Authorised Therapeutic confirmatory (Phase III) severe von Willebrand Disease

A preventive study of bleeding episodes for children with severe von Willebrand disease (VWD).

EU CTIS ID: 2023-509877-22-00

What this study is testing

To prospectively evaluate the efficacy of vonicog alfa (rVWF) prophylaxis in children based on the annualized bleeding rate (ABR) with intra-patient control (on-study compared to historical) for all bleeding episodes classified by the investigator as either spontaneous or traumatic while on prophylactic treatment with vonicog alfa (rVWF).

  • 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

  • The subject has a documented diagnosis of severe VWD (baseline VWF:RCo <20 IU/dL) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1, type 2 (2A, 2B, 2M, 2N), or type 3. Diagnosis is confirmed, as applicable, by genetic testing and/or by multimer analysis, which may be documented in patient's history or tested at screening.
  • The subject is <18 years of age at the time of screening.
  • Prescreening treatment requirements: a. The subject has been receiving On Demand (OD) therapy with VWF products for at least 12 months (for subjects ≥2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event (excluding menorrhagia/heavy menstrual bleeding, as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD subjects); or b. The subject has been receiving prophylactic treatment with plasma derived (pd)VWF products for at least 12 months prior to screening (for subjects ≥2 years of age) and switching to prophylaxis with vonicog alfa (rVWF) is recommended by the investigator (Switch subjects), c. For subjects <2 years of age, the required duration for prior OD therapy with VWF products or for prior prophylactic treatment with pdVWF products is at least 6 months. Prior OD subjects <2 years of age should have experienced at least 1 VWF-treated bleeding event during the last 6 months based on medical records and be recommended to receive prophylactic treatment by the investigator.
  • For subjects ≥2 years of age, the subject has available records that reliably evaluate type, frequency, severity, and treatment of bleeding episodes for at least 12 months preceding enrollment. For subjects <2 years of age, the subject has available records that reliably evaluate type, frequency, severity and treatment of bleeding episodes for at least 6 months preceding enrollment.
  • If ≥12 years old at the time of screening, the subject has a body mass index (BMI) ≥15 but <40 kg/m2. If ≥2 to <12 years old at the time of screening, the subject has a BMI of ≥5th and <95th percentile (per Centers for Disease Control and Prevention [CDC] clinical charts). For younger subjects who are <2 years old, the ""weight-for-age"" clinical charts (5th to 95th percentile) provided by the CDC should be utilized to ensure the subject has a body weight of ≥5th and <95th percentiles based on gender (for clinical charts provided by CDC, refer to: https://www.cdc.gov/growthcharts/clinical_charts.htm).
  • Female subjects of childbearing potential (ie, had onset of menses/reached puberty) must have a negative blood/urine pregnancy test result at screening and agree to employ highly effective birth control measures (as defined in the protocol) for the duration of their participation in the study.

