Disease · MONDO_0000437
cerebellar ataxia
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. cerebellar ataxia. Diseases, record MONDO_0000437. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0000437
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
- Ataxia, ataxia syndrome, cerebellar ataxias, spinocerebellar ataxia, spinocerebellar degeneration
- Open Targets
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 |
|---|---|---|---|---|---|---|---|---|
| TRORILUZOLE | CHEMBL4297586 | Protein | Phase 3 | ClinicalTrials.gov | 5 | 4 | 4 | Drugs and targets |
| DALFAMPRIDINE | CHEMBL284348 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 2 | Drugs and targets |
| RILUZOLE | CHEMBL744 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| ROVATIRELIN | CHEMBL4297622 | Small molecule | Phase 3 | ClinicalTrials.gov | 3 | 3 | 3 | Drugs and targets |
| ACETYLLEUCINE | CHEMBL1875805 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| ACETAZOLAMIDE | CHEMBL20 | Small molecule | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| TREHALOSE | CHEMBL1236395 | Oligosaccharide | Phase 2/3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| HUMAN IMMUNOGLOBULIN G | CHEMBL1201599 | Phase 2 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets | |
| RIMTUZALCAP | CHEMBL4650330 | Small molecule | Phase 2 | ClinicalTrials.gov | 2 | 1 | 1 | Drugs and targets |
| NILOTINIB | CHEMBL255863 | Small molecule | Phase 2 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
1 to 10 of 13
See all 13 drugsTargets58
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 |
|---|---|---|---|---|---|---|
| ABL1 | Bcr/Abl fusion protein | 1 | Bcr/Abl fusion protein inhibitor; Tyrosine-protein kinase ABL inhibitor | Phase 2 | https://conductscience.org/library/genes/25 | Drugs and targets |
| BCR | Bcr/Abl fusion protein | 1 | Bcr/Abl fusion protein inhibitor | Phase 2 | https://conductscience.org/library/genes/613 | Drugs and targets |
| CA1 | Carbonic anhydrase 1 | 1 | Carbonic anhydrase I inhibitor | Phase 2/3 | https://conductscience.org/library/genes/759 | Drugs and targets |
| CA12 | Carbonic anhydrase 12 | 1 | Carbonic anhydrase XII inhibitor | Phase 2/3 | https://conductscience.org/library/genes/771 | Drugs and targets |
| CA2 | Carbonic anhydrase 2 | 1 | Carbonic anhydrase II inhibitor | Phase 2/3 | https://conductscience.org/library/genes/760 | Drugs and targets |
| CA4 | Carbonic anhydrase 4 | 1 | Carbonic anhydrase IV inhibitor | Phase 2/3 | https://conductscience.org/library/genes/762 | Drugs and targets |
| KCNA1 | Voltage-gated potassium channel | 1 | Voltage-gated potassium channel blocker | Phase 3 | https://conductscience.org/library/genes/3736 | Drugs and targets |
| KCNA10 | Voltage-gated potassium channel | 1 | Voltage-gated potassium channel blocker | Phase 3 | https://conductscience.org/library/genes/3744 | Drugs and targets |
| KCNA2 | Voltage-gated potassium channel | 1 | Voltage-gated potassium channel blocker | Phase 3 | https://conductscience.org/library/genes/3737 | Drugs and targets |
| KCNA3 | Voltage-gated potassium channel | 1 | Voltage-gated potassium channel blocker | Phase 3 | https://conductscience.org/library/genes/3738 | Drugs and targets |
1 to 10 of 58
See all 58 targetsTrials19
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 |
|---|---|---|---|---|---|---|---|---|
| NCT03701399 | Troriluzole in Adult Participants With Spinocerebellar Ataxia | Phase 3 | 299 | 2025-02-10 | Spinocerebellar Ataxias, Spinocerebellar Ataxia Type 1, Spinocerebellar Ataxia Type 2, Spinocerebellar Ataxia Type 3, Spinocerebellar Ataxia Type 6, Spinocerebellar Ataxia Type 7, Spinocerebellar Ataxia Type 8, Spinocerebellar Ataxia Type 10 | TRORILUZOLE | Its record names the disease | ClinicalTrials.gov |
