Disease · MONDO_0010788

Leber hereditary optic neuropathy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. Leber hereditary optic neuropathy. Diseases, record MONDO_0010788. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010788

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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.

Name: Leber hereditary optic neuropathy; Open Targets
Open Targets ID: MONDO_0010788; Open Targets
Other names: LHON, Leber Hereditary optic atrophy, Leber optic atrophy, optic atrophy, hereditary, Leber; Open Targets
Disease Ontology: DOID:705; Disease Ontology
MeSH: D029242; MeSH
NCI Thesaurus: C84808; NCI Thesaurus
Orphanet: ORPHA:104; Orphanet
Highest stage of its drugs: Approval, for 1 of its 8 drugs, from EMA Human Drugs; Open Targets

Drugs8

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
IDEBENONE	CHEMBL252556	Small molecule	Approval	EMA Human Drugs	5	4	4	Drugs and targets
CURCUMIN	CHEMBL140	Small molecule	Phase 3	ClinicalTrials.gov	1	1	1	Drugs and targets
CYCLOSPORINE	CHEMBL160	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
CYSTEAMINE BITARTRATE	CHEMBL1200577	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ELAMIPRETIDE	CHEMBL3833370	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SONLICROMANOL	CHEMBL4802153	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
NIACINAMIDE	CHEMBL1140	Small molecule	Phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets
VATIQUINONE	CHEMBL1812161	Small molecule	Unknown	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets2

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
NQO1	NAD(P)H dehydrogenase [quinone] 1	1	Quinone reductase 1 modulator	Unknown	https://conductscience.org/library/genes/1728	Drugs and targets
PPIA	Peptidyl-prolyl cis-trans isomerase A	1	Cyclophilin A modulator	Phase 2	https://conductscience.org/library/genes/5478	Drugs and targets

Trials11

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
NCT02774005	Study to Assess the Efficacy and Safety of Raxone in LHON Patients	Phase 4	199	2023-04-21	Leber's Hereditary Optic Neuropathy (LHON)	IDEBENONE	Its record names the disease	ClinicalTrials.gov
NCT02473445	A Long-term Extension of Study RP103-MITO-001 (NCT02023866) to Assess Cysteamine Bitartrate Delayed-release Capsules (RP103) in Children With Inherited Mitochondrial Disease	Phase 2	22	2018-05-11	Mitochondrial Diseases	CYSTEAMINE BITARTRATE	Its record names the disease	ClinicalTrials.gov
NCT02693119	A Study Investigating the Safety, Tolerability, and Efficacy of Elamipretide Topical Ophthalmic Solution for Treatment of Leber's Hereditary Optic Neuropathy	Phase 2	12	2021-10-12	Leber's Hereditary Optic Neuropathy	ELAMIPRETIDE	Open Targets' reading of its record	ClinicalTrials.gov
NCT02771379	Post Authorisation Safety Study With Raxone in LHON Patients		229		Leber's Hereditary Optic Neuropathy (LHON)	IDEBENONE	Open Targets' reading of its record	ClinicalTrials.gov
NCT00747487	Study to Assess Efficacy,Safety and Tolerability of Idebenone in the Treatment of Leber's Hereditary Optic Neuropathy	Phase 2	85		Leber's Hereditary Optic Neuropathy	IDEBENONE	Open Targets' reading, confirmed by NLM's indexing of its papers	ClinicalTrials.gov
NCT00528151	A Randomized, Double-blind, Placebo-controlled Trial of Curcumin in Leber's Hereditary Optic Neuropathy (LHON)	Phase 3	70		Optic Atrophy, Hereditary, Leber	CURCUMIN	Its record names the disease	ClinicalTrials.gov
NCT02909400	The KHENERGY Study	Phase 2	20		Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MIDD	SONLICROMANOL	Open Targets' reading of its record	ClinicalTrials.gov
NCT07258667	Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy	Phase 1	13		Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON), Mitochondrial Disease, Optic Nerve Disease	NIACINAMIDE	Its record names the disease	ClinicalTrials.gov
NCT02176733	Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy	Phase 2	12		Leber Hereditary Optic Neuropathy	CYCLOSPORINE	Its record names the disease	ClinicalTrials.gov
NCT02300753	Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON]				Leber's Hereditary Optic Neuropathy	VATIQUINONE	Open Targets' reading of its record	ClinicalTrials.gov

1 to 10 of 11

See all 11 trials

Comparisons in its trials0

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.

None in this release.

Papers from its trials5

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
23263355	RESULT	1	PubMed
21788663	RESULT	1	PubMed
38428428	DERIVED	1	PubMed
30058726	DERIVED	1	PubMed
29454364	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

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_0010788

GET https://conductscience.org/api/diseases/v1/MONDO_0010788/drugs

API reference For AI assistants