301. Macular dystrophy Clinical trials / Disease details
Clinical trials : 45 / Drugs : 46 - (DrugBank : 12) / Drug target genes : 9 - Drug target pathways : 67
| 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 | NCT05244304 (ClinicalTrials.gov) | March 2022 | 20/1/2022 | Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1 | Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects | Stargardt Disease 1 | Drug: Tinlarebant;Drug: Placebo | Belite Bio, Inc | NULL | Not yet recruiting | 12 Years | 18 Years | All | 60 | Phase 3 | United States;Australia;Hong Kong;Taiwan;United Kingdom;France;Germany;Switzerland |
| 2 | NCT05266014 (ClinicalTrials.gov) | March 12, 2021 | 26/1/2022 | This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease | Phase 1/2, Open-Label, Dose-Finding Followed by 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease | Stargardt Disease | Drug: tinlarebant | RBP4 Pty Ltd | Belite Bio, Inc | Active, not recruiting | 12 Years | 18 Years | All | 13 | Phase 1/Phase 2 | Australia;Taiwan |
| 3 | NCT04545736 (ClinicalTrials.gov) | November 23, 2020 | 10/9/2020 | Oral Metformin for Treatment of ABCA4 Retinopathy | Oral Metformin for Treatment of ABCA4 Retinopathy | ABCA4 Retinopathy;Stargardt Disease;Retinal Dystrophy;Retinal Degeneration | Drug: Metformin hydrochloride | National Eye Institute (NEI) | NULL | Recruiting | 12 Years | N/A | All | 38 | Phase 1/Phase 2 | United States |
| 4 | NCT04489511 (ClinicalTrials.gov) | October 1, 2020 | 21/7/2020 | Study of STG-001 in Subjects With Stargardt Disease | A 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) Gene | Stargardt Disease-1 | Drug: STG-001 | Stargazer Pharmaceuticals, Inc. | NULL | Completed | 18 Years | 55 Years | All | 10 | Phase 2 | United States |
| 5 | NCT04239625 (ClinicalTrials.gov) | December 20, 2019 | 11/1/2020 | Open-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 Disease | Stargardt Disease;Stargardt Macular Degeneration;Stargardt Macular Dystrophy;Autosomal Recessive Stargardt Disease 1 (ABCA4-related) | Drug: ALK-001 | Alkeus Pharmaceuticals, Inc. | NULL | Enrolling by invitation | 8 Years | 70 Years | All | 140 | Phase 2 | United States |
| 6 | EUCTR2018-001496-20-IT (EUCTR) | 18/07/2019 | 17/06/2021 | 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 - SMR3438 | 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 Product Code: [EU/3/13/1208 orphan] INN or Proposed INN: (7R,8R,9R)-2.3-Dimethyl-8-hydroxy-7(2-methoxyethoxy)- 9-phenyl-7.8.9.10-tetrahydro-imidazo-[1.2-h].[1.7].- naphthyridine | Katairo GmbH | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 90 | Phase 2 | Netherlands;Germany;United Kingdom;Italy | ||
| 7 | EUCTR2018-003498-82-DK (EUCTR) | 11/06/2019 | 17/12/2018 | 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 194 | Phase 3 | United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;United Kingdom;Italy | ||
| 8 | EUCTR2018-003498-82-IT (EUCTR) | 29/05/2019 | 13/01/2021 | 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) | 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 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 Product Name: Emixustat hydrochloride Product Code: [ACU-4429] INN or Proposed INN: Emixustat hydrochloride | Acucela Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 162 | Phase 3 | France;United States;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;United Kingdom;Italy | ||
| 9 | EUCTR2018-003498-82-NL (EUCTR) | 27/05/2019 | 28/01/2019 | 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 194 | Phase 3 | France;United States;Canada;Spain;Brazil;Denmark;South Africa;Germany;Netherlands;United Kingdom;Italy | ||
| 10 | NCT03297515 (ClinicalTrials.gov) | May 15, 2019 | 22/9/2017 | 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 | NULL | Completed | 18 Years | 85 Years | All | 32 | N/A | France;Germany;Italy |
| 11 | 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 | United Kingdom;Germany;Netherlands | ||
| 12 | EUCTR2018-003498-82-ES (EUCTR) | 26/04/2019 | 22/01/2019 | 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 162 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | France;United States;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom | ||
