SCN5A sodium voltage-gated channel alpha subunit 5
ENSG00000183873 · protein coding · Open Targets profile
Pore-forming subunit of Nav1.5, a voltage-gated sodium (Nav) channel that directly mediates the depolarizing phase of action potentials in excitable membranes (PubMed:33712541, PubMed:40768348). Navs, also called VGSCs (voltage-gated sodium channels) or VDSCs (voltage-dependent sodium channels), operate by switching between closed and open conformations depending on the voltage difference across the membrane. In the open conformation they allow Na(+) ions to selectively pass through the pore, along their electrochemical gradient (PubMed:33712541, PubMed:40768348). The influx of Na(+) ions provokes membrane depolarization, initiating the propagation of electrical signals throughout cells and tissues (PubMed:1309946, PubMed:21447824, PubMed:23085483, PubMed:23420830, PubMed:25370050, PubMed:26279430, PubMed:26392562, PubMed:26776555). Nav1.5 is the predominant sodium channel expressed in myocardial cells and it is responsible for the initial upstroke of the action potential in cardiac myocytes, thereby initiating the heartbeat (PubMed:11234013, PubMed:11804990, PubMed:12569159, PubMed:1309946). Required for normal electrical conduction including formation of the infranodal ventricular conduction system and normal action potential configuration, as a result of its interaction with XIRP2 (By similarity). {ECO:0000250|UniProtKB:Q9JJV9, ECO:0000269|PubMed:11234013, ECO:0000269|PubMed:11804990, ECO:0000269|PubMed:12569159, ECO:0000269|PubMed:1309946, ECO:0000269|PubMed:19074138, ECO:0000269|PubMed:21447824, ECO:0000269|PubMed:23085483, ECO:0000269|PubMed:23420830, ECO:0000269|PubMed:24167619, ECO:0000269|PubMed:25370050, ECO:0000269|PubMed:26279430, ECO:0000269|PubMed:26392562, ECO:0000269|PubMed:26776555, ECO:0000269|PubMed:33712541, ECO:0000269|PubMed:40768348}.
Each band is a group of drugs. It runs from the kind of drug on the left to the furthest stage that drug has reached in any disease on the right. Thicker bands mean more drugs.
Approved bands are highlighted. Withdrawn means the drug was pulled from the market or from development. A combined phase counts as the later one (Phase 1/2 is shown as Phase 2). Hover a band for the drug names. Source: Open Targets Platform, fetched live.
Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.
Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.
Tractability
- Small molecule
- Approved Drug, Structure with Ligand, High-Quality Ligand, Druggable Family
- Antibody
- UniProt loc high conf, GO CC high conf, UniProt loc med conf, UniProt SigP or TMHMM, Human Protein Atlas loc
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.3009899854660034 (upper bound 0.3775799870491028). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.
Who runs trials of these drugs
Principal investigators and study chairs on 264 registry trials, updated in the last three years, of the most advanced drugs against SCN5A (vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrine), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Heather WebberThe University of Texas Health Science Center, Houston · 1 of 3 trials Phase 2+
- Abhishek DattaSoterix Medical · 1 of 2 trials Phase 2+
- Joy M SchmitzUT Houston · 1 of 2 trials Phase 2+
- Madhukar TrivediUniversity of Texas Southwestern Medical Center · 1 of 2 trials Phase 2+
- Albert AriasVirginia Commonwealth University · 1 of 1 trials Phase 2+
- Alberto Alain GabbaiRua Pedro de Toledo 655, Vila Clemento, Sao Paulo, Brazil · 1 of 1 trials Phase 2+
- All Validations Passed BadranMedical University of SC · 1 of 1 trials Phase 2+
- Andre MalbergierDepartment of Psychiatry - University of São Paulo · 1 of 1 trials Phase 2+
- Andre TappVA Puget Sound Health Care System, Tacoma and Seattle, WA and University of Washington, Department of Psychiatry and Behavioral Sciences, Seattle, WA · 1 of 1 trials Phase 2+
- Andrew VickersMemorial Sloan Kettering Cancer Center · 1 of 1 trials Phase 2+
- Apinderpreet Singh Additional ProfessorPost Graduate Institute of Medical Education and Research, Chandigarh · 1 of 1 trials Phase 2+
- Ayesha AppaUniversity of California, San Francisco · 1 of 1 trials Phase 2+
- Bruce McCarthyEmbera NeuroTherapeutics, Inc. · 1 of 1 trials Phase 2+
- Cathy J. RebackFriends Research Institute, Inc. · 1 of 1 trials Phase 2+
- Chris DanforthUniversity of Vermont · 1 of 1 trials Phase 2+
Registry trials of vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrine updated in the last three years, counted by the country group of their listed sites. A trial with sites in several groups counts in each; 248 of 253 have all sites in one country.
