Disease · MONDO_0100339
Friedreich ataxia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Friedreich ataxia. Diseases, record MONDO_0100339. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0100339
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.
- Other names
- FA, FRDA, Friedreich's Ataxia, Friedreich's tabes, Hereditary ataxia-Friedreich’s type
- Open Targets
- Highest stage of its drugs
- Approval, for 2 of its 25 drugs, from EMA Human Drugs, FDA and TTD
- Open Targets
Drugs25
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 | TTD | 11 | 9 | 9 | Drugs and targets |
| OMAVELOXOLONE | CHEMBL4303525 | Small molecule | Approval | EMA Human Drugs, FDA and TTD | 9 | 6 | 6 | Drugs and targets |
| INTERFERON GAMMA-1B | CHEMBL1201564 | Protein | Phase 3 | ClinicalTrials.gov and TTD | 7 | 6 | 5 | Drugs and targets |
| VATIQUINONE | CHEMBL1812161 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 5 | 5 | Drugs and targets |
| BOTULINUM TOXIN TYPE A | CHEMBL4297862 | Protein | Phase 3 | ClinicalTrials.gov and TTD | 4 | 2 | 0 | Drugs and targets |
| PIOGLITAZONE | CHEMBL595 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| VARENICLINE | CHEMBL1076903 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| NOMLABOFUSP | CHEMBL5095325 | Protein | Phase 2 | ClinicalTrials.gov and TTD | 7 | 5 | 5 | Drugs and targets |
| DEFERIPRONE | CHEMBL70927 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| EPOETIN ALFA | CHEMBL1201565 | Protein | Phase 2 | ClinicalTrials.gov | 2 | 2 | 1 | Drugs and targets |
1 to 10 of 25
See all 25 drugsTargets11
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 |
|---|---|---|---|---|---|---|
| EPOR | Erythropoietin receptor | 3 | Erythropoietin receptor agonist | Phase 2 | https://conductscience.org/library/genes/2057 | Drugs and targets |
| PPARG | Peroxisome proliferator-activated receptor gamma | 2 | Peroxisome proliferator-activated receptor gamma agonist | Phase 3 | https://conductscience.org/library/genes/5468 | Drugs and targets |
| CHRNA4 | Neuronal acetylcholine receptor; alpha4/beta2 | 1 | Neuronal acetylcholine receptor; alpha4/beta2 agonist; Neuronal acetylcholine receptor; alpha4/beta2 partial agonist | Phase 2/3 | https://conductscience.org/library/genes/1137 | Drugs and targets |
| CHRNB2 | Neuronal acetylcholine receptor; alpha4/beta2 | 1 | Neuronal acetylcholine receptor; alpha4/beta2 agonist; Neuronal acetylcholine receptor; alpha4/beta2 partial agonist | Phase 2/3 | https://conductscience.org/library/genes/1141 | Drugs and targets |
| HMGCR | 3-hydroxy-3-methylglutaryl-coenzyme A reductase | 1 | HMG-CoA reductase inhibitor | Early phase 1 | https://conductscience.org/library/genes/3156 | Drugs and targets |
| IFNGR1 | Interferon gamma receptor | 1 | Interferon gamma receptor agonist | Phase 3 | https://conductscience.org/library/genes/3459 | Drugs and targets |
| IFNGR2 | Interferon gamma receptor | 1 | Interferon gamma receptor agonist | Phase 3 | https://conductscience.org/library/genes/3460 | Drugs and targets |
| NFE2L2 | Nuclear factor erythroid 2-related factor 2 | 1 | Nuclear factor erythroid 2-related factor 2 activator | Approval | https://conductscience.org/library/genes/4780 | Drugs and targets |
| NQO1 | NAD(P)H dehydrogenase [quinone] 1 | 1 | Quinone reductase 1 modulator | Phase 3 | https://conductscience.org/library/genes/1728 | Drugs and targets |
| NR3C1 | Glucocorticoid receptor | 1 | Glucocorticoid receptor agonist | Early phase 1 | https://conductscience.org/library/genes/2908 | Drugs and targets |
1 to 10 of 11
See all 11 targetsTrials49
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. 4 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT00905268 | A Study of Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia (FRDA) Patients | Phase 3 | 232 | 2016-06-27 | Friedreich's Ataxia | IDEBENONE | Its record names the disease | ClinicalTrials.gov |
