DDrare: Database of Drug Development for Rare Diseases

301. 黄斑ジストロフィー
［臨床試験数：41，薬物数：47（DrugBank：12），標的遺伝子数：12，標的パスウェイ数：66］

Searched query = "Macular dystrophy", "Best disease", "Stargardt disease", "Cone dystrophy", "Cone rod dystrophy", "X-linked juvenile retinoschisis", "Central areolar choroidal 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 = 4-Methylpyrazole; 4-methylpyrazole; ACU-4429; ALK-001; ALK-001 (No generic name); ARC1905; ARC1905 20 MG/ML; Avacincaptad pegol; DAC; DHA; Device: Medmont Darl Adapted Chromatic (DAC); Device: MonCvONE electrophysiology; Device: RetMap, RM Electrode; Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A); Docosahexaenoic Acid (DHA) Dietary Supplement; Docosahexaenoic acid; EMIXUSTAT HYDROCHLORIDE (ACU-4429); EPA; Emixustat; Emixustat hydrochloride; HESC-RPE; Interferon Gamma; Interferon gamma-1b; Lentiviral vector containing ABCA4 gene; Long term follow up in all patients who received SAR422459 in previous study TDU13583; MA09-hRPE; Madeos; Metformin; Metformin hydrochloride; Other: Sham; Other: Standard of Care Sildenafil; Other: Visual Acuity (VA); Other: saline; Over the counter DHA/EPA dietary supplementation; Ranibizumab; Ranibizumab injection (0.5 mg); SAR422459; SORAPRAZAN; STG-001; Saffron supplementation; Sildenafil; Soraprazan; StarGen; Stem/progenitor cells transplantation; VA; Vett Lentiviral con gene ABCA4; Zimura

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	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
2	NCT00470977 (ClinicalTrials.gov)	May 2007	4/5/2007	Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy	Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection)	Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroidal Vasculopathy;Pseudoxanthoma Elasticum;Pathological Myopia;Multi-focal Choroiditis;Rubeosis Iridis;Von Hippel Lindau Disease;BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)	Drug: ranibizumab injection (0.5 mg)	Manhattan Eye, Ear & Throat Hospital	Genentech, Inc.	Completed	18 Years	N/A	Both	18	Phase 1;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

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

NCT00470977
(ClinicalTrials.gov)

May 2007

4/5/2007

Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy

Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection)

Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroidal Vasculopathy;Pseudoxanthoma Elasticum;Pathological Myopia;Multi-focal Choroiditis;Rubeosis Iridis;Von Hippel Lindau Disease;BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)

Drug: ranibizumab injection (0.5 mg)

Manhattan Eye, Ear & Throat Hospital

Genentech, Inc.

Completed

18 Years

N/A

Both

Phase 1;Phase 2

United States