Von Willebrand DiseaseNCT01602419
Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
An observational study of Von Willebrand Disease, run by Octapharma at 24 sites in 10 countries.
Trial summary
This is an observational study, hence there is no study hypothesis Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Von Willebrand Disease
- How many people
- 120 participants
- Where
- 24 sitesin 10 countries
- Study dates
- Oct 2012to Apr 2018
- Phase
- Observational
- 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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: VWD patients of any gender, age, or VWD type
Who may be able to join
- Patients with a diagnosis of von Willebrand Disease who have been prescribed Wilate
Who cannot join
None listed.
Trial locations and contacts
24 sites in 10 countries.
24 sites in 10 countries
Canada9 sites
University of Alberta
Edmonton, Alberta
McMaster University
Hamilton, Ontario
Queens University
Kingston, Ontario, K7L 3N6
Maisonneuve-Rosemont Hospital
Montreal, Quebec, H1T 2M4
Children's Hospital of Eastern Ontario
Ottawa, Ontario
St. John Regional Hospital
Saint John, New Brunswick
Eastern Regional Health Authority
St. John's, Newfoundland and Labrador
St. Michael's Hospital
Toronto, Ontario
Vancouver General Hospital
Vancouver, British Columbia
United States4 sites
Nicklaus Children's Hospital
Miami, Florida, 33155
Virginia Commonwealth University
Richmond, Virginia, 23219
University of Utah
Salt Lake City, Utah, 84112
Los Angeles Biomedical Research Institute
Torrance, California, 90502
Spain3 sites
Hospital Universitario de Burgos
Burgos, 09006
Hospital San Pedro de Alcantara
Cáceres, 10003
Hospital Fundacion Jiminez Diaz
Madrid, 28040
Uruguay2 sites
Hospital Pereira Rossell
Montevideo
Sanatorio Americano
Montevideo
About this study
This is an observational study, hence there is no study hypothesis
- Conditions
- Von Willebrand Disease
- Being tested
- Patients using Wilate as standard of care
- 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.
Patients using Wilate as standard of careOther
Patients with von Willebrand Disease using Wilate for a period of 2 years.
| Study group | Role | What they receive |
|---|---|---|
| Patients using Wilate as standard of care treatment | This patient population is being treated with Wilate as standard of care treatment Receives: Other: Patients using Wilate as standard of care |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of Adverse Drug Reactions (ADRs) (%)
Medical Dictionary for Regulatory Activities (MedDRA) primary system organ class preferred term. Incidence rate = number of patients reporting the event / number of patients * 100
Measured: Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185])
Tolerability Assessment of Wilate Infusions by Reason for Administration
Tolerability was assessed using a 3-point verbal rating scale (excellent; satisfactory; unsatisfactory) according to overall feeling during and after Wilate therapy and occurrence of ADRs.
Tolerability was assessed for infusions given for on-demand and prophylactic treatment, but not for infusions administered for surgeries or for the purpose of thrombogenicity assessment. In some instances, however, investigators also recorded the tolerability of infusions given for surgical prophylaxis. For infusions administered for surgeries, only those with available tolerability assessments are presented. Wilate infusion may have been administered to a patient for more than one reason and may be included in more than one category (e.g., if a patient was under Wilate prophylaxis, they could also receive Wilate for the treatment of a bleeding episode [BE] or surgery or menstruation).
Measured: During and immediately after each infusion of Wilate during the study.
Secondary outcomes (4)
Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs)
Document the efficacy of Wilate in the treatment of acute BEs, breakthrough BEs in patients receiving prophylactic treatment, and menstrual BEs.
Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis.
Measured: During and immediately after treatment of each BE.
Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis
Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to the number of breakthrough bleeds per month. The treatment regimen for prophylactic treatment was at the discretion of the investigator and differed for each patient, as did the duration of prophylactic treatment. The prophylaxis efficacy population (EFF-P) was subdivided into 2 groups, prophylaxis on a continuous basis (EFF-PC population) and prophylaxis on an intermittent basis (EFF-PI population). In total, 25 patients received Wilate for prophylaxis and of these, 17 patients received prophylaxis on a continuous basis, which was defined as: (1) patients having received continuous prophylaxis over a period of at least 3 months, with no treatment gaps longer than 14 days; or (2) patients having received continuous prophylaxis for at least 1 year with an average of 1 infusion/per week (these patients may have had gaps of more than 14 days).
