Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
NCT ID: NCT04052698
Last Updated: 2023-10-25
Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
43 participants
INTERVENTIONAL
2020-06-18
2022-04-23
Brief Summary
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Detailed Description
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Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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All patients
Patients with type 3, type 2 (except 2N), or severe type 1 VWD aged ≥6 years at screening receiving Wilate for prophylactic treatment.
Wilate
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.
Interventions
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Wilate
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.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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
Exclusion Criteria
* 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)
* 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
6 Years
ALL
No
Sponsors
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Octapharma
INDUSTRY
Responsible Party
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Principal Investigators
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Cristina Solomon
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
Pediatric Clinic of Haematology and Oncology
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
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
Communal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center"of Lviv Regional Council
Lviv, , Ukraine
Countries
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References
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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.
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.
U S Department Of Health And Human Services. Opioid abuse in the United States and Department of Health and Human Services actions to address opioid-drug-related overdoses and deaths. J Pain Palliat Care Pharmacother. 2015 Jun;29(2):133-9. doi: 10.3109/15360288.2015.1037530.
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.
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.
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.
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.
van 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.
Janssen 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.
Higham 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.
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
Related Links
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U.S Department of Health and Human Services, Health Measures
Other Identifiers
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WIL-31
Identifier Type: -
Identifier Source: org_study_id
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