301. Macular dystrophy
41 clinical trials,   47 drugs   (DrugBank: 12 drugs),   12 drug target genes,   66 drug target pathways

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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04545736
(ClinicalTrials.gov)
December 17, 202010/9/2020Oral Metformin for Treatment of ABCA4 RetinopathyOral Metformin for Treatment of ABCA4 RetinopathyABCA4 Retinopathy;Stargardt Disease;Retinal Dystrophy;Retinal DegenerationDrug: Metformin hydrochlorideNational Eye Institute (NEI)NULLRecruiting12 YearsN/AAll44Phase 1;Phase 2United States
2NCT04489511
(ClinicalTrials.gov)
October 1, 202021/7/2020Study of STG-001 in Subjects With Stargardt DiseaseA Phase 2a Study of the Safety, Pharmacokinetics and Pharmacodynamics of STG-001 in Subjects With Stargardt Disease (STGD1) Caused by Autosomal Recessive Mutation in ATP Binding Cassette Subfamily A Member 4 (ABCA4) GeneStargardt Disease-1Drug: STG-001Stargazer Pharmaceuticals, Inc.NULLRecruiting18 Years55 YearsAll12Phase 2United States
3NCT04239625
(ClinicalTrials.gov)
December 20, 201911/1/2020Open-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 DiseaseStargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)Drug: ALK-001Alkeus Pharmaceuticals, Inc.NULLEnrolling by invitation8 Years70 YearsAll140Phase 2United States
4EUCTR2018-003498-82-DK
(EUCTR)
11/06/201917/12/2018Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Product Name: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Acucela Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
194Phase 3United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;United Kingdom;Italy
5EUCTR2018-003498-82-NL
(EUCTR)
27/05/201928/01/2019Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Product Name: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Acucela Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
194Phase 3France;United States;Canada;Spain;Brazil;Denmark;South Africa;Germany;Netherlands;United Kingdom;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6NCT03297515
(ClinicalTrials.gov)
May 15, 201922/9/2017Therapeutic Potential of Omega-3 Fatty Acids Supplementation in Dry Macular Degeneration and Stargardt DiseaseProspective, Randomised, Double-blind Study to Assess the Therapeutic Potential of Omega-3 Fatty Acids Supplementation in Dry Macular Degeneration and Stargardt Disease (Macular Degeneration Omega-3 Study - MADEOSDry AMD;Stargardt Disease 1Dietary Supplement: Madeos;Dietary Supplement: PlaceboOphthalmos Research and Education InstituteNULLCompleted18 Years85 YearsAll29N/AFrance;Germany;Italy
7EUCTR2018-001496-20-DE
(EUCTR)
14/05/201912/11/2018A phase II study of soraprazan in patients with Stargardt diseaseA 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 GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2Netherlands;Germany;United Kingdom
8EUCTR2018-003498-82-ES
(EUCTR)
26/04/201922/01/2019Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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]
Acucela Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
162Phase 3France;United States;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom
9EUCTR2018-001496-20-NL
(EUCTR)
01/04/201909/05/2019A phase II study of soraprazan in patients with Stargardt diseaseA 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 GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2Netherlands;United Kingdom
10EUCTR2018-003498-82-GB
(EUCTR)
18/03/201906/12/2018Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Product Name: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Acucela Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
194Phase 3United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2018-001496-20-GB
(EUCTR)
21/01/201927/11/2018A phase II study of soraprazan in patients with Stargardt DiseaseA 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 GmbHNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
90Phase 2Netherlands;United Kingdom
12NCT03772938
(ClinicalTrials.gov)
