Von Willebrand DiseasesNCT04053699
Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
An observational study of Von Willebrand Diseases, run by Octapharma at 15 sites in 8 countries.
Trial summary
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. Read more about this study
- Who is it for?
- Ages 66 months and older
- Conditions the study is for
- Von Willebrand Diseases
- What is being tested?
- Von Willebrand Factor-Containing Product
- How many people
- 56 participants
- Where
- 15 sitesin 8 countries
- Study dates
- 25 Jun 2019to 31 Jan 2021
- 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 66 months and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Study population: Overall, 55 previously treated patients aged ≥5.5 years at the time of enrolment, with type 3, type 2 (except 2N), or severe type 1 VWD will be included into this study. Of these 55 patients, at least 6 should have type 3 VWD and at least 6 should be ≥5.5 to <16 years of age.
Who may be able to join
- Patients who meet all of the following criteria are eligible for the study:
- Male or female patients aged ≥5.5 years at the time of enrolment
- VWD type 1 (baseline von Willebrand factor activity [VWF: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 frequent on-demand treatment with a VWF-containing product
- In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation
- Voluntarily given, fully informed written and signed consent obtained before collection of any patient data
Who cannot join
- Patients who meet any of the following criteria are not eligible for the study:
- Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds
- Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months
- History, or current suspicion, of VWF or FVIII inhibitors
- Medical history of a thromboembolic event within 6 months before enrolment
- Severe liver or kidney diseases as described in the medical records
Show all 12Show fewer
- Female patients with an existing or suspected pregnancy or who are breast-feeding at the time of enrolment
- Change in hormonal contraception within 6 months before enrolment
- Cervical or uterine conditions causing abnormal uterine bleeding (including infection or dysplasia)
- Other coagulation disorders or bleeding disorders due to anatomical reasons
- Participation in an interventional clinical study during the 6-month of study period
- Inability to complete the patient diary to reliably evaluate the type, frequency, and treatment of BEs during the 6-month study period
Trial locations and contacts
15 sites in 8 countries.
15 sites in 8 countries
Hungary3 sites
Medical Centre Hungarian Defence Forces
Budapest, 1134
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
Debrecen, 4032
University Clinical Center, Department of Internal Medicine, Hematology
Pécs, 7624
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
"UMHAT Sveta Marina" EAD.
Varna, 9010
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
Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center
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
About this study
Official title: A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.
- Conditions
- Von Willebrand Diseases
- Being tested
- Von Willebrand Factor-Containing Product
- 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.
Von Willebrand Factor-Containing ProductDrug
Active substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country
| Study group | Role | What they receive |
|---|---|---|
| Patients undergoing treatment with a VWF-containing product | Patients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months Receives: Drug: Von Willebrand Factor-Containing Product |
What is being measured
The primary outcome is the main question the study is designed to answer.
Total Annualized Bleeding Rate (TABR)
The total annualized bleeding rate (TABR) will be calculated as the total number of spontaneous bleeds, traumatic BEs, and other BEs occurring in the time period between the start of data collection for each patient and the Study Completion Visit, divided by the duration (in years) between the start of data collection and the Study Completion Visit. Surgery periods, and BEs occurring within these surgery periods, will be excluded from the calculation of TABR.
Measured: Screening through study completion (6 months)
Secondary outcomes (10)
Spontaneous Annualized Bleeding Rate (SABR)
Spontaneous annualized bleeding rate (SABR), calculated in analogy with TABR. This includes all bleeding episodes that occurred spontaneously.
Measured: Screening through study completion (6 months)
Consumption of the VWF-containing Product
Data on the consumption of the VWF-containing product (VWF/FVIII IU/kg per month per patient) used for routine on-demand treatment
Measured: Screening through study completion (6 months)
Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
The efficacy assessment of bleeding episodes at the end of a BE was evaluated on a 4 point scale by the patient/legal guardian (together with the Investigator in case of on-site treatment) including the four items 'excellent,' 'good,' moderate,' and 'none.' The assessment was excellent when bleeding was completely stopped within 3 days in case of minor bleed, within 7 days in case of major bleed, and within 10 days in case of gastrointestinal bleed; Good when bleeding was completely stopped, but time and/or dose slightly exceeded expectations ; Moderate when bleeding could be stopped only by significantly exceeding time and/or dose expectations; and None when bleeding could be stopped only by using other VWF-containing products.
Measured: Screening through study completion (6 months)
Number of Surgery With Successful/Unsuccessful Efficacy Assessment
Effectiveness of VWF-containing product in surgical prophylaxis based on the proportion of surgeries successfully treated. Overall treatment efficacy will be assessed at the end of the postoperative period by the treating physician using predefined criteria of 'Excellent', 'Good', 'Moderate/Poor' or 'None'.
