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	NCT04239625 (ClinicalTrials.gov)	December 20, 2019	11/1/2020	Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)	A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease	Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)	Drug: ALK-001	Alkeus Pharmaceuticals, Inc.	NULL	Enrolling by invitation	8 Years	70 Years	All	140	Phase 2	United States
2	NCT02402660 (ClinicalTrials.gov)	August 2015	9/3/2015	Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease	A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease	Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)	Drug: ALK-001;Drug: Placebo	Alkeus Pharmaceuticals, Inc.	NULL	Recruiting	8 Years	70 Years	All	140	Phase 2	United States
3	NCT02230228 (ClinicalTrials.gov)	April 2014	27/8/2014	Phase 1 Safety Study of ALK-001 in Healthy Volunteers	A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers	Stargardt Disease;Age-related Macular Degeneration;Other Retinal Dystrophies	Drug: ALK-001 (No generic name)	Alkeus Pharmaceuticals, Inc.	NULL	Completed	21 Years	70 Years	Both	40	Phase 1	NULL

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

NCT04239625
(ClinicalTrials.gov)

December 20, 2019

11/1/2020

Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)

A Phase 2 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Long Term Safety, Tolerability, Pharmacokinetics and Effects of ALK-001 on the Progression of Stargardt Disease

Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

Drug: ALK-001

Alkeus Pharmaceuticals, Inc.

NULL

Enrolling by invitation

8 Years

70 Years

All

140

Phase 2

United States

NCT02402660
(ClinicalTrials.gov)

August 2015

9/3/2015

Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease

Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)

Drug: ALK-001;Drug: Placebo

Alkeus Pharmaceuticals, Inc.

NULL

Recruiting

8 Years

70 Years

All

140

Phase 2

United States

NCT02230228
(ClinicalTrials.gov)

April 2014

27/8/2014

Phase 1 Safety Study of ALK-001 in Healthy Volunteers

A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers

Stargardt Disease;Age-related Macular Degeneration;Other Retinal Dystrophies

Drug: ALK-001 (No generic name)

Alkeus Pharmaceuticals, Inc.

NULL

Completed

21 Years

70 Years

Both

Phase 1

NULL