DDrare: Database of Drug Development for Rare Diseases

90. 網膜色素変性症
［臨床試験数：103，薬物数：158（DrugBank：42），標的遺伝子数：52，標的パスウェイ数：107］

Searched query = "Retinitis pigmentosa", "Rod dystrophy", "Rod-Cone Dystrophy"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 100 µg Brimonidine Tartrate Implant; 200 µg Brimonidine Tartrate Implant; 400 µg Brimonidine Tartrate Implant; 4D-125 IVT Injection; 9715X; 9741X; 9742X; AAV-RPGR; AAV2-REP1; AAV2/5-hPDE6B; AAV2/5-hRKp.RPGR; AAV8-RPGR; ALLOGENEIC NEURAL RETINA-DERIVED HUMAN NEURAL RETINAL PROGENITOR CELLS EX VIVO EXPANDED; Acetate; Acetazolamide; Aflibercept; Alga Dunaliella Bardawil; Alga Dunaliella Bardawil powder; Alga Dunaliella Bardawill; Antioxidants; BMMNCs; BS01; Betamethasone; Brimonidine; Brimonidine Tartrate; Brimonidine Tartrate Posterior Segment Drug Delivery System (Brimo PS DDS) Applicator System; CD34+ bone marrow stem cells intravitreal; CIRCADIN 2 mg comprimidos de liberación prolongada; CNS10-NPC implantation; CNTO 2476; COQUN; CPK850; Cannabis; Carbidopa; Cholesterol; Ciliary Neurotrophic Factor Implant NT-501; Ciliary neurotrophic factor; Circadin; Circadin 2 mg; Combination Product: PRP combined Magnovision; Compound thrombosis capsule sig: 1.5g/tid; Conditioned Medium (CM); Continuous oxygen; Cornstarch; Cornstarch control; Cyclosporine; Cysteine; DAC; DDS; Device: Medmont Darl Adapted Chromatic (DAC); Device: MonCvONE electrophysiology; Device: RetMap, RM Electrode; Device: blue light-absorbing sunglasses; Device: dexamethasone injection; Dexamethasone; Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A); Docosahexaenoic acid; Dorzolamide; Eylea; Fluocinolone; Fluocinolone Acetonide; Fluocinolone acetonide; GS030-DP; Ginkgo; Ginkgo biloba; Ginkgo biloba pills sig: 300mg/tid; HORA-PDE6B (AAV2/5.hPDE6B); HRPC; HRPC DP; Human Embryonic Stem Cell Derived Retinal Pigment Epithelium (RPE); Human Retinal Progenitor Cells Drug Product; Human primary Retinal Pigment Epithelial (HuRPE) cells; Human retinal progenitor cells; Hydroxychloroquine; Hydroxychloroquine higher dose; Hydroxychloroquine lower dose; Iluvien; Intravitreal injection of Autologous bone marrow Stem Cell; Intravitreal injection of autologous bone marrow stem cells; Lentiviral vector containing MYO7A gene; Lentiviral vector containing the human MY07A gene; Levodopa; Levodopa-carbidopa; Lutein; Lutein (10 or 30 mg/day) capsules; Lycium Barbarum; MELATONIN; Melatonin; Minocycline; N-Acetyl Cysteine (NAC); NAC; NAC effervescent tablets; NPI-001; NT-501; Nerve Growt Factor - 2.5S; Nerve Growth Factor; Nilvadipine; Null; Nutritional Supplement; Other: Mock injection; Other: Observational; Other: Sham (no implant); Other: Sham procedure; Other: Sham-procedure (dose cohort 1&2 only); Other: Standard of Care Sildenafil; Other: Subconjunctival injection of saline; Other: Sugar pill; Other: Visual Acuity (VA); Oxygen; PRP; PS; Phosphate; Platelet rich plasma; Procedure: Sham; Procedure: hyperbaric oxygen therapy; QLT091001; QR-1123; QR-421a; RAAV.hPDE6A vector; RAAV2tYF-GRK1-RPGR; RECOMBINANT HUMAN NERVE GROWTH FACTOR (RHNGF); RST-001; Recombinant adeno-associated viral vector serotype 2.7m8 containing the ChrimsonR-tdTomato gene; Recombinant human Nerve Growth Factor (rhNGF); Retinal pigment epitheliums transplantation; RhNGF; RhNGF 180 µg/ml eye drops solution; RhNGF 60 µg/ml eye drops solution; SAR421869; Sildenafil; Sodium valproate; Stem Cell Transplantation; Stem/progenitor cells transplantation; Subretinal injection of rAAV.hPDE6A; Subretinal injection of umbilical cord blood platelet-rich plasma (CB-PRP); Timrepigene emparvovec; Tocoferol; Triamcinolone; Twice-daily dosage with 0.5% cyclosporine-A eyedrops; UF-021; UF-021 (isopropyl unoprostone); UF-021(isopropyl unoprostone); Umbilical Cord Mesenchymal Stem Cell (UC-MSC); Unoprostone; UshStat; UshStat (SAR421869); VA; Valproate; Valproic Acid; Valproic acid; Vehicle eye drops; Vitamin A; Vitamin AD sig: 1 tablet/tid; Vitamin B sig: 10mg/tid; Vitamin E; Wharton's jelly derived mesenchymal stem cell; Zuretinol Acetate; Zuretinol acetate

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2013-005393-22-DK (EUCTR)	03/11/2016	06/09/2016	A 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.	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	48	Phase 3	France;United States;Canada;Brazil;Denmark;Netherlands;Germany;United Kingdom;Switzerland

No.

TrialID

Date_
enrollment

Date_
registration

Public_title

Scientific_title

Condition

Intervention

Primary_
sponsor

Secondary_
sponsor

Recruitment_
Status

Inclusion_
agemin

Inclusion_
agemax

Inclusion_
gender

Target_
size

Phase

Countries

EUCTR2013-005393-22-DK
(EUCTR)

03/11/2016

06/09/2016

A 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.

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 3

France;United States;Canada;Brazil;Denmark;Netherlands;Germany;United Kingdom;Switzerland