Main
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
Register:
|
EUCTR |
Last refreshed on:
|
26 October 2021 |
Main ID: |
EUCTR2018-004675-13-BG |
Date of registration:
|
27/02/2019 |
Prospective Registration:
|
Yes |
Primary sponsor: |
|
Public title:
|
Study to investigate the study drug (WILATE) in patients with Von Willebrand Disease.
|
Scientific title:
|
CLINICAL STUDY TO INVESTIGATE THE EFFICACY AND SAFETY OF WILATE
DURING PROPHYLAXIS IN PREVIOUSLY TREATED PATIENTS WITH VON WILLEBRAND DISEASE (VWD)
|
Date of first enrolment:
|
|
Target sample size:
|
42 |
Recruitment status: |
Not Recruiting |
URL:
|
https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2018-004675-13 |
Study type:
|
Interventional clinical trial of medicinal product |
Study design:
|
Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:
|
Phase:
|
Human pharmacology (Phase I): no
Therapeutic exploratory (Phase II): no
Therapeutic confirmatory - (Phase III): yes
Therapeutic use (Phase IV): no
|
|
Countries of recruitment
|
Belarus
|
Bulgaria
|
Croatia
|
Hungary
|
Lebanon
|
Russian Federation
|
Ukraine
|
United States
|
Contacts
|
Name:
|
Clinical Trial Manager
|
Address:
|
Seidenstrasse 2
CH-8853
Lachen
Switzerland |
Telephone:
|
0041554512184 |
Email:
|
Sylvia.Werner@octapharma.com |
Affiliation:
|
Octapharma AG |
|
Name:
|
Clinical Trial Manager
|
Address:
|
Seidenstrasse 2
CH-8853
Lachen
Switzerland |
Telephone:
|
0041554512184 |
Email:
|
Sylvia.Werner@octapharma.com |
Affiliation:
|
Octapharma AG |
| |
Key inclusion & exclusion criteria
|
Inclusion criteria: 1. Patients aged =6 years at the time of screening 2. 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 3. Currently receiving on-demand treatment with a VWF-containing product AND having experienced at least 6 BEs (excluding menstrual bleeds) over a period of 6 months, with at least 2 of these BEs treated with a VWF containing product AND having records available to reliably evaluate the type, frequency, and treatment of BEs in this 6-month period OR Having switched to prophylactic treatment with a VWF-containing product within the past 2 years AND having records available to reliably evaluate the type, frequency, and treatment of BEs over a period of 6 months of on-demand treatment 4. 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 contraception is used, the medication class should remain unchanged for the duration of the study 5. Voluntarily given, fully informed written and signed consent obtained before any study-related procedures are conducted
Are the trial subjects under 18? yes Number of subjects for this age range: 12 F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 30 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 0
Exclusion criteria: 1. 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 2. History, or current suspicion, of VWF or FVIII inhibitors 3. Medical history of a thromboembolic event within 1 year before enrolment 4. Severe liver or kidney diseases (alanine aminotransferase [ALAT] and aspartate transaminase [ASAT] levels >5 times of upper limit of normal, creatinine >120 µmol/L) 5. Platelet count <100,000/µL at screening (except for VWD type 2B) 6. Body weight <20 kg at screening 7. Patients receiving, or scheduled to receive, immunosuppressant drugs (other than anti-retroviral chemotherapy), such as prednisone (equivalent to >10 mg/day), or similar drugs 8. Pregnant or breast-feeding at the time of enrolment 9. Cervical or uterine conditions causing abnormal uterine bleeding (including infection, dysplasia) 10. Treatment with any investigational medicinal product (IMP) in another interventional clinical study currently or within 4 weeks before enrolment 11. Other coagulation disorders or bleeding disorders due to anatomical reasons 12. Known hypersensitivity to any of the components of the study drug
Age minimum:
Age maximum:
Gender:
Female: yes Male: yes
|
Health Condition(s) or Problem(s) studied
|
Von Willebrand disease, type 3, type 2 (except 2N), or severe type 1 MedDRA version: 20.0
Level: LLT
Classification code 10055168
Term: Von Willebrand's factor deficiency
System Organ Class: 100000004850
|
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
|
Intervention(s)
|
Trade Name: Wilate 500 Product Name: Wilate Pharmaceutical Form: Lyophilisate and solvent for solution for injection INN or Proposed INN: Human Coagulation Factor VIII, Von Willebrand Factor Complex Current Sponsor code: WILATE Other descriptive name: HUMAN COAGULATION FACTOR VIII, VON WILLEBRAND FACTOR COMPLEX Concentration unit: IU international unit(s) Concentration type: equal Concentration number: 500-
Trade Name: Wilate 1000 Product Name: Wilate Pharmaceutical Form: Lyophilisate and solvent for solution for injection INN or Proposed INN: Human Coagulation Factor VIII, Von Willebrand Factor Complex Current Sponsor code: WILATE Other descriptive name: HUMAN COAGULATION FACTOR VIII, VON WILLEBRAND FACTOR COMPLEX Concentration unit: IU international unit(s) Concentration type: equal Concentration number: 1000-
|
Primary Outcome(s)
|
Main Objective: The primary objective of this study is to determine the efficacy of Wilate in the prophylactic treatment of previously treated patients with type 3, type 2 (except 2N), or severe type 1 VWD
|
Secondary Objective: The secondary objectives of this study are to: - Assess the incremental IVR of Wilate for VWF:Ac and FVIII:C over time - Determine the pharmacokinetics (PK) of WIlate for VWF:Ac and FVIII:C in paediatric patients aged 6 to 16 years - Assess the safety and tolerability of Wilate - Determine Wilate consumption data
|
Timepoint(s) of evaluation of this end point: Baseline, 1, 2, 3, 6, 9 and 12 months
|
Primary end point(s): The primary endpoint of this study is to demonstrate that prophylactic treatment with WILATE lowers the patients’ total annualized bleeding rate (TABR) observed during on-demand treatment by more than 50%.
|
Secondary Outcome(s)
|
Secondary end point(s): - Spontaneous annualised bleeding rate (SABR) calculated in analogy with TABR - Incremental IVR of Wilate for VWF:Ac (VWF:RCo and VWF:GPIbm) and FVIII:C (OS and CHR) over time (at baseline and at 1, 2, 3, 6, 9, and 12 months of treatment). - For paediatric patients, baseline PK profile characteristics of VWF:Ac (VWF:RCo), and FVIII:C (OS and CHR) based on blood samples taken pre-dose and 1, 3, 9, 24, 48, and 72 hours after dosing - Safety and tolerability of Wilate by monitoring adverse events (AEs) throughout the study - Wilate consumption data (VWF/FVIII IU/kg per month per patient) for prophylaxis
|
Timepoint(s) of evaluation of this end point: Baseline, 1, 2, 3, 6, 9 and 12 months
|
Secondary ID(s)
|
WIL-31
|
011303
|
Source(s) of Monetary Support
|
Octapharma AG
|
Ethics review
|
Status:
Approval date:
Contact:
|
Results
|
Results available:
|
|
Date Posted:
|
|
Date Completed:
|
|
URL:
|
|
|
|