You likely can't join if

  • The subject has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio less than 1.4).
  • The subject has a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
  • The subject has a history or presence of a VWF inhibitor at screening.
  • The subject is member of the study team or in a dependent relationship with one of the study team members, which includes close relatives (ie, children, partner/spouse, siblings, and parents) as well as employees.
  • The subject has a history or presence of an FVIII inhibitor with a titer ≥0.6 Bethesda units (BU) (by the Nijmegen-modified Bethesda assay) or ≥0.6 BU (by the Bethesda assay).
  • The subject has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
See the full eligibility criteria
Who can join
  • The subject has a documented diagnosis of severe VWD (baseline VWF:RCo <20 IU/dL) with a history of replacement therapy with VWF concentrate required to control bleeding and a diagnosis of VWD type 1, type 2 (2A, 2B, 2M, 2N), or type 3. Diagnosis is confirmed, as applicable, by genetic testing and/or by multimer analysis, which may be documented in patient's history or tested at screening.
  • The subject is <18 years of age at the time of screening.
  • Prescreening treatment requirements: a. The subject has been receiving On Demand (OD) therapy with VWF products for at least 12 months (for subjects ≥2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event (excluding menorrhagia/heavy menstrual bleeding, as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD subjects); or b. The subject has been receiving prophylactic treatment with plasma derived (pd)VWF products for at least 12 months prior to screening (for subjects ≥2 years of age) and switching to prophylaxis with vonicog alfa (rVWF) is recommended by the investigator (Switch subjects), c. For subjects <2 years of age, the required duration for prior OD therapy with VWF products or for prior prophylactic treatment with pdVWF products is at least 6 months. Prior OD subjects <2 years of age should have experienced at least 1 VWF-treated bleeding event during the last 6 months based on medical records and be recommended to receive prophylactic treatment by the investigator.
  • For subjects ≥2 years of age, the subject has available records that reliably evaluate type, frequency, severity, and treatment of bleeding episodes for at least 12 months preceding enrollment. For subjects <2 years of age, the subject has available records that reliably evaluate type, frequency, severity and treatment of bleeding episodes for at least 6 months preceding enrollment.
  • If ≥12 years old at the time of screening, the subject has a body mass index (BMI) ≥15 but <40 kg/m2. If ≥2 to <12 years old at the time of screening, the subject has a BMI of ≥5th and <95th percentile (per Centers for Disease Control and Prevention [CDC] clinical charts). For younger subjects who are <2 years old, the ""weight-for-age"" clinical charts (5th to 95th percentile) provided by the CDC should be utilized to ensure the subject has a body weight of ≥5th and <95th percentiles based on gender (for clinical charts provided by CDC, refer to: https://www.cdc.gov/growthcharts/clinical_charts.htm).
  • Female subjects of childbearing potential (ie, had onset of menses/reached puberty) must have a negative blood/urine pregnancy test result at screening and agree to employ highly effective birth control measures (as defined in the protocol) for the duration of their participation in the study.
  • The subject has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.
  • The subject and/or legally authorized representative is 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.
What rules you out
  • The subject has been diagnosed with pseudo VWD or another hereditary or acquired coagulation disorder other than VWD (eg, qualitative and quantitative platelet disorders or elevated prothrombin time/international normalized ratio less than 1.4).
  • The subject has a mental condition rendering him/her unable to understand the nature, scope, and possible consequences of the study and/or evidence of an uncooperative attitude.
  • The subject has a history or presence of a VWF inhibitor at screening.
  • The subject is member of the study team or in a dependent relationship with one of the study team members, which includes close relatives (ie, children, partner/spouse, siblings, and parents) as well as employees.
  • The subject has a history or presence of an FVIII inhibitor with a titer ≥0.6 Bethesda units (BU) (by the Nijmegen-modified Bethesda assay) or ≥0.6 BU (by the Bethesda assay).
  • The subject has a known hypersensitivity to any of the components of the study drugs, such as mouse or hamster proteins.
  • The subject has a medical history of immunological disorders, excluding seasonal allergic rhinitis/conjunctivitis, mild asthma, food allergies, or animal allergies.
  • The subject has a medical history of a thromboembolic event.
  • The subject is HIV-positive with an absolute helper T cell (CD4) count less than 200/mm3.
  • The subject has been diagnosed with significant liver disease per the investigator's medical assessment of the subject's current condition or medical history or as evidenced by, but not limited to, any of the following: serum alanine aminotransferase (ALT) greater than 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-Pugh class B or C.
  • The subject has been diagnosed with renal disease, with a serum creatinine level ≥2.5 mg/dL.
  • The subject has a platelet count <100,000/mL at screening (because patients with type 2B VWD are considered eligible for this study, for patients with type 2B VWD, platelet count[s] at screening will be evaluated in consultation with the sponsor, taking into consideration historical trends in platelet counts and the investigator's medical assessment of the patient's condition).
  • The subject has been treated with an immunomodulatory drug, excluding topical treatment (eg, ointments, nasal sprays), within 30 days prior to signing the informed consent (or assent, if appropriate).
  • The subject is pregnant or lactating at the time of enrollment.
  • The subject has cervical or uterine conditions causing menorrhagia or metrorrhagia (including infection, dysplasia).
  • The subject has participated in another clinical study involving another IP or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device during the course of this study.
  • The subject has not received OD or prophylactic treatment with a VWF product prior to this study.
  • The subject has a progressive fatal disease and/or life expectancy of less than 15 months.
  • The subject is unable to complete screening procedures and/or comply with the requirements of the protocol in the opinion of the investigator, based on the joint prescreening evaluation held between the investigator and the sponsor.

The study team makes the final eligibility decision.

Where it's taking place

  • Japan
  • 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 Japan; United States. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

ObesityType 2 diabetesType 1 diabetesPrediabetesWeight lossMetabolic syndromeHigh cholesterolHigh triglyceridesHigh blood pressurePCOSHypothyroidismThyroid diseaseGoutFatty liver disease (NAFLD/NASH)Heart failureCoronary artery disease

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.