLRecruiting18 YearsN/AAll9Phase 1/Phase 2Germany
32NCT03963154
(ClinicalTrials.gov)
August 19, 201922/5/2019Interventional Study of Implantation of hESC-derived RPE in Patients With RP Due to Monogenic MutationSTREAM: A Phase 1/2, Open-label, Safety, Tolerability and Preliminary Efficacy Study of Implantation Into One Eye of hESC-derived RPE in Patients With Retinitis Pigmentosa Due to Monogenic MutationRetinitis PigmentosaBiological: Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE)Centre d'Etude des Cellules SouchesNULLRecruiting18 Years65 YearsAll12Phase 1/Phase 2France
33EUCTR2016-003705-34-DE
(EUCTR)
02/07/201926/11/2018Administration of a genetically modified virus particle in patients with PDE6A-linked retinitis pigmentosaPIGMENT – PDE6A gene therapy for retinitis pigmentosa PDE6A-linked retinitis pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11]Product Name: rAAV.hPDE6A vectorUniversitätsklinikum TübingenNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
9 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noGermany
34NCT03999021
(ClinicalTrials.gov)
June 24, 201924/6/2019FIGHT-RP 1 Extension StudyA Phase 1 Open-Label Extension Study to Assess the Long-Term Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis PigmentosaRetinitis PigmentosaDrug: NAC effervescent tabletsJohns Hopkins UniversityNULLActive, not recruiting18 YearsN/AAll30Phase 1United States
35NCT04224207
(ClinicalTrials.gov)
April 1, 20196/1/2020Management of Retinitis Pigmentosa by Mesenchymal Stem Cells by Wharton's Jelly Derived Mesenchymal Stem CellsManagement of Retinitis Pigmentosa by Wharton's Jelly Derived Mesenchymal Stem Cells: Preliminary Clinical ResultsRetinitis Pigmentosa;Inherited Retinal DystrophyBiological: Wharton's jelly derived mesenchymal stem cellAnkara Universitesi TeknokentNULLCompleted18 Years60 YearsAll32Phase 3Turkey
36NCT03780257
(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
37NCT04068207
(ClinicalTrials.gov)
February 22, 201923/8/2019Minocycline Treatment in Retinitis PigmentosaThe Efficacy and Safety of Oral Minocycline in the Treatment of Retinitis Pigmentosa: An Open-label Clinical TrialRetinitis Pigmentosa;Inherited Retinal Dystrophy;Retina DisorderDrug: MinocyclineSun Yat-sen UniversityNULLRecruiting18 Years60 YearsAll35Phase 2China
38NCT04312672
(ClinicalTrials.gov)
February 18, 201915/7/2019Long Term Follow-Up Gene Therapy Study for XLRP RPGRLong Term Follow-Up Study of Participants Following an Open Label, Multi-Centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-Associated Virus Vector (AAV2-RPGR) for Gene Therapy of Adults and Children With X-Linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)X-Linked Retinitis PigmentosaBiological: AAV-RPGRMeiraGTx UK II LtdNULLRecruiting5 YearsN/AMale36Phase 1/2United Kingdom
39EUCTR2018-002433-38-FR
(EUCTR)
15/02/201916/11/2018Study to evaluate QR-421a in subjects with retinitis pigmentosa (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 Gene - STELLAR Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR TherapeuticsNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
18 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Canada;Belgium
40EUCTR2013-005393-22-NL
(EUCTR)
04/01/201920/09/2016A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision.A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes
MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QLT091001
Product Code: QLT091001
INN or Proposed INN: Zuretinol Acetate
Other descriptive name: QLT091001
QLT Inc.NULLNA Female: yes
Male: yes
48 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noFrance;United States;Canada;Brazil;Denmark;Germany;Netherlands;United Kingdom;Switzerland
41NCT03772938
(ClinicalTrials.gov)
December 13, 20183/11/2018Stem Cells Therapy in Degenerative Diseases of the RetinaStem Cells Therapy in Degenerative Diseases of the RetinaRetinal Degeneration;Retinitis Pigmentosa;Age Related Macular Degeneration;Stargardt Disease 1Biological: Stem/progenitor cells transplantationPomeranian Medical University SzczecinNULLUnknown status18 Years65 YearsAll30Phase 1Poland
42EUCTR2017-003104-42-FI
(EUCTR)
20/11/201816/10/2018A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent studyA Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)
MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2-REP1
Product Code: AAV2-REP1
INN or Proposed INN: timrepigene emparvovec
Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
INN or Proposed INN: Cotoretigene Toliparvovec
Other descriptive name: AAV8-RPGR
NightstaRx Ltd (A Biogen Company)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Brazil;Denmark;Netherlands;Germany;United Kingdom
43NCT04315025
(ClinicalTrials.gov)
October 7, 20188/3/2020Safety Issues of Peribulbar Injection of UC-MSC in Patients With Retinitis PigmentosaSafety Issues of Peribulbar Injection of Umbilical Cord Mesenchymal Stem Cell (UC-MSC) in Patients With Retinitis PigmentosaRetinitis PigmentosaBiological: Umbilical Cord Mesenchymal Stem Cell (UC-MSC);Biological: Conditioned Medium (CM)PT. Prodia Stem Cell IndonesiaNULLCompleted18 Years65 YearsAll18Phase 1/Phase 2Indonesia
44NCT03078309
(ClinicalTrials.gov)
September 1, 201810/2/2017The Effects of Cannabis on Visual Functions in Healthy and Retinitis Pigmentosa PatientsA Controlled Study of the Effect of Cannabis on Visual Functions in Healthy Subjects and in Retinitis Pigmentosa PatientsRetinitis Pigmentosa;Retinal Degeneration;CannabisDrug: cannabisHadassah Medical OrganizationHebrew University of JerusalemRecruiting18 Years70 YearsAll50Early Phase 1Israel
45EUCTR2018-000425-31-GB
(EUCTR)
