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_ gender	Target_ size	Phase	Countries
1	EUCTR2018-001496-20-DE (EUCTR)	14/05/2019	12/11/2018	A phase II study of soraprazan in patients with Stargardt disease	A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease	Stargardt Disease MedDRA version: 20.1;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: Soraprazan INN or Proposed INN: SORAPRAZAN	Katairo GmbH	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	90	Phase 2	Netherlands;Germany;United Kingdom
2	EUCTR2018-001496-20-NL (EUCTR)	01/04/2019	09/05/2019	A phase II study of soraprazan in patients with Stargardt disease	A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease	Stargardt Disease;Therapeutic area: Diseases [C] - Eye Diseases [C11]	Product Name: Soraprazan INN or Proposed INN: SORAPRAZAN	Katairo GmbH	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	90	Phase 2	Netherlands;United Kingdom
3	EUCTR2018-001496-20-GB (EUCTR)	21/01/2019	27/11/2018	A phase II study of soraprazan in patients with Stargardt Disease	A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease - SMR-3438	Stargardt Disease;Therapeutic area: Diseases [C] - Eye Diseases [C11]	Product Name: Soraprazan INN or Proposed INN: SORAPRAZAN	Katairo GmbH	NULL	Authorised-recruitment may be ongoing or finished	Female: yes Male: yes	90	Phase 2	Netherlands;United Kingdom

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TrialID

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Public_title

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Condition

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Recruitment_
Status

Inclusion_
agemin

Inclusion_
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EUCTR2018-001496-20-DE
(EUCTR)

14/05/2019

12/11/2018

A phase II study of soraprazan in patients with Stargardt disease

A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease

Stargardt Disease
MedDRA version: 20.1;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: Soraprazan
INN or Proposed INN: SORAPRAZAN

Katairo GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Netherlands;Germany;United Kingdom

EUCTR2018-001496-20-NL
(EUCTR)

01/04/2019

09/05/2019

A phase II study of soraprazan in patients with Stargardt disease

A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt disease

Stargardt Disease;Therapeutic area: Diseases [C] - Eye Diseases [C11]

Product Name: Soraprazan
INN or Proposed INN: SORAPRAZAN

Katairo GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Netherlands;United Kingdom

EUCTR2018-001496-20-GB
(EUCTR)

21/01/2019

27/11/2018

A phase II study of soraprazan in patients with Stargardt Disease

A multi-national, multi-centre, double-masked, placebo-controlled proof of concept trial to evaluate the safety and efficacy of oral soraprazan in Stargardt Disease - SMR-3438

Stargardt Disease;Therapeutic area: Diseases [C] - Eye Diseases [C11]

Product Name: Soraprazan
INN or Proposed INN: SORAPRAZAN

Katairo GmbH

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

Phase 2

Netherlands;United Kingdom