Von Willebrand DiseasesPhase 3NCT04052698
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
A Phase 3 study testing Wilate in people with Von Willebrand Diseases, run by Octapharma at 14 sites in 8 countries.
Trial summary
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis. Read more about this study
- Who is it for?
- Ages 6 years and older
- Conditions the study is for
- Von Willebrand Diseases
- What is being tested?
- Wilate
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 43 participants
- Where
- 14 sitesin 8 countries
- Study dates
- 18 Jun 2020to 23 Apr 2022
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patients who meet all of the following criteria are eligible for the study:
- Aged ≥6 years at the time of screening
- VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (RCo)] <30 IU/dL, 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding
- Currently receiving on-demand treatment with a VWF-containing product with at least 1, and an average of ≥2, documented spontaneous BEs per month in the last 6 months, with at least 2 of these BEs requiring treatment with a VWF-containing product
- Availability of records to reliably evaluate type, frequency, and treatment of BEs for at least 6 months of on-demand treatment before screening
- Female patients of child-bearing potential must have a negative urine pregnancy test at screening and agree to use adequate birth control measures; in case hormonal contra-ception is used, the medication class should remain unchanged for the duration of the study
- All patients to provide voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted
Who cannot join
- Patients who meet any of the following criteria are not eligible for the study:
- Having received on-demand or prophylactic treatment with a VWF-containing product but having no records available to reliably evaluate the type, frequency, and treatment of BEs over a period of at least 6 months of on-demand treatment
- History, or current suspicion, of VWF or FVIII inhibitors
- Medical history of a thromboembolic event within 1 year before enrolment
- Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)
- Platelet count <100,000/µL at screening (except for VWD type 2B)
Show all 13Show fewer
- Body weight <20 kg at screening
- Patients receiving, or scheduled to receive, immunosuppressant drugs (other than an-tiretroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs
- Pregnant or breast-feeding at the time of enrolment
- Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia)
- Treatment with any IMP in another interventional clinical study currently or within 4 weeks before enrolment
- Other coagulation disorders or bleeding disorders due to anatomical reasons
- Known hypersensitivity to any of the components of the study drug
Trial locations and contacts
14 sites in 8 countries.
14 sites in 8 countries
Lebanon3 sites
Hotel Dieu de France Hospital
Beirut, BP166830
American University of Beirut Medical Center
Beirut
Nini Hospital
Tripoli
Bulgaria2 sites
"Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
Sofia
Pediatric Clinic of Haematology and Oncology
Varna, 9010
Hungary2 sites
Medical Centre Hungarian Defence Forces
Budapest, 1134
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
Debrecen, 4032
Russia2 sites
Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal
Kirov, 610027
Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis
Moscow, 119049
Ukraine2 sites
State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology
Kyiv, 01135
Communal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center"of Lviv Regional Council
Lviv, 79035
United States1 site
Children's Healthcare of Atlanta
Atlanta, Georgia, 30329
Belarus1 site
Republican Research Center for Radiation Medicine and Human Ecology
Homyel, 290
About this study
Official title: Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.
- Conditions
- Von Willebrand Diseases
- Being tested
- Wilate
- Medical subject headings
- von Willebrand Diseases
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
WilateDrug
Also called von Willebrand factor / Factor VIII (plasma derived)
Produced from the plasma of human donors, Wilate is presented as a powder or solvent for intravenous injection containing normally 500 IU or 1000 IU human VWF and human FVIII per vial. The ratio between VWF ristocetin co-factor activity (VWF:RCo) and FVIII:C is 1:1. The product contains approximately 100 IU/ml human VWF when reconstituted with 5ml/10mL water for injection with 0.1% polysorbate 80.
The specific activity of Wilate is ≥67 IU VWF:RCo/mg protein. The injection or infusion rate should not exceed 2-3mL per minute.
| Study group | Role | What they receive |
|---|---|---|
| All patients | Experimental | Patients with type 3, type 2 (except 2N), or severe type 1 VWD aged ≥6 years at screening receiving Wilate for prophylactic treatment. Receives: Drug: Wilate |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Annualized Bleeding Rate (TABR)
The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds (except menstrual bleeds) occurring in the time period between first dose of the investigational medicinal product (IMP) and the Study Completion Visit, divided by the duration (in years) between first dose of IMP and the Study Completion Visit.
Measured: 12 months
Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29
Estimated TABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
Measured: 12 Months
Secondary outcomes (7)
Spontaneous Annualized Bleeding Rate (SABR)
Spontaneous annualized bleeding rate (SABR) calculated in analogy with TABR
Measured: 12 months
Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29.
