301. 黄斑ジストロフィー ［臨床試験数：25，薬物数：28（DrugBank：7），標的遺伝子数：6，標的パスウェイ数：45］
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.
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT03297515 | May 15, 2019 | 11 November 2019 | Therapeutic Potential of Omega-3 Fatty Acids Supplementation in Dry Macular Degeneration and Stargardt Disease | Prospective, 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 - MADEOS | Dry AMD;Stargardt Disease 1 | Dietary Supplement: Madeos;Dietary Supplement: Placebo | Ophthalmos Research and Education Institute | Recruiting | 18 Years | 85 Years | All | 60 | N/A | France;Germany;Italy | |
| 2 | NCT03772938 | December 13, 2018 | 18 December 2018 | Stem Cells Therapy in Degenerative Diseases of the Retina | Stem Cells Therapy in Degenerative Diseases of the Retina | Retinal Degeneration;Retinitis Pigmentosa;Age Related Macular Degeneration;Stargardt Disease 1 | Biological: Stem/progenitor cells transplantation | Pomeranian Medical University Szczecin | Recruiting | 18 Years | 65 Years | All | 30 | Phase 1 | Poland | |
| 3 | NCT03772665 | November 7, 2018 | 11 November 2019 | Safety and Efficacy of Emixustat in Stargardt Disease | A 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 | Stargardt Disease | Drug: Emixustat;Drug: Placebo | Acucela Inc. | Recruiting | 16 Years | N/A | All | 162 | Phase 3 | United States;Brazil;Canada;Denmark;France;Germany;Italy;Netherlands;South Africa;Spain;United Kingdom | |
| 4 | EUCTR2017-004783-35-ES | 09/04/2018 | 11 June 2018 | A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subjects with Autosomal Recessive Stargardt Disease | 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 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 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 CORPORATION | Authorised | Female: yes Male: yes | 120 | Phase 2 | France;United States;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom | |||
| 5 | EUCTR2010-023111-34-IT | 22/03/2018 | 10 October 2018 | A Phase I/IIa Study of SAR422459 in Patients With Stargardt's Macular Degeneration | A 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 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 DEVELOPPEMENT | Not Recruiting | Female: yes Male: yes | 46 | Phase 1;Phase 2 | France;United States;Netherlands;Italy | |||
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 6 | NCT03364153 | January 12, 2018 | 11 November 2019 | Zimura 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 Disease | Stargardt Disease 1 | Drug: Zimura;Other: Sham | Ophthotech Corporation | Not recruiting | 18 Years | 50 Years | All | 95 | Phase 2 | United States;France;Germany;Hungary;Israel;Italy;Spain;United Kingdom;Canada | |
| 7 | EUCTR2018-003498-82-FR | 30 April 2019 | Evaluation of Emixustat for the Treatment of Stargardt Disease | A 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 | 162 | Phase 3 | United States;France;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom | ||||
| 8 | NCT03033108 | January 2017 | 21 January 2019 | Pharmacodynamic Study of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease | A Phase 2a Multicenter, Randomized, Masked Study Evaluating the Pharmacodynamics of Emixustat Hydrochloride in Subjects With Macular Atrophy Secondary to Stargardt Disease | Stargardt Disease;Macular Atrophy | Drug: Emixustat | Acucela Inc. | Not recruiting | 18 Years | N/A | All | 23 | Phase 2 | United States | |
| 9 | EUCTR2010-023111-34-NL | 24/02/2016 | 11 April 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 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 développement | Not Recruiting | Female: yes Male: yes | 46 | Phase 1/2a | France;United States;Netherlands;Italy | |||
| 10 | NCT02402660 | August 2015 | 24 June 2019 | Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease | 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 Disease | Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related) | Drug: ALK-001;Drug: Placebo | Alkeus Pharmaceuticals, Inc. | Recruiting | 12 Years | 60 Years | All | 50 | Phase 2 | United States | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 11 | NCT02338973 | January 14, 2015 | 26 August 2019 | Interferon 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 Degeneration | Drug: Interferon gamma-1b | National Eye Institute (NEI) | Not recruiting | 12 Years | N/A | All | 4 | Phase 1/Phase 2 | United States | |
| 12 | NCT02230228 | April 2014 | 30 March 2015 | Phase 1 Safety Study of ALK-001 in Healthy Volunteers | A Phase 1, Open Label, Repeat Dose Study to Investigate the Safety and Pharmacokinetics of 4-week Daily Dosing of ALK-001 in Healthy Volunteers | Stargardt Disease;Age-related Macular Degeneration;Other Retinal Dystrophies | Drug: ALK-001 (No generic name) | Alkeus Pharmaceuticals, Inc. | Not recruiting | 21 Years | 70 Years | Both | 40 | Phase 1 | ||
