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	JPRN-UMIN000023618	2016/09/01	01/09/2016	Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa		Cataract associated with patients with retinitis pigmentosa	Cataract surgery and implantation of 1P intraocular lens (PCB00V) Cataract surgery and implantation of 3P intraocular lens (VA-70AD)	Kyushu University Hospital	NULL	Complete: follow-up complete	40years-old	Not applicable	Male and Female	40	Not selected	Japan
2	NCT04356716 (ClinicalTrials.gov)	November 11, 2014	20/4/2020	Sildenafil for Treatment of Choroidal Ischemia	Sildenafil for Treatment of Choroidal Ischemia	Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa	Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)	Columbia University	NULL	Recruiting	18 Years	N/A	All	25	Phase 2	United States
3	NCT01399515 (ClinicalTrials.gov)	March 2011	3/5/2011	Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa		Retinitis Pigmentosa;Retinal Diseases;Eye Diseases;Eye Disease, Hereditary;Retinal Degeneration	Drug: Valproic Acid	Seoul National University Hospital	NULL	Completed	18 Years	N/A	Both	200	Phase 2	Korea, Republic of
4	JPRN-JMA-IIA00053	24/12/2010	22/11/2010	Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa	Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa	Retinitis Pigmentosa	Intervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL.	Yasuhiko Hirami	Masayo Takahashi, Yasuo Kurimoto	Completed	>=20 YEARS	No Limit	BOTH	30	NOT APPLICABLE	Japan
5	NCT01233609 (ClinicalTrials.gov)	November 2010	1/11/2010	Trial of Oral Valproic Acid for Retinitis Pigmentosa	A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa	Retinitis Pigmentosa	Drug: Valproic Acid;Drug: Placebo	Foundation Fighting Blindness Clinical Research Institute	United States Department of Defense	Completed	18 Years	N/A	All	90	Phase 2	United States

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

JPRN-UMIN000023618

2016/09/01

01/09/2016

Comparison of posterior capsule opacification between 2 intraocular lenses in cataract patients associated with retinitis pigmentsa

Cataract associated with patients with retinitis pigmentosa

Cataract surgery and implantation of 1P intraocular lens (PCB00V)
Cataract surgery and implantation of 3P intraocular lens (VA-70AD)

Kyushu University Hospital

NULL

Complete: follow-up complete

40years-old

Not applicable

Male and Female

Not selected

Japan

NCT04356716
(ClinicalTrials.gov)

November 11, 2014

20/4/2020

Sildenafil for Treatment of Choroidal Ischemia

Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa

Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)

Columbia University

NULL

Recruiting

18 Years

N/A

All

Phase 2

United States

NCT01399515
(ClinicalTrials.gov)

March 2011

3/5/2011

Efficacy and Safety of Oral Valproic Acid for Retinitis Pigmentosa

Retinitis Pigmentosa;Retinal Diseases;Eye Diseases;Eye Disease, Hereditary;Retinal Degeneration

Drug: Valproic Acid

Seoul National University Hospital

NULL

Completed

18 Years

N/A

Both

200

Phase 2

Korea, Republic of

JPRN-JMA-IIA00053

24/12/2010

22/11/2010

Clinical Trial of Therapeutic Effect of Valproic Acid in Retinitis Pigmentosa

Retinitis Pigmentosa

Intervention type:DRUG. Intervention1:Sodium valproate, Dose form:TABLET, Route of administration:ORAL.

Yasuhiko Hirami

Masayo Takahashi, Yasuo Kurimoto

Completed

>=20 YEARS

No Limit

BOTH

NOT APPLICABLE

Japan

NCT01233609
(ClinicalTrials.gov)

November 2010

1/11/2010

Trial of Oral Valproic Acid for Retinitis Pigmentosa

A Phase II Multiple Site, Randomized, Placebo-Controlled Trial of Oral Valproic Acid for Autosomal Dominant Retinitis Pigmentosa

Retinitis Pigmentosa

Drug: Valproic Acid;Drug: Placebo

Foundation Fighting Blindness Clinical Research Institute

United States Department of Defense

Completed

18 Years

N/A

All

Phase 2

United States