Status:
COMPLETED
Clinical Study to Investigate the Efficacy and Safety of Wilate During Prophylaxis in Previously Treated Patients With VWD
Lead Sponsor:
Octapharma
Conditions:
Von Willebrand Diseases
Eligibility:
All Genders
6+ years
Phase:
PHASE3
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 dat...
Eligibility Criteria
Inclusion
- 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
- 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
Key Trial Info
Start Date :
June 18 2020
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
April 23 2022
Estimated Enrollment :
43 Patients enrolled
Trial Details
Trial ID
NCT04052698
Start Date
June 18 2020
End Date
April 23 2022
Last Update
October 25 2023
Active Locations (14)
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1
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
2
Republican Research Center for Radiation Medicine and Human Ecology
Homyel, Belarus, 290
3
"Specialized Hospital for Active Treatment of Haematological Diseases" EAD, Sofia
Sofia, Bulgaria
4
Pediatric Clinic of Haematology and Oncology
Varna, Bulgaria, 9010