Estimated SABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
Measured: 12 Months
Wilate Consumption for Prophylaxis (mFAS Population)
Data on the consumption of Wilate (VWF/FVIII IU/kg per month and per week per patient) for prophylactic treatment
Measured: 12 months
Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo)
Incremental VWF:RCo IVR of Wilate over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment)
Measured: From baseline and 12-month visit
Incremental In Vivo Recovery (IVR) of FVIII
FVIII:C of Wilate in pediatric patients (at baseline PK visit) measured by chromogenic assay
Measured: Baseline and 12-month visit
Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
Treatment efficacy will be assessed at the end of a BE by the patient using predefined criteria of 'Excellent', 'Good', 'Moderate' or 'None'. All effectiveness ratings assessed as either "excellent" or "good" will be considered "successfully treated".
Measured: 12 months
Wilate Exposure for Prophylaxis (mFAS Population)
Data on the exposure days of Wilate prophylactic treatment
Measured: 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Aug 2019
- Study start18 Jun 2020
- Primary completion23 Apr 2022
- Study completion23 Apr 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (16)Papers the sponsor has linked to this study.
- Other trials for Von Willebrand DiseasesStudies of the same condition, open and under way.
- More trials run by OctapharmaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 43 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cristina Solomon, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04052698
- Sponsor's protocol ID
- WIL-31
- Status last verified
- Sep 2023
- First submitted
- 9 Aug 2019
- Last updated
- 25 Oct 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Bodo I, Eikenboom J, Montgomery R, Patzke J, Schneppenheim R, Di Paola J; von Willebrand factor Subcommittee of the Standardization and Scientific Committee of the International Society for Thrombosis and Haemostasis. Platelet-dependent von Willebrand factor activity. Nomenclature and methodology: communication from the SSC of the ISTH. J Thromb Haemost. 2015 Jul;13(7):1345-50. doi: 10.1111/jth.12964. Epub 2015 May 9. No abstract available.
PubMed 25858564Sadler JE. A revised classification of von Willebrand disease. For the Subcommittee on von Willebrand Factor of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 1994 Apr;71(4):520-5.
PubMed 8052974Rodeghiero F, Castaman G, Tosetto A. How I treat von Willebrand disease. Blood. 2009 Aug 6;114(6):1158-65. doi: 10.1182/blood-2009-01-153296. Epub 2009 May 27.
PubMed 19474451Castaman G, Goodeve A, Eikenboom J; European Group on von Willebrand Disease. Principles of care for the diagnosis and treatment of von Willebrand disease. Haematologica. 2013 May;98(5):667-74. doi: 10.3324/haematol.2012.077263.
PubMed 23633542U. S. Department of Health and Human Services. Health Measures. Available at http://www.healthmeasures.net/explore-measurement-systems/promis.
Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, Ader D, Fries JF, Bruce B, Rose M; PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care. 2007 May;45(5 Suppl 1):S3-S11. doi: 10.1097/01.mlr.0000258615.42478.55.
PubMed 17443116Hays RD, Spritzer KL, Schalet BD, Cella D. PROMIS(R)-29 v2.0 profile physical and mental health summary scores. Qual Life Res. 2018 Jul;27(7):1885-1891. doi: 10.1007/s11136-018-1842-3. Epub 2018 Mar 22.
PubMed 29569016Maruish M. User's manual for the SF-36v2 Health Survey (3rd edition). Optum Incorporated; 2011.
Saris-Baglama, R,DeRosa, M, Raczek, A, Bjorner, J,Turner-Bowker, D, Ware, J. The SF-10™ Health Survey for Children: A User's Guide. QualityMetric Incorporated; 2007.
Feldman BM, Funk SM, Bergstrom BM, Zourikian N, Hilliard P, van der Net J, Engelbert R, Petrini P, van den Berg HM, Manco-Johnson MJ, Rivard GE, Abad A, Blanchette VS. Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: validity of the hemophilia joint health score. Arthritis Care Res (Hoboken). 2011 Feb;63(2):223-30. doi: 10.1002/acr.20353.
PubMed 20862683van Galen KPM, Timmer MA, de Kleijn P, Fischer K, Foppen W, Schutgens REG, Eikenboom J, Meijer K, Cnossen MH, Fijnvandraat K, van der Bom JG, Laros-van Gorkom BAP, Leebeek FWG, Mauser-Bunschoten EP, Win Study Group OBOT. Joint assessment in von Willebrand disease. Validation of the Haemophilia Joint Health score and Haemophilia Activities List. Thromb Haemost. 2017 Aug 1;117(8):1465-1470. doi: 10.1160/TH16-12-0967. Epub 2017 May 11.
PubMed 28492695Janssen CA, Scholten PC, Heintz AP. A simple visual assessment technique to discriminate between menorrhagia and normal menstrual blood loss. Obstet Gynecol. 1995 Jun;85(6):977-82. doi: 10.1016/0029-7844(95)00062-V.