| NCT02960893 | Trial in Adult Participants With Spinocerebellar Ataxia (SCA) | Phase 2/3 | 141 | 2020-08-14 | Spinocerebellar Ataxias, Spinocerebellar Ataxia Genotype Type 1, Spinocerebellar Ataxia Genotype Type 2, Spinocerebellar Ataxia Genotype Type 3, Spinocerebellar Ataxia Genotype Type 6, Spinocerebellar Ataxia Genotype Type 7, Spinocerebellar Ataxia Genotype Type 8, Spinocerebellar Ataxia Genotype Type 10 | TRORILUZOLE | Its record names the disease | ClinicalTrials.gov |
| NCT04679389 | Acetazolamide Efficacy in Ataxia in PMM2-CDG | Phase 2/3 | 25 | 2025-03-20 | Pmm2-CDG, CDG1A | ACETAZOLAMIDE | Its record names the disease | ClinicalTrials.gov |
| NCT01811706 | Dalfampridine and Gait in Spinocerebellar Ataxias | Not applicable | 20 | 2015-01-12 | Spinocerebellar Ataxias Type 1, Spinocerebellar Ataxias Type 2, Spinocerebellar Ataxias Type 3, Spinocerebellar Ataxias Type 6 | DALFAMPRIDINE | Its record names the disease | ClinicalTrials.gov |
| NCT00202930 | Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma | Phase 2 | 4 | 2021-01-22 | Neuroblastoma, Opsoclonus-myoclonus | RITUXIMAB | Its record names the disease | ClinicalTrials.gov |
| NCT06529146 | Real-World Data Study of Troriluzole-Treated Patients With Spinocerebellar Ataxia (SCA) Compared to a Matched Natural History Control | 909 | Spinocerebellar Ataxias | TRORILUZOLE | Its record names the disease | ClinicalTrials.gov | ||
| NCT01970098 | A Confirmatory Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | Phase 3 | 374 | Spinocerebellar Degeneration | ROVATIRELIN | Its record names the disease | ClinicalTrials.gov | |
| NCT01384435 | A Phase II Double Blind Comparative Study of KPS-0373 in Patients With Spinocerebellar Degeneration (SCD) | Phase 2 | 200 | SCD | ROVATIRELIN | Its record names the disease | ClinicalTrials.gov | |
| NCT07040137 | Confirmatory Study 3 of KPS-0373 in Patients With Spinocerebellar Degeneration | Phase 3 | 142 | Spinocerebellar Degeneration | ROVATIRELIN | Its record names the disease | ClinicalTrials.gov | |
| NCT03932669 | Effect of Nilotinib in Cerebellar Ataxia Patients | Phase 2 | 70 | Ataxia, Cerebellar, Ataxia, Progressive | NILOTINIB | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 19
See all 19 trialsComparisons in its trials8
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 |
|---|---|---|---|---|---|
| Troriluzole vs Placebo: deaths from any cause | 1 of 109 | 1 of 109 | 218 | NCT03701399 | Trial comparisons |
| Troriluzole vs Placebo: serious adverse events | 6 of 108 | 8 of 109 | 217 | NCT03701399 | Trial comparisons |
| Dalfampridine vs Placebo: serious adverse events | 0 of 20 | 0 of 19 | 39 | NCT01811706 | Trial comparisons |
| Acetazolamide vs Placebo: Abnormal Blood pH Value | 0 of 13 | 1 of 12 | 25 | NCT04679389 | Trial comparisons |
| Acetazolamide vs Placebo: Electrolyte Balance Testing | 0 of 13 | 1 of 12 | 25 | NCT04679389 | Trial comparisons |
| Acetazolamide vs Placebo: Urine Calcium Excretion Testing | 0 of 13 | 0 of 12 | 25 | NCT04679389 | Trial comparisons |
| Acetazolamide vs Placebo: deaths from any cause | 0 of 13 | 0 of 12 | 25 | NCT04679389 | Trial comparisons |
| Acetazolamide vs Placebo: serious adverse events | 1 of 13 | 3 of 12 | 25 | NCT04679389 | Trial comparisons |
Papers from its trials8
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_0000437
GET https://conductscience.org/api/diseases/v1/MONDO_0000437/targets?page=2