Stargardt Disease clinical trials
The registry lists 66 studies for Stargardt Disease across more than 10 countries. 25 are recruiting or about to start. 45 of them are mainly about Stargardt Disease (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.
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Who runs them
Astellas Institute for Regenerative Medicine (5) · Alkeus Pharmaceuticals, Inc. (4) · Ray Therapeutics, Inc. (3) · National Eye Institute (NEI) (3) · Nanoscope Therapeutics Inc. (3) · AAVantgarde Bio Srl (2) · Sanofi (2) · Johns Hopkins University (2)
What is being tested here
Drugs under test in Stargardt Disease studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
9studies finish main data collection within a year
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 9
- Dec 2026An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 Gene · Splice Bio
- Dec 2026Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1) · West China Hospital
- Dec 2026A Study of DC6001 Tablet in Healthy Chinese Adult Subjects · Heronova Pharmaceuticals
- Feb 2027Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD) · Ray Therapeutics, Inc.
- Feb 2027An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease · HuidaGene Therapeutics Co., Ltd.
- May 2027Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT Study · Nanoscope Therapeutics Inc.
- Jun 2027An Observational Study in Children and Adults With Stargardt Disease · AAVantgarde Bio Srl
- Jul 2027Stem Cell Ophthalmology Treatment Study II · MD Stem Cells
- Aug 2027Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH) · Ascidian Therapeutics, Inc
Who these studies take
Of the 22 Stargardt Disease studies recruiting now, 14 (64%) set no upper age limit and 7 (32%) stop at 75 or younger. 13 (59%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 5 (23%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 9 (41%) | 9 (41%) |
| 18 | 21 (95%) | 21 (95%) |
| 30 | 21 (95%) | 21 (95%) |
| 40 | 21 (95%) | 21 (95%) |
| 50 | 19 (86%) | 19 (86%) |
| 65 | 17 (77%) | 17 (77%) |
| 75 | 15 (68%) | 15 (68%) |
| 85 | 15 (68%) | 15 (68%) |
Across all 66 studies, recruiting or not: 36% stop at 75 or younger and 30% at 65; 56% take children; 0% are for women only and 0% for men only; 17% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 50 of 66. Browse all 66 → or search them.
- Rod and Cone Mediated Function in Retinal DiseaseRecruitingNCT026179661 country
- Phase 1 Study of C.001 in Retinal DegenerationRecruitingNCT07594236Phase 11 country
- A Study About the Safety of a Single ASP2020 Eye Injection and if it Helps People With Vision Loss From Stargardt-type Eye ConditionsRecruitingNCT07734064Phase 11 country
- A Study to Learn How Stargardt-type Eye Conditions Progress in Children and AdultsRecruitingNCT074255741 country
- Prescreening Study to Identify Potential Stargardt Participants for ACDN-01 Clinical Trials (STARPATH)RecruitingNCT064453221 country
- A Study of AAVB-039 in Participants With Stargardt Disease (STGD1)RecruitingNCT07161544Phase 1/23 countries
- A Phase 1, First-in-Human Study of CITY-RBP4 in Healthy Volunteers and STGD1 ParticipantsRecruitingNCT07836452Phase 11 country
- Study of ALK-001 on the Progression of Stargardt DiseaseRecruitingNCT07419334Phase 31 country
- The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal DegenerationRecruitingNCT06319872Phase 11 country
- Safety and Preliminary Efficacy of VG801 in Patients With ABCA4 Mutation-associated Retinal Dystrophy (Stargardt Disease)RecruitingNCT07002398Phase 1/21 country
- Restoration of Central Vision With PRIMA in Patients With Photoreceptor DegenerationRecruitingNCT07266584Not applicable1 country
- Stem Cell Ophthalmology Treatment Study IIRecruitingNCT03011541Not applicable3 countries
- Inherited Retinal Degenerative Disease RegistryRecruitingNCT024359401 country
- Phase 1/2 Open-Label Dose-Escalation Study to Evaluate Safety of a Single Intravitreal Injection of RTx-021 in Patients With Stargardt DiseaseRecruitingNCT07439887Phase 1/21 country
- Development and Evaluation of Functional Visual Field and Navigation Endpoints in Moderate to Profound Inherited Retinal Disease (DEFINE-IRD)RecruitingNCT075026641 country