Measured: From start of surgery until end of post-operative period (within 8 days after surgery)
Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)
QoL assessment based on the results from the PROMIS-29 survey to monitor and evaluate the physical, mental, and social health in all patients, using a scale of a minimum score of 0 and a maximum score of 10, with higher scores representing a better outcome. The survey covers seven domains from the most relevant areas of self-reported health (depression, anxiety, physical function, pain interference, fatigue, sleep disturbance and ability to participate in social roles and activities) for the majority of people with chronic illness. PROMIS scores have a mean of 50 and standard deviation (SD) of 10 in a referent population. Full details of cut off points for each domain can be found here: https://www.healthmeasures.net/score-and-interpret/interpret-scores/promis/promis-score-cut-points
Measured: At screening visit
Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)
QoL assessment based on the results from the SF-36v2 questionnaire to measure functional health and well-being in patients ≥16 years. SF-36v2 ranks 8 different domains using a scale standardized with a scoring algorithm to obtain a score ranging from 0 to 100.The eight health domains include physical functioning (PF), role physical (RP), bodily pain (BP), general health problems (GH), vitality (VT), social functioning (SF), role emotional (RE) and general mental health (MH). Higher scores indicate better health status, and a mean score of 50 has been articulated as a normative value for all scales.
Measured: At screening visit
Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10)
QoL assessment based on the results from a SF-10 parent-completed questionnaire for patients ≥5.5 and <16 years of age, in order to score physical and psychosocial health. SF-10 uses norm-based scoring where scales have a standardized mean value of 50 and standard deviation of 10.
Measured: At screening
Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)
Joint health status will be assessed using the Hemophilia Joint Health Score (HJHS), which has been specifically validated for the assessment of the clinical outcome in VWD. HJHS evaluates six index joints to produce a score between 0-124. Higher scores indicate worse joint health.
Measured: At screening
Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score
Bleeding information from each menstrual period while in this study will be collected using the Pictorial Blood Loss Assessment Chart (PBAC). The PBAC will be provided to all female patients of child-bearing potential. The data documented in the PBAC and the investigator-calculated final score will be recorded in the eCRF. The PBAC records pad and tampon use (as either light [1 point], medium [5 points], or heavy [10 points] flow), clots (small [1 point] or large [5 points]), and flooding episodes (1 point each) which can be recorded as many times as necessary any day of the month. The PBAC is scored from 0 (no bleeding) onwards, with a score of >100 defining abnormal coagulation and heavy menstrual bleeding (corresponds to >80ml of blood loss per menstrual cycle).
Measured: Screening through study completion (6 months)
Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate
Noxious and unintended reactions arising from the use of Wilate will be monitored throughout the study.
Measured: Screening through study completion (6 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 start25 Jun 2019
- Primary completion31 Jan 2021
- Study completion31 Jan 2021
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 (10)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
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 56 participants (actual)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Cristina Solomon, MD, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04053699
- Sponsor's protocol ID
- WIL-29
- Status last verified
- Mar 2023
- First submitted
- 23 Jul 2019
- Last updated
- 7 Dec 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Sadler 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 23633542Mondorf W, Siegmund B, Mahnel R, Richter H, Westfeld M, Galler A, Pollmann H. Haemoassist--a hand-held electronic patient diary for haemophilia home care. Haemophilia. 2009 Mar;15(2):464-72. doi: 10.1111/j.1365-2516.2008.01941.x. Epub 2009 Feb 16.
PubMed 19226411Broderick CR, Herbert RD, Latimer J, Mathieu E, van Doorn N, Curtin JA. Feasibility of short message service to document bleeding episodes in children with haemophilia. Haemophilia. 2012 Nov;18(6):906-10. doi: 10.1111/j.1365-2516.2012.02869.x. Epub 2012 Jun 11.
PubMed 22681182Sholapur NS, Barty R, Wang G, Almonte T, Heddle NM. A survey of patients with haemophilia to understand how they track product used at home. Haemophilia. 2013 Sep;19(5):e289-95. doi: 10.1111/hae.12170. Epub 2013 May 15.
PubMed 23672744Maruish 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.
Hays 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 29569016Sidonio RF Jr, Boban A, Dubey L, Inati A, Kiss C, Boda Z, Lissitchkov T, Nemes L, Novik D, Peteva E, Taher AT, Timofeeva MA, Vilchevska KV, Vdovin V, Werner S, Knaub S, Djambas Khayat C. von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Adv. 2024 Mar 26;8(6):1405-1414. doi: 10.1182/bloodadvances.2023011742.
