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A study to determine the effectiveness, safety, and tolerability of the Recombinant Von Willebrand Factor administered with or without Advate for children diagnosed with Severe von Willebrand Disease who experience bleeding episodes and/or will undergo major, minor or oral surgery procedures.
EU CTIS ID: 2023-509769-18-00
What this study is testing
To evaluate the hemostatic efficacy and safety of vonicog alfa, with or without ADVATE, in the treatment and control of nonsurgical bleeding events in pediatric subjects (<18 years of age) diagnosed with severe, hereditary VWD.
- Therapeutic confirmatory (Phase III)
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Diagnosis of severe VWD (defined as VWF:RCo <20%): a. Type 1 (VWF:RCo <20 IU/dL); or b. Type 2A (VWF:RCo <20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (FVIII:C <10% and historically documented genetics), Type 2M; or c. Type 3 (VWF:Ag ≤3 IU/dL).
- Age 0 to <18 years at the time of screening.
- The subject has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
- If female of childbearing potential, subject presents with a negative serum pregnancy test.
- If applicable, subject agrees to employ adequate birth control measures for the duration of the study.
- Subject and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a prescreening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the subject's compliance with the study requirements.
You likely can't join if
- Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio >1.4)
- Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C.
- Diagnosis of renal disease, with a serum creatinine level ≥2.5 mg/dL.
- Immunomodulatory drug treatment other than anti-retroviral chemotherapy (eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day (excluding topical treatment [eg, ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate).
- If female, subject is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained.
- Subject has participated in another clinical study involving an IP, other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study.
See the full eligibility criteria
Who can join
- Diagnosis of severe VWD (defined as VWF:RCo <20%): a. Type 1 (VWF:RCo <20 IU/dL); or b. Type 2A (VWF:RCo <20 IU/dL), Type 2B (as diagnosed by genotype), Type 2N (FVIII:C <10% and historically documented genetics), Type 2M; or c. Type 3 (VWF:Ag ≤3 IU/dL).
- Age 0 to <18 years at the time of screening.
- The subject has provided assent (if appropriate) and legally authorized representative(s) has provided informed consent.
- If female of childbearing potential, subject presents with a negative serum pregnancy test.
- If applicable, subject agrees to employ adequate birth control measures for the duration of the study.
- Subject and/or the legally authorized representative are willing and able to comply with the requirements of the protocol, which should also be confirmed based on a prescreening evaluation held between the Investigator and the Sponsor, to ensure no eminent risk is present that could challenge the subject's compliance with the study requirements.
- Additional inclusion criteria for previously treated subjects as well as for subjects undergoing surgery: 1. Unable to tolerate, inadequately responsive to not a good candidate for 1-deamino-8-D-arginine vasopressin (DDAVP). Examples of subjects who are not good candidates for DDAVP include subjects with type 2B or type 3 VWD. 2. The subject has had a minimum of 1 documented bleed requiring VWF coagulation factor replacement therapy (ie, treatment with a VWF product) during the previous 12 months prior to enrollment and overall historically 3 or more exposure days (EDs) to VWF replacement therapy.
- Additional inclusion criterion for previously untreated subjects: The subject has not received prior VWF coagulation factor replacement therapy.
What rules you out
- Diagnosis of pseudo-VWD or another hereditary or acquired coagulation disorder (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio >1.4)
- Diagnosis of significant liver disease, as evidenced by, but not limited to, any of the following: serum alanine aminotransferase 5 times the upper limit of normal; hypoalbuminemia; portal vein hypertension (eg, presence of otherwise unexplained splenomegaly, history of esophageal varices) or liver cirrhosis classified as Child B or C.
- Diagnosis of renal disease, with a serum creatinine level ≥2.5 mg/dL.
- Immunomodulatory drug treatment other than anti-retroviral chemotherapy (eg, α-interferon, or corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day (excluding topical treatment [eg, ointments, nasal sprays]), within 30 days prior to signing the informed consent (or assent, if appropriate).
- If female, subject is pregnant or lactating at the time informed consent (or assent, if appropriate) is obtained.
- Subject has participated in another clinical study involving an IP, other than vonicog alfa with or without ADVATE, or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP other than vonicog alfa or investigational device during the course of this study.
- Subject's legal representative is a family member or employee of the Investigator.
- History or presence of a VWF inhibitor at Screening.
- History or presence of a FVIII inhibitor with a titer ≥0.4 Bethesda units (BU) (by Nijmegen assay) or ≥0.6 BU (by Bethesda assay.)
- Documented history of a VWF:RCo half-life <6 hours.
- Known hypersensitivity to any of the components of the study drug, such as mouse or hamster proteins.
- Medical history of immunological disorders, excluding seasonal allergic rhinitis/ conjunctivitis/ asthma, food allergies, or animal allergies
- Medical history of a thromboembolic event.
- Human immunodeficiency virus positive, with an absolute CD4 count <200/mm3.
- In the judgment of the Investigator, the subject has another clinically significant concomitant disease (eg, uncontrolled hypertension, cancer) that may pose additional risks for the subject.
The study team makes the final eligibility decision.
Where it's taking place
- Turkey
- Russian Federation
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Turkey; Russian Federation; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.