- A Study of DC6001 Tablet in Healthy Chinese Adult SubjectsRecruitingNCT07417566Phase 11 country
- Wide Field OCTA in Ocular DiseasesNot yet recruitingNCT07298174
- Inherited Retinal Diseases: Natural History and Genotype-Phenotype CorrelationsNot yet recruitingNCT072658951 country
- An Observational Study in Subjects to Follow the Progression of Stargardt Disease Type 1 (STGD1) Caused by Bi-Allelic Autosomal Recessive Mutations in the ABCA4 GeneRecruitingNCT064350003 countries
- Feasibility and Tolerability Study of Smart Contact Lens With Healthy Subjects and Patients With Stargardt's DiseaseNot yet recruitingNCT06989658Not applicable1 country
- An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT DiseaseRecruitingNCT07063251Early Phase 11 country
- A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)RecruitingNCT06942572Phase 1/21 country
- A Prospective Observational Study to Assess the Reliability and Validity of the MLSDTRecruitingNCT068054741 country
- Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal DegenerationRecruitingNCT063752391 country
- Structural and Functional Characterization of Rare Ocular DiseasesRecruitingNCT052580321 country
- Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)Active, not recruitingNCT06467344Phase 1/21 country
- Visual Performance Measures in a Virtual Reality Environment for Assessing Clinical Trial Outcomes in Those With Severely Reduced VisionCompletedNCT042817321 country
- Oral Metformin for Treatment of ABCA4 RetinopathyActive, not recruitingNCT04545736Phase 1/21 country
- Phase 2 Tolerability and Effects of ALK-001 on Stargardt DiseaseActive, not recruitingNCT02402660Phase 21 country
- Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE)Active, not recruitingNCT04239625Phase 21 country
- Phase I/II Follow-up Study of SAR422459 in Patients With Stargardt's Macular DegenerationActive, not recruitingNCT01736592Phase 22 countries
- A Phase 2/3 Trial to Assess the Efficacy and Safety of OCU410ST for Stargardt DiseaseActive, not recruitingNCT05956626Phase 2/31 country
- Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt DiseaseCompletedNCT05244304Phase 311 countries
- An Observational Study in Children and Adults With Stargardt DiseaseActive, not recruitingNCT065918062 countries
- Zimura Compared to Sham in Patients With Autosomal Recessive Stargardt Disease (STGD1)CompletedNCT03364153Phase 28 countries
- A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt DiseaseActive, not recruitingNCT06388083Phase 2/33 countries
- ABCA4-associated Disease in Childhood and Adolescence - a Phenotype StudyCompletedNCT063771501 country
- Phase 1 Safety Study of ALK-001 in Healthy VolunteersCompletedNCT02230228Phase 1
- Function and Imaging Assessments for G1961E-associated Stargardt DiseaseActive, not recruitingNCT056740581 country
- Non-interventional Long Term Follow-up Study of Participants Previously Enrolled in the STARLIGHT StudyActive, not recruitingNCT060481851 country
- 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)CompletedNCT01469832Phase 1/21 country
- Sub-retinal Transplantation of hESC Derived RPE(MA09-hRPE)Cells in Patients With Stargardt's Macular DystrophyCompletedNCT01345006Phase 1/21 country
- Long Term Follow Up of Sub-retinal Transplantation of hESC Derived RPE Cells in Stargardt Macular Dystrophy PatientsCompletedNCT024456121 country
- Safety and Efficacy of Emixustat in Stargardt DiseaseCompletedNCT03772665Phase 311 countries
- This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt DiseaseCompletedNCT05266014Phase 1/22 countries
- Safety and Efficacy of a Single Subretinal Injection of JWK006 Gene Therapy in Subjects With Stargardt Disease(STGD1)Active, not recruitingNCT06300476Phase 1/21 country
- Safety and Effects of a Single Intravitreal Injection of vMCO-010 Optogenetic Therapy in Subjects With Stargardt DiseaseCompletedNCT05417126Phase 21 country
- The Effect of Duration Between Sessions on Microperimetric Biofeedback Training in Patients With MaculopathiesCompletedNCT05904444Not applicable1 country
- Oxidative Stress In Stargardt Disease, Age Related Macular Degeneration and Diabetic RetinopathyTerminatedNCT028757041 country
- Phase I/IIA Study of SAR422459 in Participants With Stargardt's Macular DegenerationTerminatedNCT01367444Phase 1/22 countries