SCNN1A sodium channel epithelial 1 subunit alpha
ENSG00000111319 · protein coding · Open Targets profile
This is one of the three pore-forming subunits of the heterotrimeric epithelial sodium channel (ENaC), a critical regulator of sodium balance and fluid homeostasis (PubMed:30251954, PubMed:32729833, PubMed:8023962, PubMed:8278374, PubMed:9792722). ENaC operates in epithelial tissues, where it mediates the electrodiffusion of sodium ions from extracellular fluid through the apical membrane of cells, with water following osmotically (PubMed:24124190, PubMed:28710092, PubMed:8278374). It plays a key role in maintaining sodium homeostasis through electrogenic sodium reabsorption in the kidneys (PubMed:12107247). Additionally, ENaC is essential for airway surface liquid homeostasis, which is crucial for proper mucus clearance (PubMed:24124190, PubMed:28710092). {ECO:0000269|PubMed:12107247, ECO:0000269|PubMed:24124190, ECO:0000269|PubMed:28710092, ECO:0000269|PubMed:30251954, ECO:0000269|PubMed:32729833, ECO:0000269|PubMed:8023962, ECO:0000269|PubMed:8278374, ECO:0000269|PubMed:9792722}.
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 Withdrawn means the drug was pulled from the market or from development.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, Druggable Family
- Antibody
- UniProt loc high conf, GO CC high conf, UniProt SigP or TMHMM
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.9475499987602234 (upper bound 1.1648999452590942). 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 15 registry trials, updated in the last three years, of the most advanced drugs against SCNN1A (amiloride, triamterene, cofirasersen, idrevloride), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Adriana C FortiCentro de Estudos de Diabetes e Hipertensão · 1 of 1 trials Phase 2+
- Camila Manrique AcevedoUniversity of Missouri-Columbia · 1 of 1 trials Phase 2+
- Daniela G BarbieriCEDOES - Centro de Diagnóstico e Pesquisa de Osteoporose do Espírito Santo · 1 of 1 trials Phase 2+
- DECHANET AlineAssistance Publique - Hôpitaux de Paris (AP-HP) · 1 of 1 trials Phase 2+
- Fábio José C FucciInstituto de Moléstias Cardiovasculares de Tatuí · 1 of 1 trials Phase 2+
- Fernando Augusto A CostaFGM - Clínica Paulista de Doenças Cardiovasculares · 1 of 1 trials Phase 2+
- Ferruh ArtuncUniversity Hospital Tuebingen · 1 of 1 trials Phase 2+
- Hamid DjalilianUniversity of California, Irvine · 1 of 1 trials Phase 2+
- Humberto FreitasHospital São Camilo · 1 of 1 trials Phase 2+
- Ian WilkinsonCambridge University Hospitals NHS Foundation Trust · 1 of 1 trials Phase 2+
- Jose Carlos A AyobCentro de Pesquisas Clínicas do Instituto de Moléstias Cardiovasculares · 1 of 1 trials Phase 2+
- M. SalihErasmus Medical Center · 1 of 1 trials Phase 2+
- Marise L CastroIMA - Instituto de Medicina Avançada · 1 of 1 trials Phase 2+
- Michel LANTERI-MINETCentre Hospitalier Universitaire de Nice · 1 of 1 trials Phase 2+
- Paulo Cesar V JardimLiga de Hipertensão Arterial - Universidade Federal de Goiás · 1 of 1 trials Phase 2+
Registry trials of amiloride, triamterene, cofirasersen, idrevloride 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; 15 of 15 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. Source: ClinicalTrials.gov, fetched live.
9 trials of drugs against SCNN1A were stopped early
Terminated, withdrawn and suspended interventional trials of amiloride, triamterene, cofirasersen, idrevloride, 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 9 most recent of 9.
- terminated · 2025-01-14 · University Hospital Tuebingen · Amiloride“Low recruiting rate and decision to evaluate as pilot study”
- terminated · 2022-06-03 · Eurofarma Laboratorios S.A. · matched by the registry's drug synonyms“EF decided suspended the study because the investigational product was changed.”
- terminated · 2018-07-20 · State University of New York - Upstate Medical University · amiloride“Study stopped due to lack of recruitment”
- terminated · 2018-05-11 · University of New Mexico · Amiloride“Currently closed for data analysis.”
- withdrawn · 2015-07-09 · Gilead Sciences · matched by the registry's drug synonyms“Development program terminated.”
- terminated · 2015-06-09 · St George's, University of London · Amiloride 5 mg bd“Insufficient accrual rate”
- withdrawn · 2014-05-28 · VA Salt Lake City Health Care System · amiloride“lack of enrollment”
- terminated · 2012-10-15 · Assistance Publique - Hôpitaux de Paris · matched by the registry's drug synonyms“end of recruitment period was reach”
- terminated · 2009-05-06 · University of Aarhus · Amiloride, hydrochlorothiazide“Due to recent findings relating MRI contrast to nephrogenic systemic fibrosis”
Paediatric incentives and obligations for SCNN1A's drugs
For amiloride, triamterene, cofirasersen, idrevloride: 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 · SCNN1A 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 · EMEA-002935-PIP01-20-M03 (Idrevloride, Treatment of primary ciliary dyskinesia): PM: decision on the application for modification of an agreed PIP, 15 May 2023Rule: An EMA PIP decision (agreement, modification or waiver) lists the drug as its active substance, exact name. Source: EMA EMEA-002935-PIP01-20-M03, 2026-11-11
- EU orphan designation: no · No EU orphan designation names amiloride, triamterene, cofirasersen, idrevlorideRule: 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: no · EMA lists no centrally authorised medicine of amiloride, triamterene, cofirasersen, idrevlorideRule: 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 amiloride, triamterene, cofirasersen, idrevlorideRule: 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 · 5 interventional studies of amiloride, triamterene, cofirasersen, idrevloride include patients under 18; 0 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 5 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.