Site counts are not patients per country, and trials registered only outside ClinicalTrials.gov are not covered. Where sites are is a fact about the record, not a judgement of its quality. Europe is the EU and EEA, the UK and Switzerland. 11 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
119 trials of drugs against SCN5A were stopped early; 18 give no reason
Terminated, withdrawn and suspended interventional trials of vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrine, newest update first, live from ClinicalTrials.gov. Each reason is quoted exactly as the registry gives it; we do not sort reasons into safety, efficacy or business. Dates are the last registry update. Showing the 25 most recent of 119.
- terminated · 2026-09-14 · Milton S. Hershey Medical Center · matched by the registry's drug synonyms“technical difficulties in sample analysis”
- terminated · 2026-06-26 · Tsai-Wei Huang · matched by the registry's drug synonyms“The study was terminated prior to reaching the planned sample size due to slow participant accrual. Recruitment was substantially hindered by the COVID-19 pandemic, which restricted access to hospice inpatient units, and by the inherent difficulty of”
- terminated · 2026-06-02 · Bristol-Myers Squibb · matched by the registry's drug synonyms“Business objectives have changed”
- terminated · 2026-05-04 · Ottawa Heart Institute Research Corporation · matched by the registry's drug synonyms“Low recruitment. Pilot trial.”
- suspended · 2026-04-07 · University of California, Los Angeles · matched by the registry's drug synonyms“Departmental support”
- withdrawn · 2026-03-13 · Christopher D. Verrico · matched by the registry's drug synonyms“The study was closed due to the inability to acquire the study drug.”
- terminated · 2026-02-06 · Q2i, LLC · matched by the registry's drug synonyms“Enrollment was impacted by a massive epidemiological shift that led to reduced rates of presentation to the ED for opioid overdose. Despite more than a year of adjusting protocols and site expansion, we were not able to meet study enrollment goals.”
- terminated · 2025-11-14 · University of North Carolina, Chapel Hill · Fosphenytoin“Low recruitment”
- withdrawn · 2025-10-20 · University of Wisconsin, Madison · matched by the registry's drug synonyms“Lab schedule, Resources”
- withdrawn · 2025-10-20 · Mclean Hospital · matched by the registry's drug synonyms“Study was never funded because lorcaserin was pulled from FDA approval”
- terminated · 2025-06-24 · Tonix Pharmaceuticals, Inc. · matched by the registry's drug synonyms“Tonix has discontinued enrollment and terminated the Phase 2 CATALYST study (TNX-CE-CI202) because enrollment in this emergency department-based study was slower than projected. The study was not discontinued for safety or efficacy reasons.”
- withdrawn · 2025-06-03 · University of North Carolina, Chapel Hill · matched by the registry's drug synonyms“Suspended due to COVID-19. Protocol did not resume post COVID due to funding.”
- terminated · 2025-02-25 · National Institute on Drug Abuse (NIDA) · matched by the registry's drug synonyms“inadequate resources to complete study”
- terminated · 2025-02-07 · Salvatore Campanella · matched by the registry's drug synonyms“Difficulties in recruitment (single addictions become rare)”
- terminated · 2025-01-29 · NYU Langone Health · matched by the registry's drug synonyms“This study was terminated due to the departure of the Principal Investigator from the Sponsoring institution.”
- terminated · 2024-11-06 · Tyme, Inc · SM-88 used with MPS (methoxsalen, phenytoin, sirolimus)“Sponsor's decision”
- suspended · 2024-07-16 · New York State Psychiatric Institute · matched by the registry's drug synonyms“Funding”
- terminated · 2024-05-29 · Columbia University · matched by the registry's drug synonyms“PI left the institution”
- terminated · 2024-05-23 · William Stoops · Cocaine“COVID-19”
- terminated · 2024-04-08 · UCB Biopharma SRL · matched by the registry's drug synonyms“Terminated (The study stopped prematurely due to enrolment challenges, the termination was not linked to any safety issues.)”