| NCT00993967 | Long-Term Safety and Tolerability of Idebenone in Friedreich's Ataxia Patients (MICONOS Extension) | Phase 3 | 200 | 2018-03-05 | Freidreich's Ataxia | IDEBENONE | Its record names the disease | ClinicalTrials.gov |
| NCT02255435 | A Study to Learn About the Effects and Safety of RTA 408 (Omaveloxolone) in People Aged 16 to 40 With Friedreich's Ataxia | Phase 2 | 172 | 2022-11-29 | Friedreich Ataxia | OMAVELOXOLONE | Its record names the disease | ClinicalTrials.gov |
| NCT02415127 | Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia | Phase 3 | 92 | 2017-12-08 | Friedreich's Ataxia | INTERFERON GAMMA-1B | Its record names the disease | ClinicalTrials.gov |
| NCT02593773 | Safety, Tolerability and Efficacy of ACTIMMUNE® Dose Escalation in Friedreich's Ataxia Study | Phase 3 | 86 | 2018-05-18 | Friedreich's Ataxia | INTERFERON GAMMA-1B | Its record names the disease | ClinicalTrials.gov |
| NCT00537680 | Study to Assess the Efficacy, Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia | Phase 3 | 70 | 2011-12-29 | Friedreich's Ataxia | IDEBENONE | Its record names the disease | ClinicalTrials.gov |
| NCT00697073 | Study to Assess the Safety and Tolerability of Idebenone in the Treatment of Friedreich's Ataxia Patients | Phase 3 | 68 | 2011-08-04 | Friedreich's Ataxia | IDEBENONE | Its record names the disease | ClinicalTrials.gov |
| NCT03214588 | Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia | Phase 2 | 67 | 2019-12-19 | Friedreich Ataxia | LUVADAXISTAT | Its record names the disease | ClinicalTrials.gov |
| NCT02797080 | Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia | Phase 3 | 38 | 2018-05-01 | Friedreich's Ataxia | INTERFERON GAMMA-1B | Its record names the disease | ClinicalTrials.gov |
| NCT01303406 | Patient Reported Outcomes in Friedreich's Ataxia Patients After Withdrawal From Treatment With Idebenone (PROTI) | Phase 3 | 29 | 2016-03-09 | Friedreich's Ataxia | IDEBENONE | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 49
See all 49 trialsComparisons in its trials6
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 |
|---|---|---|---|---|---|
| Interferon γ-1b vs Placebo: serious adverse events | 1 of 47 | 2 of 45 | 92 | NCT02415127 | Trial comparisons |
| Idebenone vs Placebo: Patient Assessment of Treatment Assignment: Comparison of the Proportions of Patients Randomised to Idebenone and Placebo Who Assessed That They Received Idebenone | 8 of 16 | 6 of 13 | 29 | NCT01303406 | Trial comparisons |
| Idebenone vs Placebo: serious adverse events | 1 of 16 | 0 of 13 | 29 | NCT01303406 | Trial comparisons |
| EGb 761® 120 mg vs Placebo: serious adverse events | 1 of 11 | 1 of 11 | 22 | NCT00824512 | Trial comparisons |
| Low Dose (20-30mg) vs High Dose (40-60 mg): deaths from any cause | 0 of 8 | 0 of 8 | 16 | NCT05168774 | Trial comparisons |
| Low Dose (20-30mg) vs High Dose (40-60 mg): serious adverse events | 2 of 8 | 2 of 8 | 16 | NCT05168774 | Trial comparisons |
Papers from its trials16
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 |
|---|---|---|---|
| 39201893 | RESULT | 1 | PubMed |
| 21779958 | RESULT | 2 | PubMed |
| 40614535 | DERIVED | 1 | PubMed |
| 36524101 | DERIVED | 1 | PubMed |
| 33324899 | DERIVED | 1 | PubMed |
| 33068037 | DERIVED | 1 | PubMed |
| 32677106 | DERIVED | 1 | PubMed |
| 31495483 | DERIVED | 1 | PubMed |
| 30656180 | DERIVED | 1 | PubMed |
| 24794816 | DERIVED | 1 | PubMed |
1 to 10 of 16
See all 16 papers from its trialsData 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_0100339
GET https://conductscience.org/api/diseases/v1/MONDO_0100339/drugs?page=2