DDrare: Database of Drug Development for Rare Diseases

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	EUCTR2010-023111-34-NL (EUCTR)	24/02/2016	04/02/2016	A Phase I/IIa Study of SAR422459 in Patients With Stargardt Macular Degeneration	A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients with Stargardt's Macular Degeneration.	Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease. MedDRA version: 18.1;Level: PT;Classification code 10062766;Term: Stargardt's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]	Product Name: SAR422459 Product Code: Lentiviral vector containing ABCA4 gene INN or Proposed INN: SAR422459	sanofi-aventis recherche et développement	NULL	Not Recruiting			Female: yes Male: yes	46	Phase 1;Phase 2a	France;United States;Netherlands;Italy
2	EUCTR2010-023111-34-FR (EUCTR)	18/07/2011	08/03/2012	A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.	A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.	Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.	Product Name: StarGen Product Code: Lentiviral vector containing ABCA4 gene INN or Proposed INN: StarGen	Oxford BioMedica (UK) Ltd	NULL	Not Recruiting			Female: yes Male: yes	28	Phase 1;Phase 2	France;Netherlands;Italy

No.

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Scientific_title

Condition

Intervention

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Status

Inclusion_
agemin

Inclusion_
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Inclusion_
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EUCTR2010-023111-34-NL
(EUCTR)

24/02/2016

04/02/2016

A Phase I/IIa Study of SAR422459 in Patients With Stargardt Macular Degeneration

A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients with Stargardt's Macular Degeneration.

Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.
MedDRA version: 18.1;Level: PT;Classification code 10062766;Term: Stargardt's disease;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Eye Diseases [C11]

Product Name: SAR422459
Product Code: Lentiviral vector containing ABCA4 gene
INN or Proposed INN: SAR422459

sanofi-aventis recherche et développement

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2a

France;United States;Netherlands;Italy

EUCTR2010-023111-34-FR
(EUCTR)

18/07/2011

08/03/2012

A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected StarGen, Administered to Patients with Stargardt's Macular Degeneration.

Stargardt Macular Dystrophy, also known as fundus flavimaculatus or Stargardt disease.

Product Name: StarGen
Product Code: Lentiviral vector containing ABCA4 gene
INN or Proposed INN: StarGen

Oxford BioMedica (UK) Ltd

NULL

Not Recruiting

Female: yes
Male: yes

Phase 1;Phase 2

France;Netherlands;Italy