Disease · Orphanet_903
Von Willebrand disease
6 sourcesdiseases-2026-09-18-0.1.0
Cite this record
ConductScience Library. Von Willebrand disease. Diseases, record Orphanet_903. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/Orphanet_903
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
Angiohemophilia, Angiohemophilias, Hemophilia, Vascular, Hereditary von Willebrand disease, VON WILLEBRAND DIS, VON WILLEBRANDS DIS, Vascular Hemophilia, Vascular Hemophilias, von Willebrand Disease, Recessive Form, von Willebrand Diseases, von Willebrand's Disease, von Willebrand's Diseases, von Willebrands Disease, von Willebrands Diseases
- Open Targets
- Highest stage of its drugs
- Approval, for 1 of its 16 drugs, from EMA Human Drugs, PMDA and TTD
- Open Targets
Drugs16
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 |
|---|---|---|---|---|---|---|---|---|
| VONICOG ALFA | CHEMBL2107873 | Protein | Approval | EMA Human Drugs, PMDA and TTD | 6 | 3 | 3 | Drugs and targets |
| VON WILLEBRAND FACTOR HUMAN | CHEMBL4298126 | Phase 3 | ClinicalTrials.gov | 10 | 10 | 8 | Drugs and targets | |
| TRANEXAMIC ACID | CHEMBL877 | Small molecule | Phase 3 | ClinicalTrials.gov | 4 | 4 | 4 | Drugs and targets |
| MOROCTOCOG ALFA | CHEMBL2109137 | Protein | Phase 3 | ClinicalTrials.gov | 3 | 3 | 0 | Drugs and targets |
| OCTOCOG ALFA | CHEMBL4297873 | Protein | Phase 3 | ClinicalTrials.gov | 3 | 3 | 0 | Drugs and targets |
| ANTIHEMOPHILIC FACTOR, PEGYLATED (MW 20000) HUMAN SEQUENCE RECOMBINANT | CHEMBL4594584 | Phase 3 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets | |
| LONOCTOCOG ALFA | CHEMBL4594603 | Protein | Phase 3 | ClinicalTrials.gov | 2 | 2 | 0 | Drugs and targets |
| EFMOROCTOCOG ALFA | CHEMBL4297945 | Protein | Phase 3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| TUROCTOCOG ALFA | CHEMBL2108286 | Protein | Phase 3 | ClinicalTrials.gov | 1 | 1 | 0 | Drugs and targets |
| RONDAPTIVON PEGOL | CHEMBL5314642 | Oligonucleotide | Phase 2 | ClinicalTrials.gov and TTD | 4 | 2 | 2 | Drugs and targets |
1 to 10 of 16
See all 16 drugsTargets6
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 |
|---|---|---|---|---|---|---|
| F8 | Coagulation factor VIII | 9 | Coagulation factor VIII exogenous protein | Phase 3 | https://conductscience.org/library/genes/2157 | Drugs and targets |
| VWF | von Willebrand factor | 3 | von Willebrand factor exogenous protein; von Willebrand factor inhibitor | Approval | https://conductscience.org/library/genes/7450 | Drugs and targets |
| F10 | Coagulation factor IX and X | 1 | Coagulation factor IX and X other | Phase 1 | https://conductscience.org/library/genes/2159 | Drugs and targets |
| F9 | Coagulation factor IX and X | 1 | Coagulation factor IX and X other | Phase 1 | https://conductscience.org/library/genes/2158 | Drugs and targets |
| IL11RA | Interleukin-11 receptor subunit alpha | 1 | Interleukin 11 receptor alpha agonist | Phase 2 | https://conductscience.org/library/genes/3590 | Drugs and targets |
| PLG | Plasminogen | 1 | Plasminogen inhibitor | Phase 3 | https://conductscience.org/library/genes/5340 | Drugs and targets |
Trials17
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. 3 more are not shown, since the checks do not confirm them.