December 13, 20183/11/2018Stem Cells Therapy in Degenerative Diseases of the RetinaStem Cells Therapy in Degenerative Diseases of the RetinaRetinal Degeneration;Retinitis Pigmentosa;Age Related Macular Degeneration;Stargardt Disease 1Biological: Stem/progenitor cells transplantationPomeranian Medical University SzczecinNULLUnknown status18 Years65 YearsAll30Phase 1Poland
13NCT03772665
(ClinicalTrials.gov)
November 7, 201830/11/2018Safety and Efficacy of Emixustat in Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride With Placebo for the Treatment of Macular Atrophy Secondary to Stargardt DiseaseStargardt DiseaseDrug: Emixustat;Drug: PlaceboKubota Vision Inc.NULLActive, not recruiting16 YearsN/AAll194Phase 3United States;Brazil;Canada;Denmark;France;Germany;Italy;Netherlands;South Africa;Spain;United Kingdom
14EUCTR2017-004783-35-DE
(EUCTR)
10/07/201823/01/2018A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt DiseaseA Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease Autosomal Recessive 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: Zimura
Product Code: ARC1905
INN or Proposed INN: avacincaptad pegol
Other descriptive name: ARC1905 20 MG/ML
IVERIC bioNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 2United States;France;Hungary;Canada;Spain;Israel;Germany;United Kingdom;Italy
15EUCTR2017-004783-35-GB
(EUCTR)
29/05/201826/01/2018A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt DiseaseA Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease Autosomal Recessive 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: Zimura
Product Code: ARC1905
INN or Proposed INN: avacincaptad pegol
Other descriptive name: ARC1905 20 MG/ML
IVERIC bioNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 2United States;France;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2017-004783-35-ES
(EUCTR)
09/04/201802/02/2018A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt DiseaseA Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease Autosomal Recessive 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: Zimura
Product Code: ARC1905
INN or Proposed INN: avacincaptad pegol
Other descriptive name: ARC1905 20 MG/ML
OPHTHOTECH CORPORATIONNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
120Phase 2France;United States;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom
17EUCTR2010-023111-34-IT
(EUCTR)
22/03/201828/09/2018A Phase I/IIa Study of SAR422459 in Patients With Stargardt's Macular DegenerationA Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients with Stargardt's Macular Degeneration. - na Stargardt Macular Degeneration, also known as Stargardt Macular Dystrophy, fundus flavimaculatus or 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: SAR422459
Product Code: Vett Lentiviral con gene ABCA4
INN or Proposed INN: SAR422459
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNULLNot RecruitingFemale: yes
Male: yes
46Phase 1;Phase 2France;United States;Netherlands;Italy
18EUCTR2017-004783-35-HU
(EUCTR)
21/03/201801/02/2018A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt DiseaseA Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects with Autosomal Recessive Stargardt Disease Autosomal Recessive 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: Zimura
Product Code: ARC1905
INN or Proposed INN: avacincaptad pegol
Other descriptive name: ARC1905 20 MG/ML
IVERIC bioNULLNot RecruitingFemale: yes
Male: yes
70Phase 2United States;France;Hungary;Canada;Spain;Israel;Germany;United Kingdom;Italy
19NCT03364153
(ClinicalTrials.gov)
January 12, 20181/12/2017Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)A Phase 2b Randomized, Double-masked, Controlled Trial to Establish the Safety and Efficacy of Zimura™ (Complement C5 Inhibitor) Compared to Sham in Subjects With Autosomal Recessive Stargardt DiseaseStargardt Disease 1Drug: Zimura;Other: ShamIVERIC bio, Inc.NULLRecruiting18 Years50 YearsAll120Phase 2United States;Canada;France;Germany;Hungary;Israel;Italy;Spain;United Kingdom
20NCT03033108
(ClinicalTrials.gov)
January 201720/1/2017Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt DiseaseA Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt DiseaseStargardt Disease;Macular AtrophyDrug: EmixustatKubota Vision Inc.NULLCompleted18 YearsN/AAll23Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT02617966
(ClinicalTrials.gov)
