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Trial details
CLINICAL STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF WILATE DURING PROPHYLAXIS IN PREVIOUSLY TREATED PATIENTS WITH VON WILLEBRAND DISEASE (VWD)
Current status:
Approved
|
Date registered:
06/07/2020
|
Date last updated:
09/06/2020
Trial version(s)
Current: 31/05/2020
Click here to view the tips and fields' descriptions
Main Information
Primary registry identifying number
LBCTR2020063508
Protocol number
WIL-31
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Study is not registered in the country of origin (Switzerland) as the number of Von Willebrand Disease patients is low. Letter from the Sponsor is attached.
Type of registration
Prospective
Date of registration in national regulatory agency
01/06/2020
Primary sponsor
Octapharma AG
Primary sponsor: Country of origin
Switzerland
Public title
CLINICAL STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF WILATE DURING PROPHYLAXIS IN PREVIOUSLY TREATED PATIENTS WITH VON WILLEBRAND DISEASE (VWD)
Acronym
WIL-31
Scientific title
CLINICAL STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF WILATE DURING PROPHYLAXIS IN PREVIOUSLY TREATED PATIENTS WITH VON WILLEBRAND DISEASE (VWD)
Acronym
WIL-31
Brief summary of the study: English
This is a prospective, non-controlled, international, multi-center phase 3 study investigating the efficacy and safety of Wilate in previously treated patients with Von Willebrand Disease. Participation in the study may help by reduce the number of bleeding episodes requiring on demand treatment. The treatment of VWD is already approved in Lebanon by the Ministry of Health under the name Wilate. The main goal of this study is to find out to what extent Wilate, when administered regularly and prophylactically, prevents the occurrence of bleeding episodes in patients with type 3, type 2 (except 2N), or severe type 1 VWD. Participating patients will be followed up for 12 months during which they will visit their treating physician 7 times.
Brief summary of the study: Arabic
هذه دراسة مستقبلية ، غير خاضعة للرقابة ، دولية ، متعددة المراكز ، مرحلة 3 ، تحقق في فعالية وسلامة Wilate في المرضى الذين سبق علاجهم بمرض فون ويليبراند. قد تساعد المشاركة في الدراسة عن طريق تقليل عدد نوبات النزف التي تتطلب معالجة عند الطلب. تمت الموافقة على علاج VWD بالفعل في لبنان من قبل وزارة الصحة تحت اسم Wilate. الهدف الرئيسي من هذه الدراسة هو معرفة إلى أي مدى يمنع Wilate ، عند إعطائه بانتظام وبشكل وقائي ، حدوث نوبات النزف في المرضى الذين يعانون من النوع 3 ، النوع 2 (باستثناء 2N) ، أو النوع VWD الشديد 1. ستتم متابعة المرضى المشاركين لمدة 12 شهرًا يزورون خلالها الطبيب المعالج 7 مرات
Health conditions/problem studied: Specify
Von Willebrand Disease
Interventions: Specify
This is an open label study to provide VWD patients with Wilate prophylactically.
Key inclusion and exclusion criteria: Inclusion 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
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
6
Key inclusion and exclusion criteria: Age maximum
80
Key inclusion and exclusion criteria: Exclusion criteria
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/mL 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
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Prophylaxis
Trial scope: Specify scope
Study design: Allocation
N/A
Study design: Masking
Open (masking not used)
Study design: Control
Uncontrolled
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Single
IMP has market authorization
Yes, Lebanon and Worldwide
IMP has market authorization: Specify the countries
Worldwide
Name of IMP
Wilate
Year of authorization
2005
Month of authorization
2
Type of IMP
Plasma derived
Pharmaceutical class
plasma-derived coagulation factor complex consisting of von Willebrand factor/coagulation factor VIII complex
Therapeutic indication
Von Willebrand Disease type 3, type 2 (except 2N), and severe type 1.
Therapeutic benefit
To decrease bleeding events in patients with VWD.
Biospecimen retention
Samples with DNA**
Biospecimen description
The genetic testing will be done only to patients with VWD 2B using a blood sample. In patients aged ≥17 years, this blood sample will be taken during the Baseline IVR Visit, and in patients aged 6–16 years, it will be taken during the Screening Visit. If no mutations related to type 2B VWD are identified, the same sample will be used to perform genetic testing to exclude the presence of pseudo-VWD.
Target sample size
28
Actual enrollment target size
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
01/06/2020
Date of study closure: Type
Anticipated
Date of study closure: Date
30/11/2021
Recruitment status
Pending
Date of completion
IPD sharing statement plan
No
IPD sharing statement description
Undecided
Additional data URL
https://clinicaltrials.gov/ct2/show/NCT04052698?term=WIL-31&draw=2&rank=1
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
Clinicaltrials.gov
NCT04052698
EudraCT
2018-004675-13
Sources of Monetary or Material Support
Name
Octapharma AG
Secondary Sponsors
Name
N/A
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Hanen
Hamid
Beirut
Lebanon
+9613761145
hanen.hamid@ergomedplc.com
Ergomed PLC
Scientific
Irina
Kruzhkova
Lachen
Switzerland
+41554512173
Irina.kruzhkova@octapharma.com
Octapharma AG
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu de France
Dr. Claudia Khayat
Pediatric Hematolgy
Approved
American University of Beirut-Medical Center
Dr. Ali Taher
Hematology/Oncology
Not approved
Nini Hospital
Dr. Adlette Inati
Pediatric Hematology
Not approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
04/02/2020
Mrs. Virginia El Khoury
cue@usj.edu.lb
+961 1 421 229
Countries of Recruitment
Name
Lebanon
United States of America
Belarus
Bulgaria
Croatia
Hungary
Russian Federation
Ukraine
Health Conditions or Problems Studied
Condition
Code
Keyword
Hematology
Von Willebrand s disease (D68.0)
Von Willebrand Disease
Interventions
Intervention
Description
Keyword
Intravenous injection
prophylactic IV Infusion of Wilate
Wilate
PK sampling
only applicable for patients younger than 17 years
Pharmacokinetic testing
Primary Outcomes
Name
Time points
Measure
total annualised bleeding rate decrease by more than 50%
12 months after recruitment
Number of on demand treatment with Von Willebrand Factor Containing product
Key Secondary Outcomes
Name
Time points
Measure
Spontaneous annualised bleeding rate
1 month, 2 months, 3 months, 6 months, 9 months and 12 months after recruitment
Number of on demand treatment with Von Willebrand Factor Containing product
Von Willebrand Factor Activity
1 month, 2 months, 3 months, 6 months, 9 months and 12 months after recruitment
Blood testing and number of on demand treatment with Von Willebrand containing product
Safety and tolerability of Wilate
From the inclusion of patients till 12 months after recruitment
Adverse Events experienced by participating patients
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Download as PDF
Save a PDF copy of the summary of the trial