| 13 | NCT02941991 | January 16, 2013 | 11 November 2019 | 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 | Not recruiting | 18 Years | N/A | All | 12 | Phase 2 | United Kingdom | |
| 14 | NCT01736592 | December 14, 2012 | 14 October 2019 | Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular Degeneration | An Open Label Study to Determine the Long Term Safety, Tolerability and Biological Activity of SAR422459 in Patients With Stargardt's Macular Degeneration | Stargardt's Disease | Drug: SAR422459 administered in previous study TDU13583 | Sanofi | Not recruiting | 6 Years | N/A | All | 46 | Phase 1/Phase 2 | United States;France | |
| 15 | NCT01625559 | September 2012 | 2 March 2015 | Safety 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 Dystrophy | Biological: MA09-hRPE | CHABiotech CO., Ltd | Not recruiting | 20 Years | N/A | Both | 3 | Phase 1 | Korea, Republic of | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 16 | NCT02445612 | July 11, 2012 | 11 November 2019 | Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy Patients | Long 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 Dystrophy | Biological: MA09-hRPE | Astellas Institute for Regenerative Medicine | Not recruiting | 18 Years | N/A | All | 13 | Phase 2 | United States | |
| 17 | NCT01469832 | November 2011 | 16 December 2017 | 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 | Not recruiting | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United Kingdom | |
| 18 | NCT01367444 | June 8, 2011 | 14 October 2019 | Phase I/II Study of SAR422459 in Patients With Stargardt's Macular Degeneration | A Phase I/IIa Dose Escalation Safety Study of Subretinally Injected SAR422459, Administered to Patients With Stargardt's Macular Degeneration | Stargardt's Disease | Drug: SAR422459 | Sanofi | Not recruiting | 6 Years | N/A | All | 27 | Phase 1/Phase 2 | United States;France | |
| 19 | NCT01345006 | April 2011 | 16 December 2017 | Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular Dystrophy | 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 Dystrophy | Biological: MA09-hRPE | Astellas Institute for Regenerative Medicine | Not recruiting | 18 Years | N/A | All | 13 | Phase 1/Phase 2 | United States | |
| 20 | NCT01278277 | February 2011 | 16 December 2017 | Saffron Supplementation in Stargardt's Disease | A Novel Therapeutic Strategy Targeting Photoreceptor Oxidative Damage in ABCR-related Retinal Degenerations | Retinal Degeneration;Genetic Disease;Single-Gene Defects;Macular Dystrophy | Dietary Supplement: Saffron supplementation;Other: placebo | Catholic University of the Sacred Heart | Not recruiting | 8 Years | 60 Years | All | 30 | Phase 1/Phase 2 | Italy | |
| No. | TrialID | Date_ enrollement | Last_Refreshed_ on | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
| 21 | NCT01773278 | December 2008 | 14 October 2019 | Cholesterol 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 Loss | Drug: Antioxidants;Drug: Cholesterol | University of Colorado, Denver | Recruiting | N/A | 65 Years | All | 100 | Phase 2 | United States | |
| 22 | NCT00420602 | September 21, 2007 | 11 June 2018 | DHA Supplementation in Patients With STGD3 | Clinical Interventions Against Stargardt Macular Dystrophy: DHA Supplementation in Patients With STGD3 | Dominantly Inherited Stargardt's Disease (STGD3) | Dietary Supplement: Over the counter DHA/EPA dietary supplementation | University of Utah | Not recruiting | 18 Years | 105 Years | All | 11 | N/A | United States | |
| 23 | NCT00470977 | May 2007 | 19 February 2015 | Treatment of Exudative and Vasogenic Chorioretinal Diseases Including Variants of AMD and Other CNV Related Maculopathy | Treatment 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 Hospital | Genentech, Inc. | Not recruiting | 18 Years | N/A | Both | 18 | Phase 1/Phase 2 | United States |
| 24 | NCT00346853 | November 2005 | 19 February 2015 | Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy | Clinical Interventions Against Stargardt Macular Dystrophy: Phase 1 Pilot Study of 4-MP as an Inhibitor of Dark Adaptation | Macular Dystrophy, Corneal | Drug: 4-Methylpyrazole;Other: saline | University of Utah | Not recruiting | 18 Years | 65 Years | Both | 10 | Phase 1 | United States | |
| 25 | NCT00060749 | May 5, 2003 | 16 December 2017 | Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy | Investigation of the Effect of Dietary Docosahexaenoic Acid (DHA) Supplementation on Macular Function in Subjects With Autosomal Dominant Stargardt-Like and Autosomal Recessive Stargardt Macular Dystrophy | Macular Degeneration | Drug: Docosahexaenoic Acid (DHA) Dietary Supplement | National Eye Institute (NEI) | Not recruiting | N/A | N/A | All | 22 | Phase 1 | United States |