30/08/201814/06/2018Long term follow-up study of gene therapy trial for X-linked Retinitis PigmentosaLong term follow-up study of participants following an open label, multicentre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Long term follow-up gene therapy study for X-linked Retinitis Pigmentosa X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2/5-hRKp.RPGRMeiraGTx UK II LtdNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
36 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
46NCT03374657
(ClinicalTrials.gov)
August 22, 201811/12/2017A First-in-human, Proof of Concept Study of CPK850 in Patients With RLBP1 Retinitis PigmentosaAn Open-label First-in-human Single Ascending Dose Study to Explore Safety, Tolerability and Efficacy of Subretinal Administration of CPK850 Gene Therapy in Patients With Retinitis Pigmentosa Due to Mutations in the Retinaldehyde Binding Protein 1 (RLBP1) GeneRetinitis PigmentosaBiological: CPK850Novartis PharmaceuticalsNULLRecruiting18 Years70 YearsAll15Phase 1/Phase 2Sweden
47NCT03566147
(ClinicalTrials.gov)
August 1, 201812/6/2018Treatment of RP and LCA by Primary RPE TransplantationTreatment of Retinitis Pigmentosa and Leber Congenital Amaurosis by Primary Retinal Pigment Epithelial Cells TransplantationLeber Congenital Amaurosis, Retinitis PigmentosaBiological: Human primary Retinal Pigment Epithelial (HuRPE) cellsEyecure Therapeutics Inc.Beijing Tongren HospitalUnknown status18 Years70 YearsAll30Early Phase 1China
48EUCTR2017-003104-42-GB
(EUCTR)
26/06/201806/02/2018A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 in an antecedent study and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent studyA Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study - SOLSTICE Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)
MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2-REP1
Product Code: AAV2-REP1
INN or Proposed INN: timrepigene emparvovec
Other descriptive name: AAV2-REP1
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
Other descriptive name: AAV8-RPGR
NightstaRx LtdNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom
49NCT03316560
(ClinicalTrials.gov)
April 16, 201810/10/2017Safety and Efficacy of rAAV2tYF-GRK1-RPGR in Subjects With X-linked Retinitis Pigmentosa Caused by RPGR MutationsA Phase 1/2 Open-Label Dose Escalation Study to Evaluate the Safety and Efficacy of AGTC-501 (rAAV2tYF-GRK1-RPGR) and a Phase 2 Randomized, Controlled, Masked, Multi-center Study Comparing Two Doses of AGTC-501 in Male Subjects With X-linked Retinitis Pigmentosa Confirmed by a Pathogenic Variant in the RPGR GeneX-Linked Retinitis PigmentosaBiological: rAAV2tYF-GRK1-RPGRApplied Genetic Technologies CorpNULLRecruiting6 Years50 YearsMale42Phase 1/Phase 2United States
50EUCTR2017-003104-42-DE
(EUCTR)
05/01/201825/09/2017A long-term follow-up study to evaluate the safety and efficacy of retinal gene therapy in subjects with Choroideremia previously treated with AAV2-REP1 and in subjects with X-Linked Retinitis Pigmentosa previously treated with AAV8-RPGR in an antecedent studyA Long-term Follow-up Study to Evaluate the Safety and Efficacy of RetinalGene Therapy in Subjects with Choroideremia Previously Treated with Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects with X-Linked Retinitis Pigmentosa Previously Treated with Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study Choroideremia (CHM)X-Linked Retinitis Pigmentosa (XLRP)
MedDRA version: 20.1;Level: LLT;Classification code 10008791;Term: Choroideremia;System Organ Class: 100000004853
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2-REP1
INN or Proposed INN: timrepigene emparvovec
Other descriptive name: Adeno-associated virus type 2 vector encoding Rab escort protein 1
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
Other descriptive name: AAV8-RPGR
NightstaRx Ltd (A Biogen Company)NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
440Phase 3France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom
51EUCTR2017-002204-27-GB
(EUCTR)
22/12/201720/10/2017A Phase 1/2a gene therapy clinical trial in Retinitis Pigmentosa subjectsA Phase 1/2a, Open-Label, Non-Randomized, Dose-Escalation Study to Evaluate the Safety and Tolerability of GS030 in Subjects with Retinitis Pigmentosa Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11]Product Name: Recombinant adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene
Product Code: GS030-DP
GENSIGHT-BIOLOGICSNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
18 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
52NCT03328130
(ClinicalTrials.gov)
November 6, 20175/10/2017Safety and Efficacy Study in Patients With Retinitis Pigmentosa Due to Mutations in PDE6B GeneSafety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients With Retinitis Pigmentosa Harbouring Mutations in the PDE6B Gene Leading to a Defect in PDE6ß ExpressionRetinitis PigmentosaBiological: AAV2/5-hPDE6BCoave TherapeuticsNULLActive, not recruiting18 YearsN/AAll17Phase 1/Phase 2France
53EUCTR2016-001429-16-FR
(EUCTR)
28/07/201712/09/2017Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression.Safety and Efficacy of a Unilateral Subretinal Administration of HORA-PDE6B in Patients Harboring Mutations in the PDE6B Gene Leading to a Defect in PDE6B Expression. Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11]Product Code: HORA-PDE6B (AAV2/5.hPDE6B)HORAMA SANULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 1;Phase 2France
54EUCTR2016-003967-21-GB
(EUCTR)