| 13 | 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 | Germany;Netherlands;United Kingdom | ||
| 14 | EUCTR2018-003498-82-GB (EUCTR) | 18/03/2019 | 06/12/2018 | 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 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 194 | Phase 3 | United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom | ||
| 15 | 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 | ||
| 16 | NCT03772938 (ClinicalTrials.gov) | December 13, 2018 | 3/11/2018 | 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 | NULL | Unknown status | 18 Years | 65 Years | All | 30 | Phase 1 | Poland |
| 17 | NCT03772665 (ClinicalTrials.gov) | November 7, 2018 | 30/11/2018 | 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 | Kubota Vision Inc. | NULL | Active, not recruiting | 16 Years | N/A | All | 194 | Phase 3 | United States;Brazil;Canada;Denmark;France;Germany;Italy;Netherlands;South Africa;Spain;United Kingdom |
| 18 | EUCTR2017-004783-35-DE (EUCTR) | 10/07/2018 | 23/01/2018 | 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 INN or Proposed INN: avacincaptad pegol Other descriptive name: ARC1905 20 MG/ML | IVERIC bio | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | United States;France;Hungary;Canada;Spain;Israel;Germany;United Kingdom;Italy | ||
| 19 | EUCTR2017-004783-35-IT (EUCTR) | 05/06/2018 | 25/01/2021 | 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 - A Clinical Trial to Assess the Safety and Efficacy of Intravitreous Administration of Zimura in Subj | 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 | IVERIC bio, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 120 | Phase 2 | France;United States;Hungary;Canada;Spain;Israel;Germany;United Kingdom;Italy | ||
| 20 | EUCTR2017-004783-35-GB (EUCTR) | 29/05/2018 | 26/01/2018 | 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 INN or Proposed INN: avacincaptad pegol Other descriptive name: ARC1905 20 MG/ML | IVERIC bio | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | United States;France;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom | ||
| 21 | EUCTR2017-004783-35-ES (EUCTR) | 09/04/2018 | 02/02/2018 | 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 INN or Proposed INN: avacincaptad pegol Other descriptive name: ARC1905 20 MG/ML | OPHTHOTECH CORPORATION | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 120 | Phase 2 | France;United States;Hungary;Canada;Spain;Israel;Germany;Italy;United Kingdom | ||
| 22 | EUCTR2010-023111-34-IT (EUCTR) | 22/03/2018 | 28/09/2018 | 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 INN or Proposed INN: SAR422459 | SANOFI-AVENTIS RECHERCHE E DEVELOPPEMENT | NULL | Not Recruiting | Female: yes Male: yes | 46 | Phase 1;Phase 2 | United States;France;Netherlands;Italy | ||
| 23 | EUCTR2017-004783-35-HU (EUCTR) | 21/03/2018 | 01/02/2018 | 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 INN or Proposed INN: avacincaptad pegol Other descriptive name: ARC1905 20 MG/ML | IVERIC bio | NULL | Not Recruiting | Female: yes Male: yes | 70 | Phase 2 | United States;France;Hungary;Canada;Spain;Israel;Germany;United Kingdom;Italy | ||
| 24 | NCT03364153 (ClinicalTrials.gov) | January 12, 2018 | 1/12/2017 | 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 | IVERIC bio, Inc. | NULL | Recruiting | 18 Years | 60 Years | All | 120 | Phase 2 | United States;Canada;France;Germany;Hungary;Israel;Italy;Spain;United Kingdom |
| 25 | NCT03033108 (ClinicalTrials.gov) | January 2017 | 20/1/2017 | 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 | Kubota Vision Inc. | NULL | Completed | 18 Years | N/A | All | 23 | Phase 2 | United States |
| 26 | 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 1/2 | France;United States;Netherlands;Italy | ||
| 27 | NCT02402660 (ClinicalTrials.gov) | August 2015 | 9/3/2015 | 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. | NULL | Recruiting | 8 Years | 70 Years | All | 140 | Phase 2 | United States |
| 28 | NCT02338973 (ClinicalTrials.gov) | January 14, 2015 | 14/1/2015 | 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) | NULL | Terminated | 12 Years | N/A | All | 4 | Phase 1/Phase 2 | United States |
