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	NCT02941991 (ClinicalTrials.gov)	January 16, 2013	20/10/2016	A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)	Follow-up to 5 Years of a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)	Stargardt's Macular Dystrophy	Biological: hESC-RPE	Astellas Institute for Regenerative Medicine	NULL	Completed	18 Years	N/A	All	12	Phase 1;Phase 2	United Kingdom
2	NCT01469832 (ClinicalTrials.gov)	November 2011	8/11/2011	Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)	A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)	Stargardt's Macular Dystrophy	Biological: MA09-hRPE	Astellas Institute for Regenerative Medicine	NULL	Completed	18 Years	N/A	All	12	Phase 1;Phase 2	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

NCT02941991
(ClinicalTrials.gov)

January 16, 2013

20/10/2016

A Follow up Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

Follow-up to 5 Years of a Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

Stargardt's Macular Dystrophy

Biological: hESC-RPE

Astellas Institute for Regenerative Medicine

NULL

Completed

18 Years

N/A

All

Phase 1;Phase 2

United Kingdom

NCT01469832
(ClinicalTrials.gov)

November 2011

8/11/2011

Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)

A Phase I/II, Open-Label, Multi-Center, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial (hESC-RPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)