09/06/201714/09/2017Gene Therapy Trial for People with Retinitis Pigmentosa (progressive reduction in vision) due to a gene defect on Chromosome X.An open label, multi-centre, Phase I/II dose escalation trial of a recombinant adeno-associated virus vector (AAV2/5-hRKp.RPGR) for gene therapy of adults and children with X-linked Retinitis Pigmentosa owing to defects in Retinitis Pigmentosa GTPase Regulator (RPGR) - Gene Therapy Trial for People with Retinitis Pigmentosa: RPGR X-Linked Retinitis Pigmentosa caused by mutations in the RPGR gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV2/5-hRKp.RPGR
INN or Proposed INN: AAV2/5-hRKp.RPGR
MeiraGTx UK II LimitedNULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
71 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
55NCT03116113
(ClinicalTrials.gov)
March 16, 201729/3/2017A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using BIIB112A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)X-Linked Retinitis PigmentosaBiological: BIIB112NightstaRx Ltd, a Biogen CompanyNULLCompleted10 YearsN/AMale50Phase 1/Phase 2United States;United Kingdom
56NCT03073733
(ClinicalTrials.gov)
March 1, 20172/3/2017Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells in Adults With Retinitis PigmentosaA Prospective, Multicenter, Randomized, Study of the Safety and Efficacy of Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)Retinitis PigmentosaBiological: human retinal progenitor cells;Other: Mock injectionjCyte, IncCalifornia Institute for Regenerative Medicine (CIRM)Completed18 YearsN/AAll84Phase 2United States
57NCT03063021
(ClinicalTrials.gov)
February 15, 201716/2/2017The FIGHT-RP1 StudyA Phase 1 Open Label Dose Ranging Study to Assess the Safety and Tolerability of N-Acetylcysteine (NAC) in Patients With Retinitis Pigmentosa (FIGHT-RP1 Study)Retinitis PigmentosaDrug: N-Acetyl Cysteine (NAC)Johns Hopkins UniversityNULLCompleted18 YearsN/AAll30Phase 1United States
58EUCTR2016-003852-60-GB
(EUCTR)
09/01/201709/11/2016A Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa using AAV8A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR) - XIRIUS Study X-Linked retinitis pigmentosa (XLRP)
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: AAV8-RPGR
Product Code: AAV8-RPGR
Other descriptive name: AAV8-RPGR
NightstaRx LimitedNULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
63Phase 1;Phase 2;Phase 3United States;United Kingdom
59NCT04252534
(ClinicalTrials.gov)
January 1, 201731/1/2020Management of Retinitis Pigmentosa Via Electromagnetic Stimulation and Platelet Rich PlasmaManagement of Retinitis Pigmentosa Via Repetitive Electromagnetic Stimulation and Autologous Platelet Rich PlasmaRetinitis PigmentosaCombination Product: PRP combined Magnovision;Biological: PRPAnkara Universitesi TeknokentNULLCompleted18 Years60 YearsAll60N/ANULL
60EUCTR2013-005393-22-DK
(EUCTR)
03/11/201606/09/2016A study in subjects with rare inherited eye conditions caused by gene mutations to see if treatment with QLT091001 is safe and works to improve subjects' vision.A Study of the Efficacy and Safety of QLT091001 in Subjects with Inherited Retinal Disease (IRD) Caused by Mutation in Retinal Pigment Epithelium Protein 65 (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) Inherited retinal disease (IRD) phenotypically diagnosed as Leber congenital amaurosis (LCA) or retinitis pigmentosa (RP) caused by mutations in the retinal pigment epithelium protein 65 (RPE65) or lecithin:retinol acyltransferase (LRAT) genes
MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 19.0;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QLT091001
Product Code: QLT091001
INN or Proposed INN: Zuretinol Acetate
Other descriptive name: QLT091001
QLT Inc.NULLNot RecruitingFemale: yes
Male: yes
48Phase 3United States;France;Canada;Brazil;Denmark;Netherlands;Germany;United Kingdom;Switzerland
61EUCTR2016-002523-28-GB
(EUCTR)
16/09/201605/07/2016A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa.A Pilot Study Evaluating the Effect of Intravitreal Fluocinolone Acetonide (0.19mg) in Patients with Retinitis Pigmentosa - Iluvien for IRD Retinitis Pigmetosa.
MedDRA version: 19.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Body processes [G] - Genetic Phenomena [G05]
Trade Name: Iluvien
Product Name: Iluvien
Product Code: n/a
INN or Proposed INN: Fluocinolone Acetonide
Moorfields Eye HospitalNULLNot Recruiting Female: yes
Male: yes
20 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
62JPRN-UMIN000023618
2016/09/0101/09/2016Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa Cataract associated with patients with retinitis pigmentosaCataract surgery and implantation of 1P intraocular lens (PCB00V)
Cataract surgery and implantation of 3P intraocular lens (VA-70AD)
Kyushu University HospitalNULLComplete: follow-up complete40years-oldNot applicableMale and Female40Not selectedJapan
63NCT04238858
(ClinicalTrials.gov)
September 1, 201618/1/2020Effects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis PigmentosaEffects of Subtenon-injected Autologous Platelet-rich Plasma on Visual Functions in Eyes With Retinitis PigmentosaRetinitis PigmentosaBiological: Platelet rich plasmaAnkara Universitesi TeknokentNULLCompleted18 Years55 YearsAll48N/ATurkey
64JPRN-UMIN000022686
2016/07/0101/07/2016The Clinical Efficacy of Carbonic Anhydrase Inhibitors and Steroids in the Management of Cystoid Macular Edema in Patients with Retinitis PigmentosaThe Clinical Efficacy of Carbonic Anhydrase Inhibitors and Steroids in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa - The Clinical Efficacy in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa retinitis pigmentosa1% dorzolamide eye drop three times daily in each eye
0.1% betamethasone sodium phosphate eye drop three times daily in each eye
oral 250mg acetazolamide two tablets daily
40mg triamcinolone acetonide into the sub-Tenon capsule