| 29 | NCT04356716 (ClinicalTrials.gov) | November 11, 2014 | 20/4/2020 | Sildenafil for Treatment of Choroidal Ischemia | Sildenafil for Treatment of Choroidal Ischemia | Choroidal Ischemia;Vitelliform Macular Dystrophy;Age-related Macular Degeneration;Central Serous Retinopathy;Retinitis Pigmentosa | Drug: Sildenafil;Other: Standard of Care Sildenafil;Diagnostic Test: Ocular Coherence Tomography-Angiography (OCT-A);Other: Visual Acuity (VA) | Columbia University | NULL | Recruiting | 18 Years | N/A | All | 25 | Phase 2 | United States |
| 30 | NCT02230228 (ClinicalTrials.gov) | April 2014 | 27/8/2014 | 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. | NULL | Completed | 21 Years | 70 Years | Both | 40 | Phase 1 | NULL |
| 31 | NCT02941991 (ClinicalTrials.gov) | January 16, 2013 | 20/10/2016 | 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 | NULL | Completed | 18 Years | N/A | All | 12 | Phase 1/2 | United Kingdom |
| 32 | NCT01736592 (ClinicalTrials.gov) | December 14, 2012 | 26/11/2012 | 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: Long term follow up in all patients who received SAR422459 in previous study TDU13583 | Sanofi | NULL | Active, not recruiting | 6 Years | N/A | All | 27 | Phase 1/Phase 2 | United States;France |
| 33 | NCT01625559 (ClinicalTrials.gov) | September 2012 | 18/6/2012 | 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 | NULL | Active, not recruiting | 20 Years | N/A | Both | 3 | Phase 1 | Korea, Republic of |
| 34 | NCT02445612 (ClinicalTrials.gov) | July 11, 2012 | 13/5/2015 | 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 | NULL | Completed | 18 Years | N/A | All | 13 | Phase 1/2 | United States |
| 35 | NCT01469832 (ClinicalTrials.gov) | December 13, 2011 | 8/11/2011 | 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 | NULL | Completed | 18 Years | N/A | All | 12 | Phase 1/Phase 2 | United Kingdom |
| 36 | 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 | ||
| 37 | NCT01345006 (ClinicalTrials.gov) | June 16, 2011 | 28/4/2011 | 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 | NULL | Completed | 18 Years | N/A | All | 13 | Phase 1/Phase 2 | United States |
| 38 | NCT01367444 (ClinicalTrials.gov) | June 8, 2011 | 3/6/2011 | Phase I/IIA Study of SAR422459 in Participants 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 | NULL | Terminated | 6 Years | N/A | All | 27 | Phase 1/Phase 2 | United States;France |
| 39 | NCT01278277 (ClinicalTrials.gov) | February 2011 | 14/1/2011 | 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 | NULL | Active, not recruiting | 8 Years | 60 Years | All | 30 | Phase 1/Phase 2 | Italy |
| 40 | NCT00420602 (ClinicalTrials.gov) | September 21, 2007 | 10/1/2007 | 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 | NULL | Completed | 18 Years | 105 Years | All | 11 | N/A | United States |
| 41 | NCT00470977 (ClinicalTrials.gov) | May 2007 | 4/5/2007 | 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. | Completed | 18 Years | N/A | Both | 18 | Phase 1/Phase 2 | United States |
| 42 | NCT00346853 (ClinicalTrials.gov) | November 2005 | 28/6/2006 | 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 | NULL | Completed | 18 Years | 65 Years | All | 10 | Phase 1 | United States |
| 43 | NCT00060749 (ClinicalTrials.gov) | May 5, 2003 | 9/5/2003 | 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) | NULL | Completed | N/A | N/A | All | 22 | Phase 1 | United States |
| 44 | EUCTR2018-003498-82-FR (EUCTR) | 14/12/2018 | 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 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. | NULL | NA | Female: yes Male: yes | 162 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): no | United States;France;Canada;Brazil;Spain;Denmark;South Africa;Netherlands;Germany;Italy;United Kingdom | |||
| 45 | EUCTR2018-003498-82-DE (EUCTR) | 21/12/2018 | 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 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. | NULL | NA | Female: yes Male: yes | 194 | Phase 3 | United States;France;Canada;Spain;Brazil;Denmark;South Africa;Netherlands;Germany;United Kingdom;Italy |