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 301. Macular dystrophy
 [ 25 clinical trials,    28 drugs(DrugBank: 7 drugs),    6 target genes / 45 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 of the data. Export date: 11/21/2019, 11/20/2019. Trials are sorted by Date_enrolment from most recent to oldest in the table.

Search in Page    e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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PhaseCountries
1NCT03297515May 15, 201911 November 2019Therapeutic 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 InstituteRecruiting18 Years85 YearsAll60N/AFrance;Germany;Italy
2NCT03772938December 13, 201818 December 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 SzczecinRecruiting18 Years65 YearsAll30Phase 1Poland
3NCT03772665November 7, 201811 November 2019Safety 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: PlaceboAcucela Inc.Recruiting16 YearsN/AAll162Phase 3United States;Brazil;Canada;Denmark;France;Germany;Italy;Netherlands;South Africa;Spain;United Kingdom
4EUCTR2017-004783-35-ES09/04/201811 June 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 DiseaseAutosomal 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
Pharmaceutical Form: Solution for injection
INN or Proposed INN: avacincaptad pegol
Current Sponsor code: ARC1905
Other descriptive name: ARC1905 20 MG/ML
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 20-
OPHTHOTECH CORPORATIONAuthorisedFemale: yes
Male: yes
120Phase 2France;United States;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom
5EUCTR2010-023111-34-IT22/03/201810 October 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. - naStargardt 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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: SAR422459
Current Sponsor code: SAR422459
Concentration unit: U unit(s)
Concentration type: range
Concentration number: 180000-1800000
SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENTNot RecruitingFemale: yes
Male: yes
46Phase 1;Phase 2France;United States;Netherlands;Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
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Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
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size
PhaseCountries
6NCT03364153January 12, 201811 November 2019Zimura 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: ShamOphthotech CorporationNot recruiting18 Years50 YearsAll95Phase 2United States;France;Germany;Hungary;Israel;Italy;Spain;United Kingdom;Canada
7EUCTR2018-003498-82-FR30 April 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
Pharmaceutical Form: Tablet
INN or Proposed INN: Emixustat hydrochloride
CAS Number: 1141934-97-5
Current Sponsor code: ACU-4429
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Product Name: Emixustat hydrochloride
Product Code: ACU-4429
Pharmaceutical Form: Tablet
INN or Proposed INN: Emixustat hydrochloride
CAS Number: 1141934-97-5
Current Sponsor code: ACU-4429
Other descriptive name: EMIXUSTAT HYDROCHLORIDE (ACU-4429)
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use
Acucela Inc.Not Available Female: yes
Male: yes
162Phase 3United States;France;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom
8NCT03033108January 201721 January 2019Pharmacodynamic 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: EmixustatAcucela Inc.Not recruiting18 YearsN/AAll23Phase 2United States
9EUCTR2010-023111-34-NL24/02/201611 April 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
Pharmaceutical Form: Suspension for injection
INN or Proposed INN: SAR422459
Current Sponsor code: SAR422459
Concentration unit: U unit(s)
Concentration type: range
Concentration number: 180000-1800000
sanofi-aventis recherche et développementNot RecruitingFemale: yes
Male: yes
46Phase 1/2aFrance;United States;Netherlands;Italy
10NCT02402660August 201524 June 2019Phase 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.Recruiting12 Years60 YearsAll50Phase 2United States
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
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sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT02338973January 14, 201526 August 2019Interferon 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)Not recruiting12 YearsN/AAll4Phase 1/Phase 2United States
12NCT02230228April 201430 March 2015Phase 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.Not recruiting21 Years70 YearsBoth40Phase 1
13NCT02941991January 16, 201311 November 2019A 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 MedicineNot recruiting18 YearsN/AAll12Phase 2United Kingdom
14NCT01736592December 14, 201214 October 2019Phase 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: SAR422459 administered in previous study TDU13583SanofiNot recruiting6 YearsN/AAll46Phase 1/Phase 2United States;France
15NCT01625559September 20122 March 2015Safety 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., LtdNot recruiting20 YearsN/ABoth3Phase 1Korea, Republic of
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT02445612July 11, 201211 November 2019Long 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 MedicineNot recruiting18 YearsN/AAll13Phase 2United States
17NCT01469832November 201116 December 2017Safety 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 MedicineNot recruiting18 YearsN/AAll12Phase 1/Phase 2United Kingdom
18NCT01367444June 8, 201114 October 2019Phase I/II 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 DegenerationStargardt's DiseaseDrug: SAR422459SanofiNot recruiting6 YearsN/AAll27Phase 1/Phase 2United States;France
19NCT01345006April 201116 December 2017Sub-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 MedicineNot recruiting18 YearsN/AAll13Phase 1/Phase 2United States
20NCT01278277February 201116 December 2017Saffron 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 HeartNot recruiting8 Years60 YearsAll30Phase 1/Phase 2Italy
No.TrialIDDate_
enrollement
Last_Refreshed_
on
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
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Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21NCT01773278December 200814 October 2019Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)Cholesterol and Antioxidant Treatment in Patients With Smith-Lemli-Opitz Syndrome (SLOS)Smith-Lemli-Opitz Syndrome;Cone-Rod Dystrophy;Hearing LossDrug: Antioxidants;Drug: CholesterolUniversity of Colorado, DenverRecruitingN/A65 YearsAll100Phase 2United States
22NCT00420602September 21, 200711 June 2018DHA 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 UtahNot recruiting18 Years105 YearsAll11N/AUnited States
23NCT00470977May 200719 February 2015Treatment 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.Not recruiting18 YearsN/ABoth18Phase 1/Phase 2United States
24NCT00346853November 200519 February 2015Phase 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 UtahNot recruiting18 Years65 YearsBoth10Phase 1United States
25NCT00060749May 5, 200316 December 2017Effect 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)Not recruitingN/AN/AAll22Phase 1United States

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