Disease · MONDO_0019353
Stargardt disease
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Stargardt disease. Diseases, record MONDO_0019353. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0019353
On this page
Disease
Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.
- Highest stage of its drugs
- Phase 3, for 3 of its 7 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs7
The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.
| Drug | ChEMBL ID | Type | Open Targets' stage | Stage reported by | Reports | Of them trials | Trials shown here | Source |
|---|---|---|---|---|---|---|---|---|
| TINLAREBANT | CHEMBL3967849 | Small molecule | Phase 3 | ClinicalTrials.gov and TTD | 4 | 2 | 2 | Drugs and targets |
| GILDEURETINOL ACETATE | CHEMBL5314607 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 1 | 1 | Drugs and targets |
| EMIXUSTAT HYDROCHLORIDE | CHEMBL2107829 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| GILDEURETINOL | CHEMBL5314606 | Small molecule | Phase 2 | ClinicalTrials.gov and TTD | 4 | 2 | 2 | Drugs and targets |
| STG-001 | CHEMBL4650305 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| DOCONEXENT | CHEMBL367149 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| FOMEPIZOLE | CHEMBL1308 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
Targets4
The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.
| Target | Protein | Its drugs that act on it | Mechanisms | Highest stage of those drugs | Gene in Genes | Source |
|---|---|---|---|---|---|---|
| ADH1A | Alcohol dehydrogenase class I | 1 | Alcohol dehydrogenase class I inhibitor | Phase 1 | https://conductscience.org/library/genes/124 | Drugs and targets |
| ADH1B | Alcohol dehydrogenase class I | 1 | Alcohol dehydrogenase class I inhibitor | Phase 1 | https://conductscience.org/library/genes/125 | Drugs and targets |
| ADH1C | Alcohol dehydrogenase class I | 1 | Alcohol dehydrogenase class I inhibitor | Phase 1 | https://conductscience.org/library/genes/126 | Drugs and targets |
| RPE65 | Retinoid isomerohydrolase | 1 | Retinoid isomerohydrolase inhibitor | Phase 3 | https://conductscience.org/library/genes/6121 | Drugs and targets |
Trials9
Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT03772665 | Safety and Efficacy of Emixustat in Stargardt Disease | Phase 3 | 194 | 2023-08-03 | Stargardt Disease | EMIXUSTAT HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT07419334 | Study of ALK-001 on the Progression of Stargardt Disease | Phase 3 | 230 | Stargardt Disease | GILDEURETINOL ACETATE | Its record names the disease | ClinicalTrials.gov | |
| NCT04239625 | Open-Label Extension: Tolerability and Effects of ALK-001 on Stargardt Disease (TEASE) | Phase 2 | 200 | Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related) | GILDEURETINOL | Its record names the disease | ClinicalTrials.gov | |
| NCT02402660 | Phase 2 Tolerability and Effects of ALK-001 on Stargardt Disease | Phase 2 | 160 | Stargardt Disease, Stargardt Macular Degeneration, Stargardt Macular Dystrophy, Autosomal Recessive Stargardt Disease 1 (ABCA4-related) | GILDEURETINOL | Its record names the disease | ClinicalTrials.gov | |
| NCT05244304 | Phase 3, Randomized, Placebo-Controlled Study of Tinlarebant to Explore Safety and Efficacy in Adolescent Stargardt Disease | Phase 3 | 104 | Stargardt Disease 1 | TINLAREBANT | Its record names the disease | ClinicalTrials.gov | |
| NCT06388083 | A Phase 2/3 Study to Evaluate the Efficacy and Safety of Tinlarebant in Subjects With Stargardt Disease | Phase 2/3 | 60 | STGD1, Stargardt Disease 1 | TINLAREBANT | Its record names the disease | ClinicalTrials.gov | |
| NCT00060749 | Effect of DHA Supplements on Macular Function in Patients With Stargardt Macular Dystrophy and Stargardt-like Macular Dystrophy | Phase 1 | 22 | Macular Degeneration | DOCONEXENT | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT00346853 | Phase 1 Pilot Study of 4-MP to Treat Stargardt Macular Dystrophy | Phase 1 | 10 | Macular Dystrophy, Corneal | FOMEPIZOLE | Its record names the disease | ClinicalTrials.gov | |
| NCT04489511 | Study of STG-001 in Subjects With Stargardt Disease | Phase 2 | 10 | Stargardt Disease-1 | STG-001 | Its record names the disease | ClinicalTrials.gov |
Comparisons in its trials2
Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.
| Comparison | Group 1, with the outcome | Group 2, with the outcome | Participants | Trial | Source |
|---|---|---|---|---|---|
| Emixustat vs Placebo: deaths from any cause | 0 of 127 | 0 of 66 | 193 | NCT03772665 | Trial comparisons |
| Emixustat vs Placebo: serious adverse events | 9 of 127 | 2 of 66 | 193 | NCT03772665 | Trial comparisons |
Papers from its trials2
The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.
Data dates
The date of each source this release read.
Sources 6
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ChEMBL, through Open TargetsCC BY-SA 3.0
- ClinicalTrials.govNo license stated
- NLM's MeSHNLM's terms
- Open TargetsCC0 1.0
- PubMedNLM's terms
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- ClinicalTrials.govnone stated
- NLM's MeSHNLM's
- Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
- PubMedNLM's terms
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this disease from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/diseases/v1/MONDO_0019353
GET https://conductscience.org/api/diseases/v1/MONDO_0019353/drugs