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	NCT01736592 (ClinicalTrials.gov)	December 14, 2012	26/11/2012	Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration	An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration	Stargardt's Disease	Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583	Sanofi	NULL	Active, not recruiting	6 Years	N/A	All	27	Phase 1;Phase 2	United States;France

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

NCT01736592
(ClinicalTrials.gov)

December 14, 2012

26/11/2012

Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration

An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration

Stargardt's Disease

Drug: Long term follow up in all patients who received SAR422459 in previous study TDU13583

Sanofi

NULL

Active, not recruiting

6 Years

N/A

All

Phase 1;Phase 2

United States;France