March 24, 201628/11/2015Rod and Cone Mediated Function in Retinal DiseaseRod and Cone Mediated Function in Retinal DiseaseRetinal Degeneration;Retinitis Pigmentosa;Stargardt's DiseaseDevice: Medmont Darl Adapted Chromatic (DAC);Device: RetMap, RM Electrode;Device: MonCvONE electrophysiologyNational Eye Institute (NEI)NULLRecruiting5 YearsN/AAll370N/AUnited States
22EUCTR2010-023111-34-NL
(EUCTR)
24/02/201604/02/2016A Phase I/IIa Study of SAR422459 in Patients With Stargardt Macular DegenerationA 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 développementNULLNot RecruitingFemale: yes
Male: yes
46Phase 1;Phase 2aFrance;United States;Netherlands;Italy
23NCT02402660
(ClinicalTrials.gov)
August 20159/3/2015Phase 2 Tolerability and Effects of ALK-001 on Stargardt DiseaseA 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 DiseaseStargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related)Drug: ALK-001;Drug: PlaceboAlkeus Pharmaceuticals, Inc.NULLRecruiting8 Years70 YearsAll140Phase 2United States
24NCT02338973
(ClinicalTrials.gov)
January 14, 201514/1/2015Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)Pilot Phase I/II Study of the Evaluation of Interferon Gamma-1b Administered Topically for Macular Edema/Intraretinal Schisis Cysts in Rod-Cone Dystrophy (RCD) and Enhanced S-Cone Syndrome (ESCS)Inherited Ophthalmic Diseases;Inherited Retinal DegenerationDrug: Interferon gamma-1bNational Eye Institute (NEI)NULLTerminated12 YearsN/AAll4Phase 1;Phase 2United States
25NCT04356716
(ClinicalTrials.gov)
November 11, 201420/4/2020Sildenafil for Treatment of Choroidal IschemiaSildenafil for Treatment of Choroidal IschemiaChoroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis PigmentosaDrug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA)Columbia UniversityNULLRecruiting18 YearsN/AAll25Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
26NCT02230228
(ClinicalTrials.gov)
April 201427/8/2014Phase 1 Safety Study of ALK-001 in Healthy VolunteersA Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy VolunteersStargardt Disease;Age-related Macular Degeneration;Other Retinal DystrophiesDrug: ALK-001 (No generic name)Alkeus Pharmaceuticals, Inc.NULLCompleted21 Years70 YearsBoth40Phase 1NULL
27NCT02941991
(ClinicalTrials.gov)
January 16, 201320/10/2016A 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 DystrophyBiological: hESC-RPEAstellas Institute for Regenerative MedicineNULLCompleted18 YearsN/AAll12Phase 1;Phase 2United Kingdom
28NCT01736592
(ClinicalTrials.gov)
December 14, 201226/11/2012Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular DegenerationAn Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular DegenerationStargardt's DiseaseDrug: Long term follow up in all patients who received SAR422459 in previous study TDU13583SanofiNULLActive, not recruiting6 YearsN/AAll27Phase 1;Phase 2United States;France
29NCT01625559
(ClinicalTrials.gov)
September 201218/6/2012Safety and Tolerability of MA09-hRPE Cells in Patients With Stargardt's Macular Dystrophy(SMD)A Phase I, Open-Label, Prospective Study to Determine the Safety and Tolerability of Sub-retinal Transplantation of Human Embryonic Stem Cell Derived Retinal Pigmented Epithelial(MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy(SMD)Stargardt's Macular DystrophyBiological: MA09-hRPECHABiotech CO., LtdNULLActive, not recruiting20 YearsN/ABoth3Phase 1Korea, Republic of
30NCT02445612
(ClinicalTrials.gov)
July 11, 201213/5/2015Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy PatientsLong Term Follow Up to 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 (MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)Stargardt's Macular DystrophyBiological: MA09-hRPEAstellas Institute for Regenerative MedicineNULLCompleted18 YearsN/AAll13Phase 1;Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31NCT01469832
(ClinicalTrials.gov)
November 20118/11/2011Safety 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 DystrophyBiological: MA09-hRPEAstellas Institute for Regenerative MedicineNULLCompleted18 YearsN/AAll12Phase 1;Phase 2United Kingdom
32EUCTR2010-023111-34-FR
(EUCTR)
18/07/201108/03/2012A 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) LtdNULLNot RecruitingFemale: yes
