Disease · MONDO_0020249
hereditary optic neuropathy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. hereditary optic neuropathy. Diseases, record MONDO_0020249. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0020249
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.
Drugs13
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 |
|---|---|---|---|---|---|---|---|---|
| EPOETIN BETA | CHEMBL2109092 | Protein | Phase 3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| AFLIBERCEPT | CHEMBL1742982 | Protein | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CLEMASTINE | CHEMBL1626 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| METFORMIN | CHEMBL1431 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| METHYLPREDNISOLONE | CHEMBL650 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TEPROTUMUMAB | CHEMBL1743079 | Antibody | Phase 2 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| ANTILYMPHOCYTE IMMUNOGLOBULIN (HORSE) | CHEMBL2108981 | Phase 1 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets | |
| CENEGERMIN | CHEMBL4297852 | Protein | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| CYCLOPHOSPHAMIDE | CHEMBL1200796 | Small molecule | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| INSULIN HUMAN | CHEMBL1201631 | Protein | Phase 1 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 13
See all 13 drugsTargets63
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 |
|---|---|---|---|---|---|---|
| VEGFA | Vascular endothelial growth factor A, long form | 3 | Vascular endothelial growth factor A inhibitor | Phase 2 | https://conductscience.org/library/genes/7422 | Drugs and targets |
| ADRB1 | Beta-1 adrenergic receptor | 1 | Beta-1 adrenergic receptor antagonist | Phase 1 | https://conductscience.org/library/genes/153 | Drugs and targets |
| ADRB2 | Beta-2 adrenergic receptor | 1 | Beta-2 adrenergic receptor antagonist | Phase 1 | https://conductscience.org/library/genes/154 | Drugs and targets |
| COXFA4 | Mitochondrial complex I (NADH dehydrogenase) | 1 | Mitochondrial complex I (NADH dehydrogenase) inhibitor | Phase 2 | https://conductscience.org/library/genes/4697 | Drugs and targets |
| COXFA4L2 | Mitochondrial complex I (NADH dehydrogenase) | 1 | Mitochondrial complex I (NADH dehydrogenase) inhibitor | Phase 2 | https://conductscience.org/library/genes/56901 | Drugs and targets |
| EPOR | Erythropoietin receptor | 1 | Erythropoietin receptor agonist | Phase 3 | https://conductscience.org/library/genes/2057 | Drugs and targets |
| GPD2 | Glycerol-3-phosphate dehydrogenase, mitochondrial | 1 | Mitochondrial glycerol-3-phosphate dehydrogenase inhibitor | Phase 2 | https://conductscience.org/library/genes/2820 | Drugs and targets |
| HRH1 | Histamine H1 receptor | 1 | Histamine H1 receptor antagonist | Phase 2 | https://conductscience.org/library/genes/3269 | Drugs and targets |
| IGF1R | Insulin-like growth factor 1 receptor | 1 | Insulin-like growth factor I receptor antagonist | Phase 2 | https://conductscience.org/library/genes/3480 | Drugs and targets |
| INSR | Insulin receptor | 1 | Insulin receptor agonist | Phase 1 | https://conductscience.org/library/genes/3643 | Drugs and targets |
1 to 10 of 63
See all 63 targetsTrials9
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. 1 more is not shown, since the checks do not confirm it.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02855450 | Study to Evaluate Safety and Efficacy of rhNGF Eye Drops Solution Versus Vehicle in Patients With Glaucoma | Phase 1 | 60 | 2020-11-25 | Glaucoma | CENEGERMIN | Its record names the disease | ClinicalTrials.gov |
| NCT00561834 | Ranibizumab Therapy for Non-arteritic Ischemic Optic Neuropathy | Phase 1 | 2 | 2016-07-25 | Nonarteritic Anterior Ischemic Optic Neuropathy | RANIBIZUMAB | Its record names the disease | ClinicalTrials.gov |
| NCT05131828 | CCMR Two: A Phase IIa, Randomised, Double-blind, Placebo-controlled Trial of the Ability of the Combination of Metformin and Clemastine to Promote Remyelination in People With Relapsing-remitting Multiple Sclerosis Already on Disease-modifying Therapy | Phase 2 | 70 | Multiple Sclerosis | CLEMASTINE, METFORMIN | Its record names the disease | ClinicalTrials.gov | |
| NCT07594912 | Teprotumumab N01 Versus Methylprednisolone After Urgent Orbital Decompression for Dysthyroid Optic Neuropathy | Phase 2 | 60 | Thyroid Eye Disease, TED, Graves Ophthalmopathy, Optic Neuropathy | METHYLPREDNISOLONE | Its record names the disease | ClinicalTrials.gov | |
| NCT05206877 | Topical Insulin for Glaucoma | Phase 1 | 52 | Glaucoma | INSULIN HUMAN | Its record names the disease | ClinicalTrials.gov | |
| NCT03085784 | Intravitreal Aflibercept Injection for Radiation Retinopathy Trial | Phase 2 | 40 | Radiation Retinopathy | AFLIBERCEPT | Its record names the disease | ClinicalTrials.gov | |
| NCT00278486 | Hematopoietic Stem Cell Transplantation in Autoimmune-Related Retinopathy(ARRON) | Phase 1 | 2 | Retinal Disease | CYCLOPHOSPHAMIDE | Its record names the disease | ClinicalTrials.gov | |
| NCT01331616 | Optic Neuropathy in 10 Patients With Glioblastoma Receiving Bevacizumab | Early phase 1 | 0 | Glioblastoma, Optic Neuropathy | BEVACIZUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT01607671 | Treatment Study for Ischemic Optic Neuropathy With Opthalmic Timolol Maleate 0.5% | Phase 1 | 0 | Optic Neuropathy, Ischemic, Anterior Ischemic Optic Neuropathy, Ischemic Optic Neuropathy, Optic Neuropathy, Anterior Ischemic | TIMOLOL MALEATE | 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 |
|---|---|---|---|---|---|
| rhNGF vs Vehicle: deaths from any cause | 0 of 40 | 0 of 20 | 60 | NCT02855450 | Trial comparisons |
| rhNGF vs Vehicle: serious adverse events | 3 of 40 | 2 of 20 | 60 | NCT02855450 | 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_0020249
GET https://conductscience.org/api/diseases/v1/MONDO_0020249/targets?page=2