- terminated · 2023-08-28 · Georgetown University · matched by the registry's drug synonyms“Lack of efficacy”
- terminated · 2023-08-14 · Glenn-Milo Santos · matched by the registry's drug synonyms“FDA alert regarding study drug safety”
- terminated · 2023-08-14 · Yale University · matched by the registry's drug synonyms“Could not obtain additional funding to continue the research.”
- terminated · 2023-06-23 · Noel Jabbour · matched by the registry's drug synonyms“End of funding”
- terminated · 2023-04-04 · Escola Superior de Ciencias da Saude · matched by the registry's drug synonyms“COVID 19 pandemy”
Paediatric incentives and obligations for SCN5A's drugs
For vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrine: what EMA and FDA publish about paediatric plans, orphan status and pediatric approvals, and how many registered studies take children. Each line is a yes, no or not published fact from the record linked under it; none of it is a forecast.
- FDA pediatric target list: no · SCN5A is not on the listRule: Target is on FDA's Pediatric Molecular Target List (relevant targets). Source: FDA Pediatric Molecular Target List (Listed as February 2026 on the FDA Pediatric Oncology page; the file served is 9.7.22_the_relevant_molecular_target_list.xlsx, whose Summary of Changes sheet is headed 'SEPTEMBER 2022 UPDATE' (footnote date 2022-09-01)), 2026-11-11
- EU paediatric investigation plan: yes · EMA/PE/0000248063 (eslicarbazepine acetate, Treatment of epilepsy with partial-onset seizures): PM: decision on the application for modification of an agreed PIP, 10 Jul 2025; EMEA-003337-PIP01-22 (diclofenac;orphenadrine (citrate), Treatment of pain): W: decision granting a waiver in all age groups for all conditions or indications, 31 Jan 2023; EMEA-001171-PIP01-11-M02 (procaine (hydrochloride);xylitol;potassium chloride;magnesium sulfate heptahydrate, Cardioplegia): PM: decision on the application for modification of an agreed PIP, 11 Aug 2021; EMEA-000226-PIP01-08 (vernakalant hydrochloride, Atrial fibrillation): W: decision granting a waiver in all age groups for all conditions or indications, 24 Jun 2008Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA data download (dated snapshot), 2026-11-11
- EU orphan designation: no · No EU orphan designation names vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrineRule: An EU orphan designation lists the drug as its active substance (exact name) or belongs to a medicine that does; withdrawn and expired designations are listed with their status. Source: EMA data download (dated snapshot), 2026-11-11
- Authorised in the EU: yes · Zebinix: authorised since 21 Apr 2009; Brinavess: authorised since 1 Sept 2010; Exalief: lapsed since 21 Apr 2009Rule: An EMA medicine record lists the drug as its active substance or INN, exact name; national-only authorisations are not in this file. Source: EMA data download (dated snapshot), 2026-11-11
- FDA orphan designation: not published · FDA's orphan designation database turns away automated requests, so this app does not read it; search it by hand at the linkRule: Would be: an FDA orphan designation names the drug. Source: FDA orphan designations search
- FDA pediatric oncology approval: no · FDA's table lists no pediatric approval of vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrineRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-12
- Registered studies open to children: yes · 145 interventional studies of vernakalant, tocainide, procaine, cocaine, proparacaine, fosphenytoin, eslicarbazepine, procainamide, phenacemide, disopyramide, phenytoin, orphenadrine include patients under 18; 20 are recruiting, not yet recruiting or activeRule: The registry's age group includes Child (birth to 17 years), which also counts studies that set no minimum age. Source: ClinicalTrials.gov search, 2026-11-12
Rare pediatric disease voucher. FDA, content current as of 29 Apr 2026: “As of February 3, 2026, enactment of the Consolidated Appropriations Act, 2026, the rare pediatric disease PRV program will sunset after September 30, 2029. FDA may not award any PRVs under this program after September 30, 2029.” Voucher sale prices are not shown here; they appear only as company-reported news. FDA: Rare Pediatric Disease Designation and Priority Review Voucher Programs
All 81 drugs and clinical candidates as a table
Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.