PubMed 7770270Higham JM, O'Brien PM, Shaw RW. Assessment of menstrual blood loss using a pictorial chart. Br J Obstet Gynaecol. 1990 Aug;97(8):734-9. doi: 10.1111/j.1471-0528.1990.tb16249.x.
PubMed 2400752Djambas Khayat C, Dubey L, Inati A, Lissitchkov T, Novik D, Peteva E, Sidonio RF Jr, Taher AT, Vilchevska KV, Vdovin V, Boban A. Efficacy and Safety of Prophylaxis With a Plasma-Derived von Willebrand Factor/Factor VIII Concentrate (Wilate) in Patients With Type 3 von Willebrand Disease-A WIL-31 Study Sub-Analysis. Eur J Haematol. 2026 May;116(5):674-681. doi: 10.1111/ejh.70117. Epub 2026 Jan 30.
PubMed 41614378Sidonio RF Jr, Boban A, Lissitchkov T, Nemes L, Vdovin V, Khayat CD. Individualizing dosing frequency may improve the efficacy of prophylaxis in patients with von Willebrand disease-a WIL-31 subanalysis. Res Pract Thromb Haemost. 2025 Oct 10;9(7):103221. doi: 10.1016/j.rpth.2025.103221. eCollection 2025 Oct.
PubMed 41278463
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)
- ClinicalTrials.gov ID
- NCT04052698
- Acronym / sponsor ID
- WIL-31
- Conditions
- Von Willebrand Diseases
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 43 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 18 Jun 2020 (actual)
- Primary completion
- 23 Apr 2022 (actual)
- Study completion
- 23 Apr 2022 (actual)
- First posted
- 12 Aug 2019 (actual)
- Results
- Posted 25 Oct 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| All patients | Experimental | Drug: WilatePatients with type 3, type 2 (except 2N), or severe type 1 VWD aged ≥6 years at screening receiving Wilate for prophylactic treatment. |
Who may qualify
- Ages
- 6 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (7 listed)
- Aged ≥6 years at the time of screening
- VWD type 1 (baseline von Willebrand factor activity [VWF:Ristocetin Co-factor (RCo)] <30 IU/dL, 2A, 2B, 2M, or 3 according to medical history requiring substitution therapy with a VWF-containing product to control bleeding
- Currently receiving on-demand treatment with a VWF-containing product with at least 1, and an average of ≥2, documented spontaneous BEs per month in the last 6 months, with at least 2 of these BEs requiring treatment with a VWF-containing product
- Availability of records to reliably evaluate type, frequency, and treatment of BEs for at least 6 months of on-demand treatment before screening
- Female patients of child-bearing potential must have a negative urine pregnancy test at screening and agree to use adequate birth control measures; in case hormonal contra-ception is used, the medication class should remain unchanged for the duration of the study
Key exclusion criteria (13 listed)
- Having received on-demand or prophylactic treatment with a VWF-containing product but having no records available to reliably evaluate the type, frequency, and treatment of BEs over a period of at least 6 months of on-demand treatment
- History, or current suspicion, of VWF or FVIII inhibitors
- Medical history of a thromboembolic event within 1 year before enrolment
- Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate trans-aminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L)
- Platelet count <100,000/µL at screening (except for VWD type 2B)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| WilateAlso: von Willebrand factor / Factor VIII (plasma derived)Produced from the plasma of human donors, Wilate is presented as a powder or solvent for intravenous injection containing normally 500 IU or 1000 IU human VWF and human FVIII per vial. The ratio between VWF ristocetin co-factor activity (VWF:RCo) and FVIII:C is 1:1. The product contains approximately 100 IU/ml human VWF when reconstituted with 5ml/10mL water for injection with 0.1% polysorbate 80. The specific activity of Wilate is ≥67 IU VWF:RCo/mg protein. The injection or infusion rate should not exceed 2-3mL per minute. | Drug | All patients |
Primary outcome
Total Annualized Bleeding Rate (TABR)
Time frame: 12 months
The TABR was calculated as the total number of spontaneous bleeds, traumatic bleeds, and other bleeds (except menstrual bleeds) occurring in the time period between first dose of the investigational medicinal product (IMP) and the Study Completion Visit, divided by the duration (in years) between first dose of IMP and the Study Completion Visit.
Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29
Time frame: 12 Months
Estimated TABR number calculated using a negative binomial counting regression model. Comparison between this number calculated for studies WIL-29 (NCT04053699) and WIL-31. For the comparison of results from WIL-31 to WIL-29 an estimated total annualized bleeding rate was calculated for each cohort, and compared with a negative binomial counting model. As these were estimated rates, there is only one value for each cohort with no measure of spread
7 secondary outcomes
Spontaneous Annualized Bleeding Rate (SABR)
Time frame: 12 months
Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29.
