Disease · MONDO_0100135
Dravet syndrome
6 sourcesdiseases-2026-09-18-0.1.0
Download JSON
Cite this record
ConductScience Library. Dravet syndrome. Diseases, record MONDO_0100135. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0100135
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
- DS, Dravet, SME, SMEB, myoclonic epilepsy, severe, of infancy, severe myoclonic epilepsy of infancy
- Open Targets
- Highest stage of its drugs
- Approval, for 4 of its 11 drugs, from ClinicalTrials.gov, PMDA and TTD
- Open Targets
Drugs11
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 |
|---|---|---|---|---|---|---|---|---|
| CANNABIDIOL | CHEMBL190461 | Small molecule | Approval | TTD | 7 | 6 | 6 | Drugs and targets |
| FENFLURAMINE HYDROCHLORIDE | CHEMBL2106217 | Small molecule | Approval | ClinicalTrials.gov and PMDA | 7 | 6 | 6 | Drugs and targets |
| FENFLURAMINE | CHEMBL87493 | Small molecule | Approval | ClinicalTrials.gov | 5 | 4 | 4 | Drugs and targets |
| STIRIPENTOL | CHEMBL1983350 | Small molecule | Approval | ClinicalTrials.gov, PMDA and TTD | 5 | 3 | 3 | Drugs and targets |
| SOTICLESTAT | CHEMBL4298172 | Small molecule | Phase 3 | ClinicalTrials.gov | 6 | 5 | 5 | Drugs and targets |
| CLOBAZAM | CHEMBL70418 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| LORCASERIN | CHEMBL360328 | Small molecule | Phase 3 | ClinicalTrials.gov | 2 | 2 | 2 | Drugs and targets |
| DRONABINOL | CHEMBL465 | Small molecule | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ZOREVUNERSEN | CHEMBL5095386 | Oligonucleotide | Phase 3 | ClinicalTrials.gov | 1 | 1 | 1 | Drugs and targets |
| ATALUREN | CHEMBL256997 | Small molecule | Phase 2 | ClinicalTrials.gov and TTD | 2 | 1 | 1 | Drugs and targets |
1 to 10 of 11
See all 11 drugsTargets103
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 |
|---|---|---|---|---|---|---|
| HTR2C | 5-hydroxytryptamine receptor 2C | 3 | Serotonin 2c (5-HT2c) receptor agonist | Approval | https://conductscience.org/library/genes/3358 | Drugs and targets |
| CNR1 | Cannabinoid receptor 1 | 2 | Cannabinoid CB1 receptor agonist; Cannabinoid CB1 receptor negative allosteric modulator | Approval | https://conductscience.org/library/genes/1268 | Drugs and targets |
| HTR1D | 5-hydroxytryptamine receptor 1D | 2 | Serotonin 1d (5-HT1d) receptor agonist | Approval | https://conductscience.org/library/genes/3352 | Drugs and targets |
| SIGMAR1 | Sigma non-opioid intracellular receptor 1 | 2 | Sigma opioid receptor antagonist | Approval | https://conductscience.org/library/genes/10280 | Drugs and targets |
| CACNA1C | Voltage-gated L-type calcium channel | 1 | Voltage-gated L-type calcium channel blocker | Phase 2 | https://conductscience.org/library/genes/775 | Drugs and targets |
| CACNA1D | Voltage-gated L-type calcium channel | 1 | Voltage-gated L-type calcium channel blocker | Phase 2 | https://conductscience.org/library/genes/776 | Drugs and targets |
| CACNA1F | Voltage-gated L-type calcium channel | 1 | Voltage-gated L-type calcium channel blocker | Phase 2 | https://conductscience.org/library/genes/778 | Drugs and targets |
| CACNA1S | Voltage-gated L-type calcium channel | 1 | Voltage-gated L-type calcium channel blocker | Phase 2 | https://conductscience.org/library/genes/779 | Drugs and targets |
| CYP46A1 | Cholesterol 24-hydroxylase | 1 | Cholesterol 24-hydroxylase inhibitor | Phase 3 | https://conductscience.org/library/genes/10858 | Drugs and targets |
| FAU | 80S Ribosome | 1 | 80S Ribosome modulator | Phase 2 | https://conductscience.org/library/genes/2197 | Drugs and targets |
1 to 10 of 103
See all 103 targetsTrials31
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 |
|---|---|---|---|---|---|---|---|---|
