A Phase 3, Prospective, Open-label, Uncontrolled, Multicenter Study on Efficacy and Safety of Prophylaxis With Vonicog Alfa (rVWF) in Children Diagnosed With Severe Von Willebrand Disease

Status: Recruiting
Location: See all (15) locations...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

The main aim of the study is to evaluate the effectiveness of prophylaxis with vonicog alfa (recombinant von Willebrand factor \[rVWF\]) in children. This study will enroll those participants who have been previously treated with VWF product or with a plasma-derived VWF (pdVWF) product. In this study, participants will be treated with vonicog alfa (rVWF) for 12 months. During the study, participants will visit the study clinic 5 times after treatment initiation.

Eligibility
Participation Requirements
Sex: All
Maximum Age: 17
Healthy Volunteers: f
View:

• The participant has a documented diagnosis of severe VWD (baseline von Willebrand factor ristocetin cofactor activity \[VWF:RCo\] \<20 international units per deciliter \[IU/dL\]) 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 participant's history or at screening.

• The participant is \<18 years of age at the time of screening.

• Prescreening treatment requirements:

‣ The participant has been receiving OD therapy with VWF products for at least 12 months (for participants \>=2 years of age) prior to screening, has experienced at least 1 VWF-treated bleeding event during (excluding menorrhagia/heavy menstrual bleeding \[HMB\], as applicable) in the last 12 months, and prophylactic treatment is recommended by the investigator (Prior OD participants); or

⁃ The participant has been receiving prophylactic treatment with pdVWF products for at least 12 months prior to screening (for participants \>=2 years of age) and switching to prophylaxis with vonicog alfa (rVWF) is recommended by the investigator (Switch participants).

⁃ For participants \<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 participants \<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 participants \>=2 years of age, the participant has available records that reliably evaluate type, frequency, severity, and treatment of BEs for at least 12 months preceding enrollment. For participants \<2 years of age, the participant has available records that reliably evaluate type, frequency, severity and treatment of BEs for at least 6 months preceding enrollment.

• If \>=12 years old at the time of screening, the participant has a body mass index (BMI) \>=15 but \<40 kilogram per square meter (kg/m\^2). If \>=2 to \<12 years old at the time of screening, the participant has a BMI of \>=5th and \<95th percentile (per Centers for Disease Control and Prevention \[CDC\] clinical charts). For younger participants 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 participant has a body weight of \>=5th and \<95th percentile based on gender (for clinical charts provided by CDC, refer to: https://www.cdc.gov/growthcharts/clinical\_charts.htm).

• Female participants of childbearing potential (that is, 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 for the duration of their participation in the study.

• The participant has voluntarily provided assent (if appropriate) and the legally authorized representative(s) has provided informed consent.

• The participant 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 participant's compliance with the study requirements.

Locations
United States
Alabama
University of Alabama at Birmingham
RECRUITING
Birmingham
Iowa
University of Iowa Hospitals & Clinics PARENT
RECRUITING
Iowa City
Illinois
Bleeding and Clotting Disorders Institute
RECRUITING
Peoria
Indiana
Riley Hospital for Children Indiana University Health
RECRUITING
Indianapolis
Michigan
Childrens Hospital of Michigan
RECRUITING
Detroit
Minnesota
Children's Health Care d/b/a Children's Minnesota
RECRUITING
Minneapolis
New Jersey
Rutgers - Robert Wood Johnson Medical School
RECRUITING
New Brunswick
New York
New York - Presbyterian/Weill Cornell Medical Center
RECRUITING
New York
South Carolina
Medical University of South Carolina (MUSC)
RECRUITING
Charleston
Other Locations
France
Hemostase Clinique - Institut Cœur-Poumons (4eme étage aile est) Bureau 419
RECRUITING
Lille
Hopital Edouard Herriot - CHU Lyon
RECRUITING
Lyon
Ireland
Children's Health Ireland
RECRUITING
Dublin
Italy
Azienda Ospedaliera Pediatrica Santobono Pausillipon
RECRUITING
Napoli
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
RECRUITING
Turin (torino)
Japan
Nara Medical University Hospital
RECRUITING
Kashihara
Contact Information
Primary
Takeda Contact
medinfoUS@takeda.com
+1-877-825-3327
Time Frame
Start Date: 2024-11-06
Estimated Completion Date: 2030-04-11
Participants
Target number of participants: 24
Treatments
Experimental: Cohort 1: Participants With Age >=12 to <18 years
Participants with age greater than or equal to (\>=) 12 to less than (\<) 18 years who have received on-demand (OD) therapy or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 international units per kilogram (IU/kg) vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Experimental: Cohort 2: Participants With Age >=6 to <12 years
Participants with age \>=6 to \<12 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Experimental: Cohort 3: Participants With Age <6 years
Participants with age \<6 years who have received OD therapy of VWF product or prophylactic treatment with a pdVWF product will receive vonicog alfa (rVWF) with an initial dose selected within the range of 40 to 60 IU/kg vonicog alfa (rVWF), intravenous infusions, twice-weekly for 12 months. Participants may receive vonicog alfa (rVWF) with or without ADVATE intravenous infusions, when indicated (as deemed necessary for breakthrough bleeding episode treatment and perioperative bleeding management).
Sponsors
Leads: Takeda

This content was sourced from clinicaltrials.gov

Similar Clinical Trials