A Study of Vonicog Alfa (rVWF) in Children With Severe Von Willebrand Disease (vWD)

Conditions

• Von Willebrand Disease (VWD)

Eligibility Criteria

Sex

ALL

Age

N/A - 17 Years

Healthy Volunteers

Not accepted

Interventions

• Vonicog Alfa — BIOLOGICAL
  Vonicog Alfa administered by intravenous injection.
• ADVATE — BIOLOGICAL
  ADVATE administered by intravenous injection.

Study Details

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.

Key Dates

Start date

Nov 6, 2024

Status verified

May 2026

Primary completion

Apr 11, 2030

Completion

Apr 11, 2030

Study Design

Enrollment

24 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• 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).

Primary Outcome Measure

Annualized Bleeding Rate (ABR) for Spontaneous or Traumatic Bleeding Episodes as Assessed by Investigator During Prophylactic Treatment With Vonicog Alfa (rVWF) [ Time Frame: 12 months ]

Central Contacts

• Takeda Contact
  +1-877-825-3327
  [email protected]

Locations (11)

Find similar trials in Birmingham, AL

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