PubMed 38237075
Study at a glance
From the registry record, last updated by the sponsor on 7 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment
- ClinicalTrials.gov ID
- NCT04053699
- Acronym / sponsor ID
- WIL-29
- Conditions
- Von Willebrand Diseases
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 56 participants (actual)
- Lead sponsor
- OctapharmaIndustry (pharma, biotech, devices)
- Study start
- 25 Jun 2019 (actual)
- Primary completion
- 31 Jan 2021 (actual)
- Study completion
- 31 Jan 2021 (actual)
- First posted
- 12 Aug 2019 (actual)
- Results
- Posted 7 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to prospectively obtain reliable data on the bleeding and treatment pattern of patients with VWD undergoing on-demand treatment with a VWF-containing product over a period of 6 months. The data obtained will be used as a basis for historical comparisons with the bleeding and treatment pattern obtained from a clinical study on the efficacy of prophylactic treatment with a VWF/FVIII concentrate.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients undergoing treatment with a VWF-containing product | – | Drug: Von Willebrand Factor-Containing ProductPatients with type 3, type 2 (except 2N), or severe type 1 VWD undergoing routine on-demand treatment with a VWF-containing product over a period of 6 months |
Who may qualify
- Ages
- 66 months and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Overall, 55 previously treated patients aged ≥5.5 years at the time of enrolment, with type 3, type 2 (except 2N), or severe type 1 VWD will be included into this study. Of these 55 patients, at least 6 should have type 3 VWD and at least 6 should be ≥5.5 to <16 years of age.
Key inclusion criteria (6 listed)
- Male or female patients aged ≥5.5 years at the time of enrolment
- VWD type 1 (baseline von Willebrand factor activity [VWF: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 frequent on-demand treatment with a VWF-containing product
- In female patients of child-bearing potential using hormonal contraception, the medication class should remain unchanged for the duration of their study participation
- Voluntarily given, fully informed written and signed consent obtained before collection of any patient data
Key exclusion criteria (12 listed)
- Patients currently on prophylaxis for VWD (except for perioperative prophylaxis) as well as patients having received treatment once a month for menstrual bleeding, but not for any other bleeds
- Patients whose VWD treatment is planned to be switched from on-demand to prophylactic treatment in the next 6 months
- History, or current suspicion, of VWF or FVIII inhibitors
- Medical history of a thromboembolic event within 6 months before enrolment
- Severe liver or kidney diseases as described in the medical records
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 |
|---|---|---|
| Von Willebrand Factor-Containing ProductActive substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country | Drug | Patients undergoing treatment with a VWF-containing product |
Primary outcome
Total Annualized Bleeding Rate (TABR)
Time frame: Screening through study completion (6 months)
The total annualized bleeding rate (TABR) will be calculated as the total number of spontaneous bleeds, traumatic BEs, and other BEs occurring in the time period between the start of data collection for each patient and the Study Completion Visit, divided by the duration (in years) between the start of data collection and the Study Completion Visit. Surgery periods, and BEs occurring within these surgery periods, will be excluded from the calculation of TABR.
10 secondary outcomes
Spontaneous Annualized Bleeding Rate (SABR)
Time frame: Screening through study completion (6 months)
Consumption of the VWF-containing Product
Time frame: Screening through study completion (6 months)
Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
Time frame: Screening through study completion (6 months)
Number of Surgery With Successful/Unsuccessful Efficacy Assessment
Time frame: From start of surgery until end of post-operative period (within 8 days after surgery)
Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)
Time frame: At screening visit
Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)
Time frame: At screening visit
Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10)
Time frame: At screening
Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)
Time frame: At screening
Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score
Time frame: Screening through study completion (6 months)
Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate
Time frame: Screening through study completion (6 months)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 7 Dec 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- 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.
- Background: PubMed 19226411Mondorf W, Siegmund B, Mahnel R, Richter H, Westfeld M, Galler A, Pollmann H. Haemoassist--a hand-held electronic patient diary for haemophilia home care. Haemophilia. 2009 Mar;15(2):464-72. doi: 10.1111/j.1365-2516.2008.01941.x. Epub 2009 Feb 16.
- Background: PubMed 22681182Broderick CR, Herbert RD, Latimer J, Mathieu E, van Doorn N, Curtin JA. Feasibility of short message service to document bleeding episodes in children with haemophilia. Haemophilia. 2012 Nov;18(6):906-10. doi: 10.1111/j.1365-2516.2012.02869.x. Epub 2012 Jun 11.
- Background: PubMed 23672744Sholapur NS, Barty R, Wang G, Almonte T, Heddle NM. A survey of patients with haemophilia to understand how they track product used at home. Haemophilia. 2013 Sep;19(5):e289-95. doi: 10.1111/hae.12170. Epub 2013 May 15.
- 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 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.
- Derived from this study: PubMed 38237075Sidonio RF Jr, Boban A, Dubey L, Inati A, Kiss C, Boda Z, Lissitchkov T, Nemes L, Novik D, Peteva E, Taher AT, Timofeeva MA, Vilchevska KV, Vdovin V, Werner S, Knaub S, Djambas Khayat C. von Willebrand factor/factor VIII concentrate (Wilate) prophylaxis in children and adults with von Willebrand disease. Blood Adv. 2024 Mar 26;8(6):1405-1414. doi: 10.1182/bloodadvances.2023011742.
- 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, MD study director
Octapharma
15 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Diseases, run by Octapharma at 15 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 6 inclusion and 12 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 7 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Hungary, Lebanon, Bulgaria, 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.
Are results available for this study?
Yes. Results are posted in the registry (first posted 7 Dec 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: Screening through study completion (6 months)). 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 NCT04053699, which also lists 10 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 7 Dec 2023.