Kyushu University HospitalNULLComplete: follow-up complete20years-oldNot applicableMale and Female40Not applicableJapan
65NCT02837640
(ClinicalTrials.gov)
June 201622/6/2016Studying a Potential Protective Effect of L-Dopa on Retinitis PigmentosaThe Effect of L-Dopa on the Progression of Retinitis PigmentosaRetinitis PigmentosaDrug: levodopa-carbidopaBeirut Eye Specialist HospitalNULLRecruiting10 Years65 YearsBoth50Phase 2Lebanon
66NCT02661711
(ClinicalTrials.gov)
March 201613/1/2016Aflibercept for Macular Oedema With Underlying Retinitis Pigmentosa (AMOUR) StudyProspective Non-randomised Exploratory Study to Assess the Safety and Efficacy of Aflibercept (Eylea) in Cystoid Macular Oedema (CMO) Associated With Retinitis Pigmentosa (RP)Macular Oedema;Retinitis PigmentosaDrug: AfliberceptMoorfields Eye Hospital NHS Foundation TrustBayerActive, not recruiting16 YearsN/ABoth30Phase 2United Kingdom
67EUCTR2015-003723-65-GB
(EUCTR)
12/01/201606/01/2016Prospective non-randomised pilot study looking at the safety and efficacy of a drug called Eylea for patients with an eye condition called Retinitis Pigmentosa, that also have swelling at the back of the eyeAflibercept (Eylea®) for macular oedema associated with underlying Retinitis Pigmentosa (AMOUR) - AMOUR Retinitis Pigmentosa associated Cystoid Macular Oedema
MedDRA version: 18.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Trade Name: Eylea
Product Name: Eylea
Product Code: n/a
INN or Proposed INN: Aflibercept
Moorfields Eye HospitalNULLNot Recruiting Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited Kingdom
68NCT02556736
(ClinicalTrials.gov)
December 14, 201521/9/2015RST-001 Phase I/II Trial for Advanced Retinitis PigmentosaPhase I/IIa, Open-Label, Dose-Escalation Study of Safety and Tolerability of Intravitreal RST-001 in Patients With Advanced Retinitis Pigmentosa (RP)Advanced Retinitis PigmentosaDrug: RST-001AllerganNULLActive, not recruiting18 YearsN/AAll14Phase 1/Phase 2United States
69NCT02464436
(ClinicalTrials.gov)
December 201518/5/2015Safety and Tolerability of hRPC in Retinitis PigmentosaFirst-in-human Phase I/IIa, Open-Label, Prospective Study of the Safety and Tolerability of Subretinally Transplanted Human Retinal Progenitor Cells (hRPC) in Patients With Retinitis Pigmentosa (RP)Retinitis PigmentosaDrug: hRPCReNeuron LimitedNULLActive, not recruiting18 YearsN/AAll29Phase 1/Phase 2United States;Spain;United Kingdom
70NCT02320812
(ClinicalTrials.gov)
June 201516/12/2014Safety of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Retinitis PigmentosaA Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects With Retinitis Pigmentosa (RP)Retinitis Pigmentosa (RP)Biological: human retinal progenitor cellsjCyte, IncCalifornia Institute for Regenerative Medicine (CIRM)Completed18 YearsN/AAll28Phase 1/Phase 2United States
71NCT02609165
(ClinicalTrials.gov)
May 201512/10/2015Nerve Growth Factor Eye Drops Treatment in Patients With Retinitis Pigmentosa and Cystoid Macular EdemaNerve Growth Factor Eye Drops as a Novel Treatment for Vision Loss in Patients With Retinitis Pigmentosa: From Preclinical to Clinical Phase II TrialRetinitis Pigmentosa;Cystoid Macular EdemaDrug: rhNGF 180 µg/ml eye drops solution;Drug: vehicle eye dropsOspedale San RaffaeleDompé Farmaceutici S.p.ACompleted18 YearsN/AAll45Phase 2Italy
72NCT02465749
(ClinicalTrials.gov)
May 201522/5/2015Clinical Trials of Continuous Oxygen Therapy Combined With Blue Light Deprivation in the Treatment of Retinitis PigmentosaClinical Trials of Continuous Oxygen Therapy Combined With Blue Light Deprivation in the Treatment of Retinitis PigmentosaRetinitis PigmentosaDrug: Continuous oxygen;Device: blue light-absorbing sunglasses;Drug: Compound thrombosis capsule sig: 1.5g/tid;Drug: Ginkgo biloba pills sig: 300mg/tid;Drug: Vitamin B sig: 10mg/tid;Drug: Vitamin AD sig: 1 tablet/tidSun Yat-sen UniversityZhujiang Hospital;Sun Yat-Sen Memorial Hospital of Sun Yat-Sen University;Guangzhou Jeeyor Medical Research Co.,Ltd.Not yet recruiting18 Years60 YearsBoth404Phase 0China
73NCT02804360
(ClinicalTrials.gov)
January 201518/5/2016Intravitreal Dexamethasone Implant in Retinitis Pigmentosa-related Macular Edema- a Retrospective StudyDexamethasone in Retinitis Pigmentosa Cystoid Macular EdemaRetinitis Pigmentosa;Cystoid Macular EdemaDevice: dexamethasone injectionRafic Hariri University HospitalNULLEnrolling by invitation16 Years90 YearsBoth50Phase 2NULL
74NCT04356716
(ClinicalTrials.gov)
November 11, 201420/4/2020Sildenafil for Treatment of Choroidal IschemiaSildenafil for Treatment of Choroidal IschemiaChoroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis PigmentosaDrug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)Columbia UniversityNULLRecruiting18 YearsN/AAll25Phase 2United States
75NCT02280135
(ClinicalTrials.gov)
November 201422/10/2014Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis PigmentosaPhase I Clinical Trial of Intravitreal Injection of Autologous Bone Marrow Stem Cells in Patients With Retinitis PigmentosaRetinitis PigmentosaBiological: Intravitreal injection of Autologous bone marrow Stem Cell;Other: Subconjunctival injection of salineRed de Terapia CelularSpanish National Health System;Hospital Universitario Virgen de la Arrixaca;Fundacion para la Formacion e Investigacion Sanitarias de la Region de Murcia;Public Health Service, Murcia;Instituto Murciano de Investigación Biosanitaria Virgen de la ArrixacaCompleted18 Years70 YearsAll8Phase 1Spain
76NCT02244996
(ClinicalTrials.gov)
November 201417/9/2014Cone Rescue in Retinitis Pigmentosa by the Treatment of Lycium BarbarumCone Rescue in Retinitis Pigmentosa by the Treatment of Lycium BarbarumRetinitis PigmentosaDietary Supplement: Lycium Barbarum;Dietary Supplement: PlaceboThe Hong Kong Polytechnic UniversityThe University of Hong KongCompleted18 Years70 YearsAll50N/AHong Kong