| NCT03936777 | A Study to Investigate the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution in Children and Adults With Epileptic Encephalopathy Including Dravet Syndrome and Lennox-Gastaut Syndrome | Phase 3 | 412 | 2025-11-17 | Dravet Syndrome, Lennox Gastaut Syndrome, Epileptic Encephalopathy | FENFLURAMINE HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02823145 | An Open-Label Extension Trial to Assess the Long-Term Safety of ZX008 (Fenfluramine Hydrochloride HCl) Oral Solution in Children and Young Adults With Dravet Syndrome | Phase 3 | 375 | 2023-09-25 | Dravet Syndrome | FENFLURAMINE HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02682927 | A Trial of Two Fixed Doses of ZX008 (Fenfluramine HCl) in Children and Young Adults With Dravet Syndrome | Phase 3 | 262 | 2022-10-19 | Dravet Syndrome, Seizure Disorder | FENFLURAMINE HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT03635073 | A Study of Soticlestat in Adults and Children With Rare Epilepsies | Phase 2 | 156 | 2026-03-19 | Epilepsy, Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS) | SOTICLESTAT | Its record names the disease | ClinicalTrials.gov |
| NCT04940624 | A Study of Soticlestat as an Add-on Therapy in Children and Young Adults With Dravet Syndrome | Phase 3 | 144 | 2025-01-01 | Dravet Syndrome (DS) | SOTICLESTAT | Its record names the disease | ClinicalTrials.gov |
| NCT03650452 | A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies | Phase 2 | 141 | 2021-02-18 | Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome | SOTICLESTAT | Its record names the disease | ClinicalTrials.gov |
| NCT02926898 | A 2-Part Study to Investigate the Dose-Ranging Safety and Pharmacokinetics, Followed by the Efficacy and Safety of ZX008 (Fenfluramine Hydrochloride) Oral Solution as an Adjunctive Therapy in Children ≥ 2 Years Old and Young Adults With Dravet Syndrome | Phase 3 | 87 | 2022-10-19 | Dravet Syndrome | FENFLURAMINE HYDROCHLORIDE | Its record names the disease | ClinicalTrials.gov |
| NCT02318602 | Cannabidiol Oral Solution as an Adjunctive Treatment for Treatment-resistant Seizure Disorder | Phase 3 | 52 | 2018-07-26 | Seizures | CANNABIDIOL | Its record names the disease | ClinicalTrials.gov |
| NCT04572243 | A Study of Lorcaserin as Adjunctive Treatment in Participants With Dravet Syndrome | Phase 3 | 22 | 2025-10-08 | Epilepsies, Myoclonic | LORCASERIN | Its record names the disease | ClinicalTrials.gov |
| NCT02758626 | Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome | Phase 2 | 15 | 2023-02-16 | Epilepsy | ATALUREN | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 31
See all 31 trialsComparisons in its trials13
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 |
|---|---|---|---|---|---|
| Soticlestat vs Placebo: deaths from any cause | 1 of 73 | 0 of 71 | 144 | NCT04940624 | Trial comparisons |
| Soticlestat vs Placebo: serious adverse events | 7 of 73 | 10 of 71 | 144 | NCT04940624 | Trial comparisons |
| TAK-935 vs Placebo: deaths from any cause | 0 of 71 | 0 of 70 | 141 | NCT03650452 | Trial comparisons |
| TAK-935 vs Placebo: serious adverse events | 11 of 71 | 13 of 70 | 141 | NCT03650452 | Trial comparisons |
| Cohort 2: ZX008 0.5 mg/kg/Day vs Cohort 2: Matching Placebo: deaths from any cause | 0 of 43 | 0 of 44 | 87 | NCT02926898 | Trial comparisons |
| Cohort 2: ZX008 0.5 mg/kg/Day vs Cohort 2: Matching Placebo: serious adverse events | 6 of 43 | 7 of 44 | 87 | NCT02926898 | Trial comparisons |
| Ataluren vs Placebo: deaths from any cause | 0 of 15 | 0 of 14 | 29 | NCT02758626 | Trial comparisons |
| Ataluren vs Placebo: serious adverse events | 7 of 15 | 1 of 14 | 29 | NCT02758626 | Trial comparisons |
| Core Study: Lorcaserin vs Core Study: Placebo: Core Study: Number of Participants With Treatment-emergent Adverse Events (TEAEs) | 9 of 11 | 7 of 10 | 21 | NCT04572243 | Trial comparisons |
| Tuberous Sclerosis Complex vs Dravet Syndrome: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) | 2 of 2 | 1 of 1 | 3 | NCT04485104 | Trial comparisons |
1 to 10 of 13
See all 13 comparisons in its trialsPapers from its trials9
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_0100135
GET https://conductscience.org/api/diseases/v1/MONDO_0100135/targets?page=2