| Trial | Title | Phase | Participants | Results first posted | Conditions | Drugs | Linked by | Source |
|---|---|---|---|---|---|---|---|---|
| NCT02460458 | Type 3 Von Willebrand International Registries Inhibitor Prospective Study | 265 | 2016-04-19 | Type 3 Von Willebrand's Disease | VON WILLEBRAND FACTOR HUMAN | Its record names the disease | ClinicalTrials.gov | |
| NCT04053699 | Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment | 56 | 2023-12-07 | Von Willebrand Diseases | VON WILLEBRAND FACTOR HUMAN | Its record names the disease | ClinicalTrials.gov | |
| NCT02606045 | Minimize Menorrhagia in Women With Von Willebrand Disease | Phase 3 | 39 | 2024-07-01 | Von Willebrand Diseases | TRANEXAMIC ACID, VON WILLEBRAND FACTOR HUMAN | Its record names the disease | ClinicalTrials.gov |
| NCT03879135 | A Study of Recombinant Von Willebrand Factor (rVWF) in Pediatric and Adult Participants With Severe Von Willebrand Disease (VWD) | Phase 3 | 38 | 2025-09-03 | Von Willebrand Disease (VWD) | VON WILLEBRAND FACTOR HUMAN, VONICOG ALFA | Its record names the disease | ClinicalTrials.gov |
| NCT04344860 | Prevent Postpartum Hemorrhage in Women With Von Willebrand Disease: The VWD-WOMAN Trial | Phase 3 | 20 | 2025-10-31 | Von Willebrand Diseases, Postpartum Hemorrhage | TRANEXAMIC ACID, VON WILLEBRAND FACTOR HUMAN | Its record names the disease | ClinicalTrials.gov |
| NCT04146376 | Von Willebrand Factor in Pregnancy (VIP) Study | 110 | Von Willebrand Diseases | TRANEXAMIC ACID | Its record names the disease | ClinicalTrials.gov | ||
| NCT05500807 | Emicizumab for Severe Von Willebrand Disease (VWD) and VWD/Hemophilia A | Phase 1 | 40 | Von Willebrand Disease, Type 3, Concomitant VWD and Hemophilia | EMICIZUMAB | Its record names the disease | ClinicalTrials.gov | |
| NCT00632242 | ARC1779 Injection in Patients With Von Willebrand Factor-Related Platelet Function Disorders | Phase 2 | 28 | Purpura, Thrombotic Thrombocytopenic, Von Willebrand Disease Type-2b | EGAPTIVON PEGOL | Its record names the disease | ClinicalTrials.gov | |
| NCT04677803 | BT200 in Hereditary Bleeding Disorders | Phase 2 | 26 | Von Willebrand Diseases, Hemophilia A | RONDAPTIVON PEGOL | Its record names the disease | ClinicalTrials.gov | |
| NCT07410130 | Clinical Spectrum and Management of Von Willebrand Disease Among Children in Assiut Governorate | 25 | Von Willebrand Disease (VWD), Inherited Bleeding Disorders in Children, Pediatric Hemostatic Disorders | TRANEXAMIC ACID | Its record names the disease | ClinicalTrials.gov |
1 to 10 of 17
See all 17 trialsComparisons in its trials4
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 |
|---|---|---|---|---|---|
| Tranexamic Acid vs Recombinant Von Willebrand Factor: deaths from any cause | 0 of 36 | 0 of 36 | 72 | NCT02606045 | Trial comparisons |
| Tranexamic Acid vs Recombinant Von Willebrand Factor: serious adverse events | 0 of 36 | 0 of 36 | 72 | NCT02606045 | Trial comparisons |
| rVWF plus TA vs rVWF alone: deaths from any cause | 0 of 10 | 0 of 10 | 20 | NCT04344860 | Trial comparisons |
| rVWF plus TA vs rVWF alone: serious adverse events | 0 of 10 | 0 of 10 | 20 | NCT04344860 | Trial comparisons |
Papers from its trials6
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/Orphanet_903
GET https://conductscience.org/api/diseases/v1/Orphanet_903/drugs