Disease · MONDO_0010789

MELAS syndrome

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. MELAS syndrome. Diseases, record MONDO_0010789. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0010789

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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: MELAS syndrome; Open Targets
Open Targets ID: MONDO_0010789; Open Targets
Other names: MELAS, Mitochondrial Myopathy, Lactic Acidosis, Stroke-Like Episode, Syndrome, MELAS, mitochondrial encephalomyopathy, lactic acidosis and stroke, mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes, mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like episodes; Open Targets
Disease Ontology: DOID:3687; Disease Ontology
MeSH: D017241; MeSH
NCI Thesaurus: C84885; NCI Thesaurus
Orphanet: ORPHA:550; Orphanet
Highest stage of its drugs: Phase 2, for 5 of its 6 drugs, from ClinicalTrials.gov; Open Targets

Drugs6

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
ZAGOCIGUAT	CHEMBL5314620	Small molecule	Phase 2	ClinicalTrials.gov	3	3	3	Drugs and targets
ARGININE	CHEMBL1485	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DICHLOROACETIC ACID	CHEMBL13960	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
IDEBENONE	CHEMBL252556	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
SONLICROMANOL	CHEMBL4802153	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
L-CITRULLINE	CHEMBL444814	Small molecule	Phase 1	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets0

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.

None in this release.

Trials8

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
NCT00887562	Study of Idebenone in the Treatment of Mitochondrial Encephalopathy Lactic Acidosis & Stroke-like Episodes	Phase 2	27	2016-08-10	MELAS Syndrome	IDEBENONE	Its record names the disease	ClinicalTrials.gov
NCT04475549	Phase 2a Study of IW-6463 in Adults Diagnosed With Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes (MELAS)	Phase 2	8	2023-11-15	MELAS	ZAGOCIGUAT	Its record names the disease	ClinicalTrials.gov
NCT06402123	A Phase 2b Study of Zagociguat in Patients With MELAS	Phase 2	43		Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)	ZAGOCIGUAT	Its record names the disease	ClinicalTrials.gov
NCT06961344	An Open-label Extension Safety Study of MELAS Patients Who Completed TIS6463-203 (PRIZM)	Phase 2	37		Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-Like Episodes (MELAS Syndrome)	ZAGOCIGUAT	Its record names the disease	ClinicalTrials.gov
NCT00068913	Evaluating the Effectiveness of a Dichloroacetate in MELAS Syndrome	Phase 2	35		MELAS Syndrome	DICHLOROACETIC ACID	Its record names the disease	ClinicalTrials.gov
NCT02909400	The KHENERGY Study	Phase 2	20		Mitochondrial Diseases, Mitochondrial Myopathies, Mitochondrial Encephalomyopathies, MELAS, MIDD	SONLICROMANOL	Its record names the disease	ClinicalTrials.gov
NCT03952234	L-Citrulline Dose Finding Safety Study in MELAS	Phase 1	10		MELAS Syndrome	L-CITRULLINE	Its record names the disease	ClinicalTrials.gov
NCT01603446	L-arginine Therapy on Endothelium-dependent Vasodilation & Mitochondrial Metabolism in MELAS Syndrome	Phase 2	7		MELAS Syndrome	ARGININE	Its record names the disease	ClinicalTrials.gov

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 trials3

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
32881886	DERIVED	1	PubMed
30058726	DERIVED	1	PubMed
25993630	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_0010789

GET https://conductscience.org/api/diseases/v1/MONDO_0010789/drugs

API reference For AI assistants