Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
NCT ID: NCT04053699
Last Updated: 2023-12-07
Study Results
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View full resultsBasic Information
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COMPLETED
56 participants
OBSERVATIONAL
2019-06-25
2021-01-31
Brief Summary
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Detailed Description
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Conditions
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Study Design
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COHORT
PROSPECTIVE
Study Groups
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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
Von Willebrand Factor-Containing Product
Active substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country
Interventions
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Von Willebrand Factor-Containing Product
Active substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country
Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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
* 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
66 Months
ALL
No
Sponsors
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Octapharma
INDUSTRY
Responsible Party
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Principal Investigators
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Cristina Solomon, MD
Role: STUDY_DIRECTOR
Octapharma
Locations
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Children's Healthcare of Atlanta
Atlanta, Georgia, United States
Republican Research Center for Radiation Medicine and Human Ecology
Homyel, , Belarus
Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
Sofia, , Bulgaria
"UMHAT Sveta Marina" EAD.
Varna, , Bulgaria
University Hospital Centre Zagreb
Zagreb, , Croatia
Medical Centre Hungarian Defence Forces
Budapest, , Hungary
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
Debrecen, , Hungary
University Clinical Center, Department of Internal Medicine, Hematology
Pécs, , Hungary
Hotel Dieu de France Hospital
Beirut, , Lebanon
American University of Beirut Medical Center
Beirut, , Lebanon
Nini Hospital
Tripoli, , Lebanon
Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal
Kirov, , Russia
Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis
Moscow, , Russia
State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology
Kyiv, , Ukraine
Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center
Lviv, , Ukraine
Countries
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References
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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.
Rodeghiero 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.
Castaman 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.
Mondorf 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.
Broderick 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.
Sholapur 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.
Maruish 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.
Sidonio 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.
Provided Documents
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Document Type: Study Protocol
Document Type: Statistical Analysis Plan
Other Identifiers
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WIL-29
Identifier Type: -
Identifier Source: org_study_id