Time frame: 12 Months
Wilate Consumption for Prophylaxis (mFAS Population)
Time frame: 12 months
Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo)
Time frame: From baseline and 12-month visit
Incremental In Vivo Recovery (IVR) of FVIII
Time frame: Baseline and 12-month visit
Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
Time frame: 12 months
Wilate Exposure for Prophylaxis (mFAS Population)
Time frame: 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Oct 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 25858564Bodo I, Eikenboom J, Montgomery R, Patzke J, Schneppenheim R, Di Paola J; von Willebrand factor Subcommittee of the Standardization and Scientific Committee of the International Society for Thrombosis and Haemostasis. Platelet-dependent von Willebrand factor activity. Nomenclature and methodology: communication from the SSC of the ISTH. J Thromb Haemost. 2015 Jul;13(7):1345-50. doi: 10.1111/jth.12964. Epub 2015 May 9. No abstract available.
- Background: PubMed 8052974Sadler JE. A revised classification of von Willebrand disease. For the Subcommittee on von Willebrand Factor of the Scientific and Standardization Committee of the International Society on Thrombosis and Haemostasis. Thromb Haemost. 1994 Apr;71(4):520-5.
- Background: PubMed 19474451Rodeghiero F, Castaman G, Tosetto A. How I treat von Willebrand disease. Blood. 2009 Aug 6;114(6):1158-65. doi: 10.1182/blood-2009-01-153296. Epub 2009 May 27.
- Background: PubMed 23633542Castaman G, Goodeve A, Eikenboom J; European Group on von Willebrand Disease. Principles of care for the diagnosis and treatment of von Willebrand disease. Haematologica. 2013 May;98(5):667-74. doi: 10.3324/haematol.2012.077263.
- BackgroundU. S. Department of Health and Human Services. Health Measures. Available at http://www.healthmeasures.net/explore-measurement-systems/promis.
- Background: PubMed 17443116Cella D, Yount S, Rothrock N, Gershon R, Cook K, Reeve B, Ader D, Fries JF, Bruce B, Rose M; PROMIS Cooperative Group. The Patient-Reported Outcomes Measurement Information System (PROMIS): progress of an NIH Roadmap cooperative group during its first two years. Med Care. 2007 May;45(5 Suppl 1):S3-S11. doi: 10.1097/01.mlr.0000258615.42478.55.
- Background: PubMed 29569016Hays RD, Spritzer KL, Schalet BD, Cella D. PROMIS(R)-29 v2.0 profile physical and mental health summary scores. Qual Life Res. 2018 Jul;27(7):1885-1891. doi: 10.1007/s11136-018-1842-3. Epub 2018 Mar 22.
- BackgroundMaruish M. User's manual for the SF-36v2 Health Survey (3rd edition). Optum Incorporated; 2011.
- BackgroundSaris-Baglama, R,DeRosa, M, Raczek, A, Bjorner, J,Turner-Bowker, D, Ware, J. The SF-10™ Health Survey for Children: A User's Guide. QualityMetric Incorporated; 2007.
- Background: PubMed 20862683Feldman BM, Funk SM, Bergstrom BM, Zourikian N, Hilliard P, van der Net J, Engelbert R, Petrini P, van den Berg HM, Manco-Johnson MJ, Rivard GE, Abad A, Blanchette VS. Validation of a new pediatric joint scoring system from the International Hemophilia Prophylaxis Study Group: validity of the hemophilia joint health score. Arthritis Care Res (Hoboken). 2011 Feb;63(2):223-30. doi: 10.1002/acr.20353.
- Background: PubMed 28492695van Galen KPM, Timmer MA, de Kleijn P, Fischer K, Foppen W, Schutgens REG, Eikenboom J, Meijer K, Cnossen MH, Fijnvandraat K, van der Bom JG, Laros-van Gorkom BAP, Leebeek FWG, Mauser-Bunschoten EP, Win Study Group OBOT. Joint assessment in von Willebrand disease. Validation of the Haemophilia Joint Health score and Haemophilia Activities List. Thromb Haemost. 2017 Aug 1;117(8):1465-1470. doi: 10.1160/TH16-12-0967. Epub 2017 May 11.
- Background: PubMed 7770270Janssen CA, Scholten PC, Heintz AP. A simple visual assessment technique to discriminate between menorrhagia and normal menstrual blood loss. Obstet Gynecol. 1995 Jun;85(6):977-82. doi: 10.1016/0029-7844(95)00062-V.
- U.S Department of Health and Human Services, Health MeasuresLink provided by the sponsor.
- Individual participant dataSharing planned: Undecided
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Cristina Solomon study director
Octapharma
14 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Wilate in people with Von Willebrand Diseases, run by Octapharma at 14 sites in 8 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 13 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in Lebanon, Bulgaria, Hungary, Russia, Ukraine and 3 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Oct 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Total Annualized Bleeding Rate (TABR) (time frame: 12 months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04052698, which also lists 16 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Oct 2023.