Measured: At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
Efficacy Analysis of Surgical Prophylaxis
Efficacy was rated on a 4-point scale (excellent; good; moderate; none) according to overall haemostasis during and after surgery.
Measured: During and immediately after each surgery.
Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period
Patient and investigator assessment of the overall efficacy of Wilate performed at the end of the study for each patient. Efficacy was rated on a 4-point scale (excellent; good; moderate; none); criteria for assessment were not defined.
Measured: At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]).
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 May 2012 (estimated)
- Study startOct 2012
- Primary completionApr 2018
- Study completionApr 2018
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.
- Other trials for Von Willebrand DiseaseStudies 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
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 120 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01602419
- Sponsor's protocol ID
- Wil-20
- Status last verified
- Dec 2020
- First submitted
- 15 May 2012
- Last updated
- 19 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Surveillance of Safety and Efficacy of Wilate in Patients With Von Willebrand Disease
- ClinicalTrials.gov ID
- NCT01602419
- Acronym / sponsor ID
- Wil-20
- Conditions
- Von Willebrand Disease
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 120 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- Oct 2012 (actual)
- Primary completion
- Apr 2018 (actual)
- Study completion
- Apr 2018 (actual)
- First posted
- 21 May 2012 (estimated)
- Results
- Posted 10 Dec 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is an observational study, hence there is no study hypothesis
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients using Wilate as standard of care treatment | – | Other: Patients using Wilate as standard of careThis patient population is being treated with Wilate as standard of care treatment |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- VWD patients of any gender, age, or VWD type
Key inclusion criteria (1 listed)
- Patients with a diagnosis of von Willebrand Disease who have been prescribed Wilate
Key exclusion criteria (0 listed)
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 |
|---|---|---|
| Patients using Wilate as standard of carePatients with von Willebrand Disease using Wilate for a period of 2 years. | Other | Patients using Wilate as standard of care treatment |
Primary outcome
Incidence of Adverse Drug Reactions (ADRs) (%)
Time frame: Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185])
Medical Dictionary for Regulatory Activities (MedDRA) primary system organ class preferred term. Incidence rate = number of patients reporting the event / number of patients * 100
Tolerability Assessment of Wilate Infusions by Reason for Administration
Time frame: During and immediately after each infusion of Wilate during the study.
Tolerability was assessed using a 3-point verbal rating scale (excellent; satisfactory; unsatisfactory) according to overall feeling during and after Wilate therapy and occurrence of ADRs. Tolerability was assessed for infusions given for on-demand and prophylactic treatment, but not for infusions administered for surgeries or for the purpose of thrombogenicity assessment. In some instances, however, investigators also recorded the tolerability of infusions given for surgical prophylaxis. For infusions administered for surgeries, only those with available tolerability assessments are presented. Wilate infusion may have been administered to a patient for more than one reason and may be included in more than one category (e.g., if a patient was under Wilate prophylaxis, they could also receive Wilate for the treatment of a bleeding episode [BE] or surgery or menstruation).
4 secondary outcomes
Patient and Investigator Efficacy Analysis Assessment of the Treatment of Bleeding Episodes (BEs)
Time frame: During and immediately after treatment of each BE.
Efficacy Analysis for the Prevention of Breakthrough Bleeds During Prophylaxis
Time frame: At the end of the study for each patient (study duration: mean [±SD]: 805 days [±247]; median [range]: 797 days [144-1185]).
Efficacy Analysis of Surgical Prophylaxis
Time frame: During and immediately after each surgery.
Overall Efficacy Assessment by Patient and Physician at the End of the Treatment Period
Time frame: At the end of the study for each patient (study duration: mean [±SD]: 596 days [±336]; median [range]: 732 days [2-1185]).
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Von Willebrand Disease
Active, not recruitingObservational study
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RecruitingObservational study
Heavy Menstrual Bleeding Progestin Treatment in Bleeding Disorders Study
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HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
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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.
The record lists no contact. Use the official record to reach the sponsor.
24 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease, run by Octapharma at 24 sites in 10 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 1 inclusion and 0 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 19 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 24 sites in Canada, United States, Spain, Uruguay, Argentina and 5 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 10 Dec 2019). 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?
Incidence of Adverse Drug Reactions (ADRs) (%) (time frame: Throughout the duration of each patient's participation in the study (mean [± standard deviation (SD)]: 575 days [±326]; median [range]: 731 days [2-1185])). 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?
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 19 Jan 2021.