77EUCTR2014-000385-22-IT
(EUCTR)
07/10/201412/05/2014Nerve Growth Factor eye drops as a novel treatment for patients with Retinitis Pigmentosa.Nerve Growth Factor eye drops as a novel treatment for vision loss in patients with Retinitis Pigmentosa: from preclinical toclinical Phase II trial. - NEMO Retinitis pigmentosa with cystoid macular edema
MedDRA version: 17.0;Level: LLT;Classification code 10054467;Term: Macular edema;System Organ Class: 100000004853
MedDRA version: 17.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: recombinant human Nerve Growth Factor (rhNGF)
INN or Proposed INN: not yet assigned
Other descriptive name: RECOMBINANT HUMAN NERVE GROWTH FACTOR (RHNGF)
Ospedale San Raffaele di MilanoNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Phase 2Italy
78NCT01914913
(ClinicalTrials.gov)
September 201431/7/2013Clinical Study to Evaluate Safety and Efficacy of BMMNC in Retinitis PigmentosaAn Open Labeled Clinical Study to Evaluate the Safety and Efficacy OF Autologous Bone Marrow Derived Mono Nuclear Stem Cell (BMMNCs) in Retinitis Pigmentosa. It is Self Funded (Patients' Own Funding) Clinical TrialRetinitis PigmentosaBiological: BMMNCsChaitanya Hospital, PuneNULLRecruiting18 Years65 YearsBoth15Phase 1/Phase 2India
79NCT02140164
(ClinicalTrials.gov)
May 201414/5/2014Study of Oral Minocycline in Treating Bilateral Cystoid Macular Edema Associated With Retinitis PigmentosaPilot Study to Evaluate Oral Minocycline in the Treatment of Cystoid Macular Edema Associated With Retinitis PigmentosaRetinitis PigmentosaDrug: MinocyclineNational Eye Institute (NEI)The EMMES CorporationCompleted12 YearsN/AAll7Phase 1/Phase 2United States
80NCT02709876
(ClinicalTrials.gov)
April 201426/2/2016Autologous Bone Marrow-Derived CD34+, CD133+, and CD271+ Stem Cell Transplantation for Retinitis PigmentosaRetinitis PigmentosaBiological: Stem Cell TransplantationStem Cells ArabiaNULLActive, not recruiting18 Years55 YearsAll50Phase 1/Phase 2Jordan
81NCT02110225
(ClinicalTrials.gov)
January 20148/4/2014A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)A 24 Week Phase Ib/II, Multicenter, Randomized, Controlled, Parallel Group, Dose Ranging Study With a 24 Week Follow-up to Evaluate Safety and Potential Efficacy of 2 Doses (60, 180 µg/ml) of rhNGF Solution vs Vehicle in Patients With RP.Retinitis PigmentosaDrug: rhNGF 60 µg/ml eye drops solution;Drug: rhNGF 180 µg/ml eye drops solution;Drug: PlaceboDompé Farmaceutici S.p.ANULLCompleted18 YearsN/AAll50Phase 1/Phase 2Italy
82EUCTR2012-002574-31-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 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): noUnited States;France
83NCT01786395
(ClinicalTrials.gov)
March 201323/1/2013Phase III Efficacy and Safety Clinical Study of UF-021 for Treatment of Retinitis PigmentosaPhase III Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration PeriodRetinitis PigmentosaDrug: UF-021;Drug: PlaceboR-Tech Ueno, Ltd.NULLTerminated20 Years70 YearsBoth202Phase 3Japan
84EUCTR2012-002436-82-ES
(EUCTR)
13/12/201222/10/2012Melatonin effects on visual and sleep patterns anomalies in subjects diagnosed with Retinitis PigmentosaMelatonin effects on visual and sleep patterns anomalies in subjects diagnosed with Retinitis Pigmentosa To evaluate the sleep quality in patients with Retinitis Pigmentosa;Therapeutic area: Diseases [C] - Eye Diseases [C11]Trade Name: CIRCADIN 2 mg comprimidos de liberación prolongada
Product Name: Circadin 2 mg
INN or Proposed INN: Not answered
Other descriptive name: MELATONIN
Isabel Pinilla LozanoNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Spain
85EUCTR2011-004214-42-NL
(EUCTR)
04/10/201223/05/2012A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' visionAn Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) Leber Congenital AmaurosisRetinitis pigmentosa
MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Code: QLT091001QLT Inc.NULLNot RecruitingFemale: yes
Male: yes
31United States;Canada;Germany;Netherlands;United Kingdom
86EUCTR2011-004214-42-GB
(EUCTR)
03/09/201202/05/2012A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' visionAn Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) Leber Congenital Amaurosis Retinitis pigmentosa
MedDRA version: 20.0;Level: PT;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 20.0;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders ;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Code: QLT091001QLT Inc.NULLNot Recruiting Female: yes
Male: yes
31 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Canada;Netherlands;Germany;United Kingdom
87NCT01680510
(ClinicalTrials.gov)
September 20124/9/2012The Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella BardawilThe Effect of Oral Administration of 9-cis ß Carotene Rich Powder of the Alga Dunaliella Bardawil on Visual Functions in Patients With Retinitis PigmentosaRetinitis PigmentosaDietary Supplement: Alga Dunaliella Bardawil;Other: Placebo (starch)Sheba Medical CenterNULLRecruiting18 YearsN/AAll100Phase 2/Phase 3Israel
88EUCTR2011-004214-42-DE
(EUCTR)
06/08/201203/05/2012A study in patients with rare inherited eye conditions (Leber Congenital Amaurosis or Retinitis Pigmentosa), to see if repeated treatment with the study drug, QLT091001, is safe and works to improve patients' visionAn Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects with Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01) Leber Congenital AmaurosisRetinitis pigmentosa
MedDRA version: 14.1;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders
