ATP1A1 ATPase Na+/K+ transporting subunit alpha 1
ENSG00000163399 · protein coding · Open Targets profile
Catalytic subunit of the Na(+)/K(+)-ATPase pump that hydrolyzes ATP to exchange ions across the plasma membrane, exporting 3 Na(+) and importing 2 K(+) per cycle against electrochemical gradients (PubMed:29499166, PubMed:30388404, PubMed:35803952). It undergoes ATP- driven conformational changes that allow alternating binding and release of Na(+) and K(+) ions across the membrane (PubMed:35803952). This process maintains essential Na(+) and K(+) gradients for membrane potential and cellular function. Could also be part of an osmosensory signaling pathway that senses body-fluid sodium levels and controls salt intake behavior as well as voluntary water intake to regulate sodium homeostasis (By similarity). {ECO:0000250|UniProtKB:Q8VDN2, ECO:0000269|PubMed:29499166, ECO:0000269|PubMed:30388404, ECO:0000269|PubMed:35803952}.
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 Withdrawn means the drug was pulled from the market or from development.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
- Database Ubiquitination, Half-life Data, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.12197999656200409 (upper bound 0.19068999588489532). 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 127 registry trials, updated in the last three years, of the most advanced drugs against ATP1A1 (digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Andreas DiaconTASK Applied Science Clinical Research Centre · 1 of 1 trials Phase 2+
- Bill PayneWest Virginia University - Charleston Division/CAMC · 1 of 1 trials Phase 2+
- Brian H CuthbertsonSunnybrook Health Sciences Centre · 1 of 1 trials Phase 2+
- Bubu A BaniniYale University · 1 of 1 trials Phase 2+
- Bubu BaniniYale University · 1 of 1 trials Phase 2+
- Christoph HornikDuke Clinical Research Institute · 1 of 1 trials Phase 2+
- Dirk J van VeldhuisenUniversity Medical Center Groningen · 1 of 1 trials Phase 2+
- Gabe CoitlerSeismic Pharmaceuticals · 1 of 1 trials Phase 2+
- Ghada ShamsBenha University · 1 of 1 trials Phase 2+
- Guillermo Ernesto Liniado– · 1 of 1 trials Phase 2+
- Jean GremUniversity of Nebraska · 1 of 1 trials Phase 2+
- Jianqing LinThomas Jefferson University · 1 of 1 trials Phase 2+
- Jonathan MettsMoffitt Cancer Center · 1 of 1 trials Phase 2+
- Jonathan P PicciniDuke Clinical Research Organization · 1 of 1 trials Phase 2+
- Laura MetrockUniversity of Alabama at Birmingham Children's of Alabama · 1 of 1 trials Phase 2+
Registry trials of digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime 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; 115 of 124 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. 3 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
20 trials of drugs against ATP1A1 were stopped early; 2 give no reason
Terminated, withdrawn and suspended interventional trials of digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime, 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 20 most recent of 20.
- terminated · 2026-10-21 · University of Nebraska · Digoxin“Due to low accrual and futility”
- terminated · 2026-09-29 · Yale University · Intravenous digoxin“Slow accrual”
- terminated · 2026-09-10 · CAMC Health System · matched by the registry's drug synonyms“Low enrollment and PI retired.”
- terminated · 2026-05-22 · AMAG Pharmaceuticals, Inc. · AMAG-423 (digoxin immune fab)“Following a recommendation from the Data and Safety Monitoring Board (DSMB), the study was stopped early for futility. There were no safety concerns raised.”
- withdrawn · 2025-04-22 · ViiV Healthcare · matched by the registry's drug synonyms“Sponsor decision”
- withdrawn · 2025-04-01 · Turning Point Therapeutics, Inc. · Digoxin“Insufficient enrollment”
- terminated · 2025-02-20 · Damascus University · Digoxin+furosemide+captopril“lack of necessary equipment”
- withdrawn · 2024-09-20 · University of Illinois Chicago · Digoxin“Study Not Funded”
- withdrawn · 2023-02-22 · The Affiliated Nanjing Drum Tower Hospital of Nanjing University Medical School · Digoxin tablet“Administrative decision to terminate.”
- withdrawn · 2022-12-01 · Scripps Health · matched by the registry's drug synonyms“no participants enrolled”
- terminated · 2021-03-16 · Fox Chase Cancer Center · Digoxin“Slow accrual”
- terminated · 2019-03-26 · Rennes University Hospital · matched by the registry's drug synonymsNo reason given
- terminated · 2019-03-20 · Maruho Co., Ltd. · matched by the registry's drug synonyms“Lack of effect in interim analysis”
- terminated · 2019-02-15 · Aultman Health Foundation · matched by the registry's drug synonyms“Limited resources available to met accrual goal.”
- withdrawn · 2018-05-15 · University of Texas Southwestern Medical Center · Digoxin Combination“No subjects consented for enrollment on this study.”
- terminated · 2018-03-29 · University of Louisville · Erlotinib plus Digoxin“Interim analysis revealed that only 1 patient had a partial response.”
- terminated · 2018-02-22 · Western Regional Medical Center · Digoxin“PI Leaving Site”
- withdrawn · 2015-03-10 · Boehringer Ingelheim · DigoxinNo reason given
- withdrawn · 2014-10-23 · Debiopharm International SA · Istaroxime“The study was not started due to a re-evaluation of the istaroxime development program”
- withdrawn · 2014-10-23 · Debiopharm International SA · Istaroxime“The study was not started due to a re-evaluation of the istaroxime development program”
Paediatric incentives and obligations for ATP1A1's drugs
For digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime: 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 · ATP1A1 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: no · No EMA paediatric investigation plan decision names digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroximeRule: 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 digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroximeRule: 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 digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroximeRule: 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 digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroximeRule: FDA's Pediatric Oncology Drug Approvals table names the drug. Source: FDA Pediatric Oncology Drug Approvals (content current as of 2026-03-03), 2026-11-11
- Registered studies open to children: yes · 9 interventional studies of digitoxin, lanatoside c, deslanoside, acetyldigitoxin, digoxin, istaroxime include patients under 18; 1 is 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, 2026-11-11
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 6 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.
| Drug | Type | Highest stage | Indications | Source records |
|---|---|---|---|---|
| DIGITOXIN | Small molecule | approval | cardiovascular disorder, cardiac arrhythmia, heart failure, cystic fibrosis | Open Targets · ChEMBL |
| LANATOSIDE C | Small molecule | approval | cardiovascular disorder | Open Targets · ChEMBL |
| DESLANOSIDE | Small molecule | approval | cardiovascular disorder, cardiac arrhythmia, heart failure | Open Targets · ChEMBL |
| ACETYLDIGITOXIN | Small molecule | approval | cardiovascular disorder, congestive heart failure | Open Targets · ChEMBL |
| DIGOXIN | Small molecule | phase 4 | heart failure, atrial fibrillation, congestive heart failure, cardiovascular disorder, cardiac arrhythmia, Supraventricular tachycardia, atrial flutter, preeclampsia, +23 more | Open Targets · ChEMBL |
| ISTAROXIME | Small molecule | phase 2 3 | heart disorder, heart failure, Shock | Open Targets · ChEMBL |