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	EUCTR2017-003104-42-GB (EUCTR)	26/06/2018	06/02/2018	A 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 study	A 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 Ltd	NULL	Authorised-recruitment may be ongoing or finished			Female: no Male: yes	440	Phase 3	France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom

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

EUCTR2017-003104-42-GB
(EUCTR)

26/06/2018

06/02/2018

A 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 study

A 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 Ltd

NULL

Authorised-recruitment may be ongoing or finished

Female: no
Male: yes

440

Phase 3

France;United States;Canada;Finland;Denmark;Netherlands;Germany;United Kingdom