MedDRA version: 14.1;Classification code 10070667;Term: Leber's congenital amaurosis;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Code: QLT091001QLT Inc.NULLNot RecruitingFemale: yes
Male: yes
31United States;Canada;Netherlands;Germany;United Kingdom
89NCT01736059
(ClinicalTrials.gov)
July 201225/7/2012Clinical Trial of Autologous Intravitreal Bone-marrow CD34+ Stem Cells for RetinopathyA Pilot Clinical Trial of the Feasibility and Safety of Intravitreal Autologous Adult Bone Marrow Stem Cells in Treating Eyes With Vision Loss From RetinopathyNon-exudative Age-related Macular Degeneration;Diabetic Retinopathy;Retina Vein Occlusion;Retinitis Pigmentosa;Hereditary Macular DegenerationDrug: CD34+ bone marrow stem cells intravitrealUniversity of California, DavisNULLEnrolling by invitation18 Years100 YearsAll15Phase 1United States
90NCT01505062
(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
91NCT01543906
(ClinicalTrials.gov)
February 201217/2/2012Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)An Open-Label, Phase 1b, Safety/Proof-of-Concept Study to Evaluate the Effects of Oral QLT091001 in Retinitis Pigmentosa (RP) Subjects With an Autosomal Dominant Mutation in Retinal Pigment Epithelial 65 Protein (RPE65)Retinitis Pigmentosa (RP)Drug: QLT091001QLT Inc.NULLCompleted18 Years70 YearsBoth5Phase 1Canada;Ireland
92NCT01521793
(ClinicalTrials.gov)
January 20126/1/2012Repeated Treatments of QLT091001 in Subjects With Leber Congenital Amaurosis or Retinitis Pigmentosa (Extension of Study RET IRD 01)An Open-Label Study to Evaluate the Effects of Repeated Treatments of Oral QLT091001 on Safety and Vision Outcome in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin: Retinol Acyltransferase (LRAT) (Extension of Study RET IRD 01)LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa)Drug: QLT091001QLT Inc.NULLCompleted5 Years65 YearsBoth27Phase 1United States;Canada;Germany;Netherlands;United Kingdom
93NCT01530659
(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
94NCT01482195
(ClinicalTrials.gov)
August 201128/9/2011Trial of Subretinal Injection of (rAAV2-VMD2-hMERTK)Phase I Trial of Ocular Subretinal Injection of a Recombinant Adeno-Associated Virus (rAAV2-VMD2-hMERTK) Gene Vector to Patients With Retinal Disease Due to MERTK MutationsRetinal Disease;Retinitis PigmentosaBiological: Subretinal administration of rAAV2-VMD2-hMERTKRecombinant Adeno-Associated VirusKing Khaled Eye Specialist HospitalKing Faisal Specialist Hospital & Research CenterCompleted14 Years70 YearsAll6Phase 1Saudi Arabia
95NCT01560715
(ClinicalTrials.gov)
June 201119/3/2012Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis PigmentosaPhase 2 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis PigmentosaRetinitis PigmentosaBiological: intravitreal injection of autologous bone marrow stem cellsUniversity of Sao PauloNULLCompleted18 Years75 YearsAll50Phase 2Brazil
96NCT01399515
(ClinicalTrials.gov)
March 20113/5/2011Efficacy and Safety of Oral Valproic Acid for Retinitis PigmentosaRetinitis Pigmentosa;Retinal Diseases;Eye Diseases;Eye Disease, Hereditary;Retinal DegenerationDrug: Valproic AcidSeoul National University HospitalNULLCompleted18 YearsN/ABoth200Phase 2Korea, Republic of
97JPRN-JMA-IIA00053
24/12/201022/11/2010Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis PigmentosaClinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa Retinitis PigmentosaIntervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL.Yasuhiko HiramiMasayo Takahashi, Yasuo KurimotoCompleted>=20 YEARSNo LimitBOTH30NOT APPLICABLEJapan
98NCT01233609
(ClinicalTrials.gov)
November 20101/11/2010Trial of Oral Valproic Acid for Retinitis PigmentosaA Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis PigmentosaRetinitis PigmentosaDrug: Valproic Acid;Drug: PlaceboFoundation Fighting Blindness Clinical Research InstituteUnited States Department of DefenseCompleted18 YearsN/AAll90Phase 2United States
99JPRN-UMIN000005852
2010/06/0127/06/2011The Clinical Efficacy of a Topical Dorzolamide in the Management of Cystoid Macular Edema in Patients with Retinitis Pigmentosa retinitis pigmentosa1% dorzolamide three times daily in each eye during the follow-up periodKyushu university hospitalNULLComplete: follow-up complete20years-oldNot applicableMale and Female20Not applicableJapan
100EUCTR2010-019079-32-PT
(EUCTR)
19/05/201005/04/2010A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery SystemA Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System Patients who participated in a previous intravitreal Brimo PS DDS study, including 190342-028D (Retinitis pigmentosa) and 190342-032D (Geographic Atrophy from Age-related Macular Degeneration).Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9715X
INN or Proposed INN: Brimonidine Tartrate
Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9742X
INN or Proposed INN: Brimonidine Tartrate
Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9741X
INN or Proposed INN: Brimonidine Tartrate
Allergan LimitedNULLNot RecruitingFemale: yes
Male: yes
290Portugal;Czech Republic;Germany;Italy;United Kingdom
101EUCTR2010-019079-32-DE
(EUCTR)
12/05/201013/04/2010A Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery SystemA Multicenter, Patient-Masked, Safety Extension Study to Evaluate the Biodegradation of the Brimonidine Tartrate Posterior Segment Drug Delivery System Patients who participated in a previous intravitreal Brimo PS DDS study, including 190342-028D (Retinitis pigmentosa) and 190342-032D (Geographic Atrophy from Age-related Macular Degeneration).Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9715X
INN or Proposed INN: Brimonidine Tartrate
Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9742X
