Bleeding Incidence in VWD Patients Undergoing On-Demand Treatment

NCT04053699 | Completed Results FDA Drug

• 1 other identifier: WIL-29
• Study Type: observational
• Target: 56
• Locations: 8 countries
• Sites: 15

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.

Conditions

Von Willebrand Diseases

Outcome Measures

Primary Outcomes (1)

• 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.

  Screening through study completion (6 months)

Secondary Outcomes (10)

• Spontaneous Annualized Bleeding Rate (SABR)

  Screening through study completion (6 months)

• Consumption of the VWF-containing Product

  Screening through study completion (6 months)

• Number of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale

  Screening through study completion (6 months)

• Number of Surgery With Successful/Unsuccessful Efficacy Assessment

  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)

  At screening visit

Study Arms (1)

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

Drug: Von Willebrand Factor-Containing Product

Interventions

Von Willebrand Factor-Containing Product DRUG

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

Patients undergoing treatment with a VWF-containing product

Eligibility Criteria

• Age: 66 Months+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• 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.

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (15)

Children's Healthcare of Atlanta

Atlanta, Georgia, 30329, United States

Location

Republican Research Center for Radiation Medicine and Human Ecology

Homyel, Belarus

Location

Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia

Sofia, Bulgaria

Location

"UMHAT Sveta Marina" EAD.

Varna, 9010, Bulgaria

Location

University Hospital Centre Zagreb

Zagreb, 10000, Croatia

Location

Medical Centre Hungarian Defence Forces

Budapest, 1134, Hungary

Location

Debreceni Egyetem Klinikai Központ, Regionális Haemophilia és Thrombophilia Központ

Debrecen, 4032, Hungary

Location

University Clinical Center, Department of Internal Medicine, Hematology

Pécs, 7624, Hungary

Location

Hotel Dieu de France Hospital

Beirut, BP166830, Lebanon

Location

American University of Beirut Medical Center

Beirut, Lebanon

Location

Nini Hospital

Tripoli, Lebanon

Location

Federal State Budgetary Scientific Institution Kirov Scientific-Research Institute of Hematology and Blood Transfusion of Federal

Kirov, 610027, Russia

Location

Morosovskaya Children Clinical Hospital, Moscow Health Department, Department of General Hematology with the Pathology of Hemostasis

Moscow, 119049, Russia

Location

State Institution "National Children's Specialized Hospital "OKHMATDYT" of the Ministry of Health of Ukraine," Center of Hemostasis Pathology

Kyiv, 01135, Ukraine

Location

Community Institution of Lviv Oblast Council "West-Ukrainian Specialized Children's Medical Center

Lviv, 79035, Ukraine

Location

Related Publications (10)

• 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.

  PMID: 8052974 BACKGROUND

• 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.

  PMID: 19474451 BACKGROUND

• 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.

  PMID: 23633542 BACKGROUND

• 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.

  PMID: 19226411 BACKGROUND

• 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.

  PMID: 22681182 BACKGROUND

• 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.

  PMID: 23672744 BACKGROUND

• Maruish M. User's manual for the SF-36v2 Health Survey (3rd edition). Optum Incorporated; 2011.

  BACKGROUND

• 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.

  BACKGROUND

• 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.

  PMID: 29569016 BACKGROUND

• 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.

  PMID: 38237075 DERIVED

MeSH Terms

Conditions

von Willebrand Diseases

Interventions

von Willebrand Factor

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Blood Platelet Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Blood Coagulation Factors; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Biological Factors

Results Point of Contact

• Title: Sigurd Knaub, Senior VP CR&D Haematology
• Organization: Octapharma

Sigurd.Knaub@octapharma.com

01554512141

Study Officials

• Cristina Solomon, MD

  Octapharma

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 23, 2019
• First Posted: August 12, 2019
• Study Start: June 25, 2019
• Primary Completion: January 31, 2021
• Study Completion: January 31, 2021
• Last Updated: December 7, 2023
• Results First Posted: December 7, 2023

Record last verified: 2023-03

Locations

BE (1); HU (3); LE (3); UA (2); BU (2); CR (1); US (1); RU (2)