Disease · MONDO_0009637
inborn mitochondrial myopathy
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. inborn mitochondrial myopathy. Diseases, record MONDO_0009637. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0009637
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 2 of its 9 drugs, from ClinicalTrials.gov and TTD
- Open Targets
Drugs9
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 |
|---|---|---|---|---|---|---|---|---|
| ELAMIPRETIDE | CHEMBL3833370 | Protein | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| ELAMIPRETIDE HYDROCHLORIDE | CHEMBL4298161 | Protein | Phase 3 | TTD | 1 | 0 | 0 | Drugs and targets |
| MAVODELPAR | CHEMBL5314562 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| BOCIDELPAR | CHEMBL5095171 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 1 | 1 | Drugs and targets |
| BEZAFIBRATE | CHEMBL264374 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| OMAVELOXOLONE | CHEMBL4303525 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NICOTINAMIDE RIBOSIDE | CHEMBL438497 | Small molecule | Phase 1 | TTD | 1 | 0 | 0 | Drugs and targets |
| THIOCTIC ACID | CHEMBL134342 | Small molecule | Unknown | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| BOCIDELPAR SULFATE | CHEMBL5095400 | Small molecule | Unknown | USAN | 1 | 0 | 0 | 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 |
|---|---|---|---|---|---|---|
| NFE2L2 | Nuclear factor erythroid 2-related factor 2 | 1 | Nuclear factor erythroid 2-related factor 2 activator | Phase 2 | https://conductscience.org/library/genes/4780 | Drugs and targets |
| PPARA | Peroxisome proliferator-activated receptor | 1 | Peroxisome proliferator-activated receptor agonist | Phase 2 | https://conductscience.org/library/genes/5465 | Drugs and targets |
| PPARD | Peroxisome proliferator-activated receptor | 1 | Peroxisome proliferator-activated receptor agonist | Phase 2 | https://conductscience.org/library/genes/5467 | Drugs and targets |
| PPARG | Peroxisome proliferator-activated receptor | 1 | Peroxisome proliferator-activated receptor agonist | Phase 2 | https://conductscience.org/library/genes/5468 | 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 |
|---|---|---|---|---|---|---|---|---|
| NCT03323749 | A Trial to Evaluate Safety and Efficacy of Elamipretide Primary Mitochondrial Myopathy Followed by Open-Label Extension | Phase 3 | 218 | 2021-04-02 | Primary Mitochondrial Myopathy | ELAMIPRETIDE | Open Targets' reading, confirmed by NLM's indexing of its papers | ClinicalTrials.gov |
| NCT04535609 | An Efficacy and Safety Study of 24 Week Treatment With Mavodelpar (REN001) in Primary Mitochondrial Myopathy Patients | Phase 2 | 213 | 2024-04-03 | Primary Mitochondrial Myopathy | MAVODELPAR | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT05267574 | An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead) | Phase 2/3 | 155 | 2024-05-28 | Primary Mitochondrial Myopathy | MAVODELPAR | Open Targets' reading of its record | ClinicalTrials.gov |
| NCT02255422 | RTA 408 Capsules in Patients With Mitochondrial Myopathy - MOTOR | Phase 2 | 53 | 2020-09-17 | MItochondrial Myopathies | OMAVELOXOLONE | Its record names the disease | ClinicalTrials.gov |
| NCT02367014 | Safety, Tolerability, and Efficacy of MTP-131 for the Treatment of Mitochondrial Myopathy | Phase 1/2 | 36 | 2019-12-27 | Mitochondrial Myopathy | ELAMIPRETIDE | Its record names the disease | ClinicalTrials.gov |
| NCT04641962 | A Study to Evaluate ASP0367 in Participants With Primary Mitochondrial Myopathy | Phase 2 | 34 | 2025-06-17 | Primary Mitochondrial Myopathy | BOCIDELPAR | Its record names the disease | ClinicalTrials.gov |
| NCT03862846 | A Study of the Safety of REN001 in Patients With Primary Mitochondrial Myopathy | Phase 1 | 23 | Primary Mitochondrial Myopathy | MAVODELPAR | Open Targets' reading of its record | ClinicalTrials.gov | |
| NCT02398201 | A Study of Bezafibrate in Mitochondrial Myopathy | Phase 2 | 6 | Mitochondrial Diseases | BEZAFIBRATE | Its record names the disease | ClinicalTrials.gov | |
| NCT00004770 | Pilot Compassionate Use Study of Thioctic Acid Treatment in Mitochondrial Myopathy | Not applicable | 1 | Mitochondrial Myopathy | THIOCTIC ACID | 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 |
|---|---|---|---|---|---|
| Mavodelpar vs Matched Placebo: deaths from any cause | 1 of 108 | 0 of 104 | 212 | NCT04535609 | Trial comparisons |
| Mavodelpar vs Matched Placebo: serious adverse events | 8 of 108 | 7 of 104 | 212 | NCT04535609 | Trial comparisons |
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.
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_0009637
GET https://conductscience.org/api/diseases/v1/MONDO_0009637/drugs