VDR vitamin D receptor
ENSG00000111424 · protein coding · Open Targets profile
Nuclear receptor for calcitriol, the active form of vitamin D3 which mediates the action of this vitamin on cells (PubMed:10678179, PubMed:15728261, PubMed:16913708, PubMed:28698609, PubMed:37478846). Enters the nucleus upon vitamin D3 binding where it forms heterodimers with the retinoid X receptor/RXR (PubMed:28698609). The VDR-RXR heterodimers bind to specific response elements on DNA and activate the transcription of vitamin D3-responsive target genes (PubMed:28698609). Plays a central role in calcium homeostasis (By similarity). Also functions as a receptor for the secondary bile acid lithocholic acid (LCA) and its metabolites (PubMed:12016314, PubMed:32354638). {ECO:0000250|UniProtKB:P13053, ECO:0000269|PubMed:10678179, ECO:0000269|PubMed:12016314, ECO:0000269|PubMed:15728261, ECO:0000269|PubMed:16913708, ECO:0000269|PubMed:28698609, ECO:0000269|PubMed:32354638, ECO:0000269|PubMed:37478846}.
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 Withdrawn means the drug was pulled from the market or from development.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, High-Quality Pocket, Druggable Family
- PROTAC
- UniProt Ubiquitination, Database Ubiquitination, Small Molecule Binder
gnomAD loss of function constraint: o/e 0.47821998596191406 (upper bound 0.7017099857330322). 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 400 registry trials, updated in the last three years, of the most advanced drugs against VDR (dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitol), ranked by Phase 2+ trials. Live from ClinicalTrials.gov.
- Erkut BorazanciHonorHealth Research Institute · 3 of 3 trials Phase 2+
- Luke J PepponeUniversity of Rochester · 2 of 2 trials Phase 2+
- Kimmie NgDana-Farber Cancer Institute · 1 of 2 trials Phase 2+
- Mattias LorentzonSahlgrenska University Hospital, Västra Götalandsregionen · 1 of 2 trials Phase 2+
- Ademola A PopoolaDepartment of Surgery, University of Ilorin Teaching Hospital / University of Ilorin · 1 of 1 trials Phase 2+
- Adetunji T ToriolaWashington University School of Medicine · 1 of 1 trials Phase 2+
- Andrei Y HancharouThe Institute for Biophysics and Cell Engineering of the NAS of Belarus · 1 of 1 trials Phase 2+
- Andrew SeelyOttawa Hospital Research Institute · 1 of 1 trials Phase 2+
- Anne Marie Z JukicNational Institute of Environmental Health Sciences (NIEHS) · 1 of 1 trials Phase 2+
- Antonia Perez MartinCentre Hospitalier Universitaire de Nīmes · 1 of 1 trials Phase 2+
- Antonis GoulasSchool of Medicine, Aristotle University of Thessaloniki · 1 of 1 trials Phase 2+
- Aruna PradhanBrigham and Women's Hospital · 1 of 1 trials Phase 2+
- Athanasios D Anastasilakis424 General Military Hospital, Thessaloniki, Greece · 1 of 1 trials Phase 2+
- Ayman Al HendySheikh Shakbhout Medical City · 1 of 1 trials Phase 2+
- B. Catharine CravenToronto Rehabilitation Institute - UHN · 1 of 1 trials Phase 2+
Registry trials of dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitol 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; 363 of 378 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. 22 trials list no sites and are left out. Source: ClinicalTrials.gov, fetched live.
265 trials of drugs against VDR were stopped early; 23 of the 200 most recent give no reason
Terminated, withdrawn and suspended interventional trials of dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitol, 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 265.
- terminated · 2026-10-21 · Jantchou Prevost · matched by the registry's drug synonyms“Enrollment stopped at 55 of 316 planned participants due to recruitment challenges related to the COVID-19 pandemic and funding constraints. All enrolled participants completed 52-week follow-up.”
- terminated · 2026-10-01 · Inozyme Pharma · matched by the registry's drug synonyms“The study was terminated by the sponsor prior to planned completion based on a review of the data across indications and overall program considerations. Study termination was not related to participant safety.”
- terminated · 2026-08-26 · Diamyd Medical AB · matched by the registry's drug synonyms“Stopped for futility.”
- terminated · 2026-08-06 · University Hospital, Clermont-Ferrand · matched by the registry's drug synonymsNo reason given
- withdrawn · 2026-08-04 · Abramson Cancer Center at Penn Medicine · Vitamin D3 cholecalciferol)“Covid-19”
- terminated · 2026-07-02 · Sanofi · Doxercalciferol (GZ427397), Calcitriol“The study was conducted to fulfill a post-marketing commitment (PMC).FDA acknowledged the fulfillment of this PMC.”