INN or Proposed INN: Brimonidine Tartrate
Product Name: Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System
Product Code: 9741X
INN or Proposed INN: Brimonidine Tartrate
Allergan LimitedNULLNot RecruitingFemale: yes
Male: yes
290Portugal;Czech Republic;Germany;Italy;United Kingdom
102NCT01014052
(ClinicalTrials.gov)
November 200912/11/2009Safety/Proof of Concept Study of Oral QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT) MutationsPhase 1b Study to Evaluate QLT091001 in Subjects With Leber Congenital Amaurosis (LCA) or Retinitis Pigmentosa (RP) Due to Inherited Deficiencies of Retinal Pigment Epithelial 65 Protein (RPE65) or Lecithin:Retinol Acyltransferase (LRAT)LCA (Leber Congenital Amaurosis);RP (Retinitis Pigmentosa)Drug: QLT091001QLT Inc.NULLCompleted5 Years65 YearsBoth32Phase 1United States;Canada;Germany;Netherlands;United Kingdom
103EUCTR2008-004561-26-IT
(EUCTR)
01/06/200911/11/2008Inner Retinal Dysfunction of the Cone System in Inherited Photoreceptor Degenerations: A Study of Disease Sequence and Assessment of Novel Therapeutic Strategies. - NDInner Retinal Dysfunction of the Cone System in Inherited Photoreceptor Degenerations: A Study of Disease Sequence and Assessment of Novel Therapeutic Strategies. - ND retinitis pigmentosa
MedDRA version: 9.1;Level: LLT;Classification code 10038914;Term: Retinitis pigmentosa
MedDRA version: 9.1;Level: PT;Term: Retinitis pigmentosa
Product Name: Nerve Growt Factor - 2.5S
INN or Proposed INN: COQUN
POLICLINICO UNIVERSITARIO AGOSTINO GEMELLINULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
Italy
104NCT01068561
(ClinicalTrials.gov)
May 200910/2/2010Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis PigmentosaPhase 1 Study Of Autologous Bone Marrow-Derived Stem Cells Transplantation For Retinitis PigmentosaRetinitis PigmentosaBiological: intravitreal injection of autologous bone marrow stem cellsUniversity of Sao PauloNULLCompleted18 YearsN/ABoth5Phase 1Brazil
105JPRN-UMIN000006820
2009/02/0102/12/2011Neuroprotective efficacy of topical unoprostone isopropyl or nipradilol in patients with retinitis pigmentosa retinitis pigmentosa0.12% topical unoprostone isopropyl twice daily
0.25% topical nipradilol twice daily
Kyushu university hospitalNULLComplete: follow-up completeNot applicableNot applicableMale and Female50Not selectedJapan
106NCT01773278
(ClinicalTrials.gov)
December 20083/12/2012Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)Smith-Lemli-Opitz Syndrome;Cone-Rod Dystrophy;Hearing LossDrug: Antioxidants;Drug: CholesterolUniversity of Colorado, DenverNULLRecruitingN/A65 YearsAll100Phase 2United States
107JPRN-JapicCTI-090748
01/10/200820/04/2009Phase II Study of 0.15% UF-021 in patients with retinitis pigmentosaPhase II Study of 0.15% UF-021 in patients with retinitis pigmentosa retinitis pigmentosaIntervention name : UF-021 (isopropyl unoprostone)
Dosage And administration of the intervention : Eye drop
R-Tech Ueno, LtdNULL2065BOTH105Phase 2NULL
108NCT01256697
(ClinicalTrials.gov)
August 200825/2/2010The Effect of Oral Administration of 9-cis Rich Powder of the Alga Dunaliella Bardawil on Visual Functions im Patients With Retinitis PigmentosaThe Effect of Oral Administration of 9-cis Rich Powder of the Alga DunaliellaRetinitis PigmentosaDietary Supplement: Alga Dunaliella Bardawil;Dietary Supplement: Alga Dunaliella Bardawill;Other: Sugar pillSheba Medical CenterNULLCompleted18 YearsN/AFemale34N/AIsrael
109NCT00661479
(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
110NCT00458575
(ClinicalTrials.gov)
April 200710/4/2007A Study to Evaluate the Safety of CNTO 2476 in Patients With Advanced Retinitis PigmentosaA Phase I Open Label Non-comparative Study Evaluating the Safety of a Single, Unilateral, Subretinal Administration of CNTO 2476 in Advanced Retinitis PigmentosaRetinitis PigmentosaDrug: CNTO 2476Centocor, Inc.NULLTerminated18 YearsN/ABoth7Phase 1United States
111NCT00433277
(ClinicalTrials.gov)
February 20076/2/2007Autoimmunity in Retinitis PigmentosaRetinitis PigmentosaDrug: twice-daily dosage with 0.5% cyclosporine-A eyedropsSemmelweis UniversityNULLActive, not recruitingN/AN/ABothN/AHungary
112NCT00447980
(ClinicalTrials.gov)
January 20079/3/2007A Study of Encapsulated Cell Technology (ECT) Implant for Participants With Early Stage Retinitis PigmentosaA Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Field Sensitivity as the Primary OutcomeRetinitis PigmentosaDrug: NT-501Neurotech PharmaceuticalsNULLCompleted18 Years64 YearsBoth68Phase 2United States
113NCT00447993
(ClinicalTrials.gov)
January 20079/3/2007A Study of Encapsulated Cell Technology (ECT) Implant for Patients With Late Stage Retinitis PigmentosaA Phase II/III Study of Encapsulated Human NTC-201 Cell Implants Releasing Ciliary Neurotrophic Factor (CNTF) for Participants With Retinitis Pigmentosa Using Visual Acuity as the Primary OutcomeRetinitis PigmentosaDrug: NT-501Neurotech PharmaceuticalsNULLCompleted18 Years68 YearsBoth65Phase 2United States
114NCT00100230
(ClinicalTrials.gov)
September 200427/12/2004DHA and X-Linked Retinitis PigmentosaInvestigation of Effectiveness and Safety of High Dose Docosahexaenoic Acid (DHA) in X-Linked Retinitis PigmentosaRetinitis Pigmentosa;X-linked Genetic DiseasesDrug: docosahexaenoic acid OR corn/soy oil placeboRetina Foundation of the SouthwestFoundation Fighting Blindness;DSM Nutritional Products, Inc.Completed7 Years32 YearsMale78Phase 2United States
115NCT00461435
(ClinicalTrials.gov)
January 200417/4/2007Slowing the Degenerative Process, Long Lasting Effect of Hyperbaric Oxygen Therapy in Retinitis PigmentosaSlowing the Degenerative Process, Long Lasting Effect of Hyperbaric Oxygen Therapy in Retinitis PigmentosaRetinitis PigmentosaProcedure: hyperbaric oxygen therapyAzienda Policlinico Umberto INULLCompleted20 Years70 YearsBothN/AItaly
