Clinical trial NCT04053699: Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment
Official title: A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Von Willebrand Diseases
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2019-08-12
Enrollment: 56
Sex: ALL
Minimum age (years): 66
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

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

Exclusion Criteria:

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
* 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

Primary outcomes:
measure: Total Annualized Bleeding Rate (TABR)
timeFrame: Screening through study completion (6 months)
Secondary outcomes:
measure: Spontaneous Annualized Bleeding Rate (SABR)
timeFrame: Screening through study completion (6 months)
measure: Consumption of the VWF-containing Product
timeFrame: Screening through study completion (6 months)
measure: Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale
timeFrame: Screening through study completion (6 months)
measure: Number of Surgery With Successful/Unsuccessful Efficacy Assessment
timeFrame: From start of surgery until end of post-operative period (within 8 days after surgery)
measure: Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)
timeFrame: At screening visit
measure: Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)
timeFrame: At screening visit
measure: Quality of Life (QoL) Assessed Using a 10-item Short Form Health Survey (SF-10)
timeFrame: At screening
measure: Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)
timeFrame: At screening
measure: Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score
timeFrame: Screening through study completion (6 months)
measure: Number of Participants With Adverse Drug Reactions (ADRs) Associated With Use of Wilate
timeFrame: Screening through study completion (6 months)
Other outcomes:
Locations:
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta
state: Georgia
city: Gomel
country: Belarus
facility: Republican Research Center for Radiation Medicine and Human Ecology
city: Sofia
country: Bulgaria
facility: Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
city: Varna
country: Bulgaria
facility: "UMHAT Sveta Marina" EAD.
city: Zagreb
country: Croatia
facility: University Hospital Centre Zagreb
city: Budapest
country: Hungary
facility: Medical Centre Hungarian Defence Forces
city: Debrecen
country: Hungary
facility: Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ
city: Pécs
country: Hungary
facility: University Clinical Center, Department of Internal Medicine, Hematology
city: Beirut
country: Lebanon
facility: Hotel Dieu de France Hospital
city: Beirut
country: Lebanon
facility: American University of Beirut Medical Center
city: Tripoli
country: Lebanon
facility: Nini Hospital
city: Kirov
country: Russian Federation
facility: Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal
city: Moscow
country: Russian Federation
facility: Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis
city: Kyiv
country: Ukraine
facility: State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology
city: Lviv
country: Ukraine
facility: Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center
Study officials:
affiliation: Octapharma
name: Cristina Solomon, MD
role: STUDY_DIRECTOR
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citation: 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.
citation: 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.