- terminated · 2026-06-24 · University Hospital, Bordeaux · matched by the registry's drug synonyms“target number of patient not reached”
- terminated · 2026-06-15 · AbbVie · Paricalcitol“Company Decision”
- suspended · 2026-05-14 · Virginia Commonwealth University · Ergocalciferol Capsules“Insufficient staffing”
- terminated · 2026-05-08 · University of North Carolina, Chapel Hill · Vitamin D3 (cholecalciferol), Vitamin D3 (cholecalciferol) placebo“COVID-19 global pandemic, and study funding period ended 7/31/2020.”
- suspended · 2026-04-27 · Massachusetts General Hospital · matched by the registry's drug synonyms“Protocol is being modified.”
- terminated · 2026-04-23 · Melinda Telli · matched by the registry's drug synonyms“Slow accrual”
- withdrawn · 2026-04-17 · Eli Avisar, MD · matched by the registry's drug synonyms“Lack of Funding”
- terminated · 2026-04-13 · Dr. B. Catharine. Craven · matched by the registry's drug synonyms“COVID-19 Pandemic”
- terminated · 2026-04-03 · OPKO Health, Inc. · Calcifediol Oral Capsule“Cohort 1 completed and cohort 2 terminated prior to initiation per company decision”
- terminated · 2025-11-19 · Anhui Medical University · matched by the registry's drug synonyms“The study was halted at the midpoint because the interim analysis demonstrated a large effect size of the DHT group compared with the SCT group (Cohen's d = 1.58).”
- terminated · 2025-09-04 · Barichella Michela · matched by the registry's drug synonyms“COVID Pandemic and change in surgical activities in the institution”
- terminated · 2025-07-25 · OPKO Health, Inc. · matched by the registry's drug synonyms“FDA changed the post-marketing requirement necessitating a new study design and protocol.”
- terminated · 2025-06-11 · McGill University · matched by the registry's drug synonyms“Covid19 pandemic prevented continuation of study visits”
- terminated · 2025-05-07 · Massachusetts General Hospital · Topical Calcipotriene Ointment“Interim analysis did not show the required cytokine induction by topical calcipotriene to justify continuation of the trial”
- withdrawn · 2025-05-07 · Massachusetts General Hospital · matched by the registry's drug synonyms“The study was withdrawn due to lack of financial support.”
- withdrawn · 2025-05-07 · Massachusetts General Hospital · matched by the registry's drug synonyms“The study was withdrawn due to lack of financial support.”
- terminated · 2025-05-01 · Fondazione IRCCS Policlinico San Matteo di Pavia · matched by the registry's drug synonyms“Significant change in the profile of recruitable patients”
- terminated · 2025-03-21 · Dana-Farber Cancer Institute · Paricalcitol“Phase II was not pursued due to futility based on the results of the NAPOLI-3 therapeutic clinical trial.”
- terminated · 2025-03-14 · Murdoch Childrens Research Institute · matched by the registry's drug synonyms“Recruitment very slow \& ongoing funding not obtained.”
Paediatric incentives and obligations for VDR's drugs
For dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitol: 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 · VDR 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/0000241614 (calcium carbonate;cholecalciferol;risedronate sodium, Treatment of osteoporosis): W: decision granting a waiver in all age groups for all conditions or indications, 15 May 2025; EMEA-002093-PIP02-17-M02 (calcifediol, Treatment of secondary hyperparathyroidism (SHPT)): PM: decision on the application for modification of an agreed PIP, 14 Jun 2024; EMEA-001670-PIP01-14 (cholecalciferol;calcium carbonate, Combination treatment of osteoporosis): W: decision granting a waiver in all age groups for all conditions or indications, 22 Dec 2014; EMEA-001690-PIP01-14 (calcium carbonate;cholecalciferol, Prevention of skeletal related events in patients with advanced malignancies involving bone): W: decision granting a waiver in all age groups for all conditions or indications, 19 Dec 2014Rule: 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: yes · EU/3/01/052 withdrawn 31 Jul 2001: Treatment of hepatocellular carcinomaRule: 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 EU/3/01/052, 2026-11-11
- Authorised in the EU: no · EMA lists no centrally authorised medicine of dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitolRule: 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 dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitolRule: 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 · 596 interventional studies of dihydrotachysterol, paricalcitol, eldecalcitol, ergocalciferol, calcipotriene, calcitriol, calcifediol, cholecalciferol, alfacalcidol, doxercalciferol, seocalcitol, pefcalcitol include patients under 18; 65 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 18 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.