Clinical trial NCT04052698: Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
Official title: Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With Von Willebrand Disease (VWD)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Von Willebrand Diseases
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2019-08-12
Enrollment: 43
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated adult patients with VWD, to obtain additional data on the safety and efficacy of Wilate in previously treated patients with VWD undergoing regular prophylaxis.


Eligibility criteria: Inclusion Criteria:

Patients who meet all of the following criteria are eligible for the study:

* 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:

Patients who meet any of the following criteria are not eligible for the study:

* 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

Primary outcomes:
measure: Total Annualized Bleeding Rate (TABR)
timeFrame: 12 months
measure: Comparison of Total Annualized Bleeding Rates (TABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29
timeFrame: 12 Months
Secondary outcomes:
measure: Spontaneous Annualized Bleeding Rate (SABR)
timeFrame: 12 months
measure: Comparison of Spontaneous Annualized Bleeding Rates (SABR) During Prophylaxis Treatment in Study WIL-31 to On-demand Treatment in the Same Patient Population in the Preceding Study WIL-29.
timeFrame: 12 Months
measure: Wilate Consumption for Prophylaxis (mFAS Population)
timeFrame: 12 months
measure: Incremental In Vivo Recovery (IVR) of Von Willebrand Factor Activity (VWF:RCo)
timeFrame: From baseline and 12-month visit
measure: Incremental In Vivo Recovery (IVR) of FVIII
timeFrame: Baseline and 12-month visit
measure: Efficacy of Wilate in the Treatment of Breakthrough Bleeding Events (BEs)
timeFrame: 12 months
measure: Wilate Exposure for Prophylaxis (mFAS Population)
timeFrame: 12 months
Other outcomes:
measure: Efficacy Rating for Wilate in Surgical Prophylaxis
timeFrame: 12 months
measure: Quality of Life (QoL) Assessed Using the Patient-Reported Outcomes Measurement Information System (PROMIS-29)
timeFrame: 12 months
measure: Quality of Life (QoL) Assessed Using a 36-Item Short Form Health Survey, Version 2 (SF-36v2)
timeFrame: 12 months
measure: Quality of Life (QoL) Assessed Using a 10-Item Short Form Health Survey (SF-10)
timeFrame: 12 months
measure: Joint Health Status Assessed Using Hemophilia Joint Health Score (HJHS)
timeFrame: Baseline and 12 months
measure: Menstrual Bleeding Assessed Using Pictorial Blood Loss Assessment Chart (PBAC) Score
timeFrame: 12 months
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: Pediatric Clinic of Haematology and Oncology
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: 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: Communal Nonprofit Enterprise "Western Ukrainian Specialized Children's Medical Center"of Lviv Regional Council
Study officials:
affiliation: Octapharma
name: Cristina Solomon
role: STUDY_DIRECTOR
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