116NCT00065455
(ClinicalTrials.gov)
July 17, 200323/7/2003Investigating the Effect of Vitamin A Supplementation on Retinitis PigmentosaPilot Study on the Effect of Vitamin A Supplementation on Cone Function in Retinitis PigmentosaRetinitis PigmentosaDrug: Vitamin ANational Eye Institute (NEI)NULLCompleted18 YearsN/AAll11Phase 1United States
117NCT00346333
(ClinicalTrials.gov)
July 200327/6/2006Clinical Trial of Lutein for Patients With Retinitis Pigmentosa Receiving Vitamin ARandomized Clinical Trial for Retinitis PigmentosaRetinitis PigmentosaDrug: Lutein;Dietary Supplement: Cornstarch controlNational Eye Institute (NEI)NULLCompleted18 Years60 YearsAll240Phase 3United States
118NCT00063765
(ClinicalTrials.gov)
June 20033/7/2003Evaluation of Safety of Ciliary Neurotrophic Factor Implants in the EyeA Phase I Study of NT-501-10 and NT-501-6A.02, Implants of Encapsulated Human NTC-210 Cells Releasing Ciliary Neurotrophic Factor (CNTF), in Patients With Retinitis PigmentosaRetinitis PigmentosaDrug: Ciliary Neurotrophic Factor Implant NT-501National Eye Institute (NEI)NULLCompletedN/AN/ABoth10Phase 1United States
119JPRN-UMIN000007895
2001/07/0101/06/2012Effects of nilvadipine on the progression of visual field defects in patients with retinitis pigmentosaEffects of nilvadipine on the progression of visual field defects in patients with retinitis pigmentosa - Effects of nilvadipine on the progression of retinitis pigmentosa retinitis pigmentosaTreated group: oral administration of nilvadipine 4mg per day

Control group: herenien, tocoferol or no medication as patients' requests
Department of Ophthalmology, Hirosaki University Graduate School of MedicineNULLComplete: follow-up complete20years-old80years-oldMale and Female40Phase 2Japan
120NCT00029289
(ClinicalTrials.gov)
April 20019/1/2002Effects of Lutein in Retinitis PigmentosaEffects of Lutein in Retinitis PigmentosaRetinitis PigmentosaDrug: Lutein (10 or 30 mg/day) capsulesNational Center for Complementary and Integrative Health (NCCIH)NULLCompletedPhase 1/Phase 2United States
121NCT00000116
(ClinicalTrials.gov)
May 199623/9/1999Randomized Trial for Retinitis PigmentosaRandomized Trial for Retinitis PigmentosaRetinitis PigmentosaDrug: Vitamin A;Drug: Nutritional SupplementNational Eye Institute (NEI)NULLCompleted18 Years56 YearsBothPhase 3United States
122NCT00004827
(ClinicalTrials.gov)
March 199624/2/2000Study of Docosahexaenoic Acid (DHA) Supplementation in Patients With X-Linked Retinitis PigmentosaRetinitis PigmentosaDrug: docosahexaenoic acidRetina Foundation of the SouthwestNULLCompletedN/AN/AMale46N/ANULL
123NCT00000114
(ClinicalTrials.gov)
May 198423/9/1999Randomized Trial of Vitamin A and Vitamin E Supplementation for Retinitis PigmentosaRetinitis PigmentosaDrug: Vitamin E;Drug: Vitamin ANational Eye Institute (NEI)NULLCompleted18 Years49 YearsBothPhase 3NULL
124EUCTR2021-002728-19-DK
(EUCTR)
26/11/2021A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision LossA 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 - Celeste Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3France;United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom
125EUCTR2021-002729-74-NO
(EUCTR)
14/10/2021A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision lossA 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 - Sirius Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
81Phase 2;Phase 3United States;European Union;Canada;Brazil;Germany;Norway;United Kingdom
126JPRN-JapicCTI-132079
Phase 3 Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration PeriodPhase 3 Clinical Study of UF-021 for Retinitis Pigmentosa - Evaluation for a Comparative Double Masked Placebo Controlled Study Period and a Continuous Administration Period Retinitis PigmentosaIntervention name : UF-021(isopropyl unoprostone)
Dosage And administration of the intervention : Eye drop
Control intervention name : null
R-Tech Ueno, LtdNULL2070BOTHPhase 3NULL
127EUCTR2021-002729-74-DK
(EUCTR)
26/11/2021A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision lossA 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 - Sirius Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
81Phase 2;Phase 3United States;European Union;Canada;Brazil;Denmark;Netherlands;Norway;Germany;United Kingdom
128EUCTR2021-002728-19-NL
(EUCTR)
15/01/2022A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision LossA 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 - Celeste Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom
129EUCTR2021-002729-74-NL
(EUCTR)
15/01/2022A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with advanced vision lossA 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 - Sirius Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
81Phase 2;Phase 3United States;European Union;Canada;Brazil;Norway;Germany;Netherlands;United Kingdom
130EUCTR2021-002728-19-FR
(EUCTR)
23/09/2021A Phase 2/3 study to evaluate efficacy, safety, and tolerability of QR-421a in subjects with with Early to Moderate Vision LossA 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 - Celeste Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
MedDRA version: 20.0;Level: PT;Classification code 10038914;Term: Retinitis pigmentosa;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]
Product Name: QR-421a
Product Code: QR-421a
INN or Proposed INN: Not available
Other descriptive name: QR-421a
ProQR Therapeutics IV B.V.NULLNAFemale: yes
Male: yes
120Phase 2;Phase 3United States;France;European Union;Canada;Brazil;Netherlands;Norway;Germany;United Kingdom