Male: yes
28Phase 1;Phase 2France;Netherlands;Italy
33NCT01367444
(ClinicalTrials.gov)
June 8, 20113/6/2011Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular DegenerationA Phase I/IIA Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients With Stargardt's Macular DegenerationStargardt's DiseaseDrug: SAR422459SanofiNULLTerminated6 YearsN/AAll27Phase 1;Phase 2United States;France
34NCT01345006
(ClinicalTrials.gov)
April 201128/4/2011Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular DystrophyA 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 (MA09-hRPE) Cells in Patients With Stargardt's Macular Dystrophy (SMD)Stargardt's Macular DystrophyBiological: MA09-hRPEAstellas Institute for Regenerative MedicineNULLCompleted18 YearsN/AAll13Phase 1;Phase 2United States
35NCT01278277
(ClinicalTrials.gov)
February 201114/1/2011Saffron Supplementation in Stargardt's DiseaseA Novel Therapeutic Strategy Targeting Photoreceptor Oxidative Damage in ABCR-related Retinal DegenerationsRetinal Degeneration;Genetic Disease;Single-Gene Defects;Macular DystrophyDietary Supplement: Saffron supplementation;Other: placeboCatholic University of the Sacred HeartNULLActive, not recruiting8 Years60 YearsAll30Phase 1;Phase 2Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36NCT00420602
(ClinicalTrials.gov)
September 21, 200710/1/2007DHA Supplementation in Patients With STGD3Clinical Interventions Against Stargardt Macular Dystrophy: DHA Supplementation in Patients With STGD3Dominantly Inherited Stargardt's Disease (STGD3)Dietary Supplement: Over the counter DHA/EPA dietary supplementationUniversity of UtahNULLCompleted18 Years105 YearsAll11N/AUnited States
37NCT00470977
(ClinicalTrials.gov)
May 20074/5/2007Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related MaculopathyTreatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy With Intravitreal Injection of Lucentis (Ranibizumab Injection)Coats' Disease;Idiopathic Retinal Telangiectasia;Retinal Angiomatous Proliferation;Polypoidal Choroidal Vasculopathy;Pseudoxanthoma Elasticum;Pathological Myopia;Multi-focal Choroiditis;Rubeosis Iridis;Von Hippel Lindau Disease;BEST VITELLIFORM MACULAR DYSTROPHY, MULTIFOCAL (Disorder)Drug: ranibizumab injection (0.5 mg)Manhattan Eye, Ear & Throat HospitalGenentech, Inc.Completed18 YearsN/ABoth18Phase 1;Phase 2United States
38NCT00346853
(ClinicalTrials.gov)
November 200528/6/2006Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular DystrophyClinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark AdaptationMacular Dystrophy, CornealDrug: 4-Methylpyrazole;Other: salineUniversity of UtahNULLCompleted18 Years65 YearsAll10Phase 1United States
39NCT00060749
(ClinicalTrials.gov)
May 5, 20039/5/2003Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular DystrophyInvestigation of the Effect of Dietary Docosahexaenoic Acid (DHA) Supplementation on Macular Function in Subjects With Autosomal Dominant Stargardt-Like and Autosomal Recessive Stargardt Macular DystrophyMacular DegenerationDrug: Docosahexaenoic Acid (DHA) Dietary SupplementNational Eye Institute (NEI)NULLCompletedN/AN/AAll22Phase 1United States
40EUCTR2018-003498-82-FR
(EUCTR)
14/12/2018Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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]
Acucela Inc.NULLNA Female: yes
Male: yes
162Phase 3United States;France;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
41EUCTR2018-003498-82-DE
(EUCTR)
21/12/2018Evaluation of Emixustat for the Treatment of Stargardt DiseaseA Phase 3 Multicenter, Randomized, Double-Masked Study Comparing the Efficacy and Safety of Emixustat Hydrochloride with Placebo for the Treatment of Macular Atrophy Secondary to Stargardt Disease - The SeaSTAR Study (Safety and efficacy of EmixustAt 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: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Product Name: Emixustat hydrochloride
Product Code: ACU-4429
INN or Proposed INN: Emixustat hydrochloride
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Acucela Inc.NULLNAFemale: yes
Male: yes
194Phase 3United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;United Kingdom;Italy