Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment

A Prospective, Multicenter, Non-Interventional Study Evaluating the Bleeding Incidence in Patients With Von Willebrand Disease Undergoing On-Demand Treatment

Sponsors

Lead Sponsor: Octapharma

Source Octapharma
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.

Overall Status Recruiting
Start Date June 25, 2019
Completion Date December 2020
Primary Completion Date October 2020
Study Type Observational
Primary Outcome
Measure Time Frame
Total annualized bleeding rate (TABR) 6 months
Secondary Outcome
Measure Time Frame
Spontaneous annualized bleeding rate (SABR) 6 months
Consumption of the VWF-containing product 6 months
Effectiveness of VWF-containing product in the treatment of Bleeding Events (BEs) 6 months
Effectiveness of VWF-containing product in surgical prophylaxis 6 months
Quality of Life (QoL) assessed using the Patient-Reported Outcomes Measurement Information System (PROMIS-29) 6 months
Quality of Life (QoL) assessed using a 36-Item Short Form Health Survey, version 2 (SF-36v2) 6 months
Quality of Life (QoL) assessed using a 10-item Short Form Health Survey (SF-10) 6 months
Joint health status assessed using Hemophilia Joint Health Score (HJHS) 6 months
Menstrual bleeding assessed using Pictorial Blood Loss Assessment Chart (PBAC) score 6 months
Number and type of adverse drug reactions (ADRs) associated with use of Wilate 6 months
Enrollment 35
Condition
Intervention

Intervention Type: Drug

Intervention Name: VWF-containing products

Description: Active substances: VWF concentrates, VWF/FVIII concentrates, Cryoprecipitate VWF-containing products licensed in each participating country

Arm Group Label: Patients undergoing treatment with a VWF-containing product

Other Name: Wilate/ von Willebrand factor / Factor VIII (plasma derived)

Eligibility

Sampling Method: Non-Probability Sample

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

Gender: All

Minimum Age: 66 Months

Maximum Age: N/A

Healthy Volunteers: No

Overall Official
Last Name Role Affiliation
Cristina Solomon, MD Study Director Octapharma
Overall Contact

Last Name: Cristina Solomon, MD

Phone: +41554512189

Email: [email protected]

Location
Facility: Status:
Children's Healthcare of Atlanta | Atlanta, Georgia, 30329, United States Recruiting
University Clinical Health | Memphis, Tennessee, 38104, United States Recruiting
Republican Research Center for Radiation Medicine and Human Ecology | Gomel, Belarus Recruiting
Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia | Sofia, Bulgaria Recruiting Toshko Lissitchkov
"UMHAT Sveta Marina" EAD. | Varna, 9010, Bulgaria Recruiting Valeriya Kaleva
University Hospital Centre Zagreb | Zagreb, 10000, Croatia Recruiting Silva Zupančić Šalek
Medical Centre Hungarian Defence Forces | Budapest, 1134, Hungary Not yet recruiting Laszlo Nemes
Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ | Debrecen, 4032, Hungary Recruiting Csongor Kiss
University Clinical Center, Department of Internal Medicine, Hematology | Pécs, 7624, Hungary Recruiting Ágnes Nagy
Hotel Dieu de France Hospital | Beirut, BP166830, Lebanon Recruiting Claudia Khayat
IMSP Mother and Child Institute | Chisinau, MD-2062, Moldova, Republic of Recruiting Valentin Turea
Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal | Kirov, 610027, Russian Federation Recruiting Timofeeva Margarita Arkadievna
Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis | Moscow, 119049, Russian Federation Recruiting Vdovin Vladimir Victorovich
State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology | Kyiv, 01135, Ukraine Recruiting Kateryna Viktorivna Vilchevska
Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center | Lviv, 79035, Ukraine Recruiting Leonid Yaroslavovych Dubei
Location Countries

Belarus

Bulgaria

Croatia

Hungary

Lebanon

Moldova, Republic of

Russian Federation

Ukraine

United States

Verification Date

February 2020

Responsible Party

Type: Sponsor

Has Expanded Access No
Condition Browse
Arm Group

Label: Patients undergoing treatment with a VWF-containing product

Description: 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

Patient Data Undecided
Study Design Info

Observational Model: Cohort

Time Perspective: Prospective

Source: ClinicalTrials.gov