Post Trial Access Program of TAK-577 for Von Willebrand Disease (VWD)

Last updated: September 2, 2025
Sponsor: Takeda
Overall Status: Planned

Phase

N/A

Condition

N/A

Treatment

TAK-577

Clinical Study ID

NCT06173024
TAK-577 PTA
  • All Genders

Study Summary

The post-trial access program allows eligible participants to gain access to unlicensed treatment on compassionate grounds. Recombinant von Willebrand factor (rVWF) also known as TAK-577, is a medicine to help treat Von Willebrand Disease (VWD). This post-trial access program enables continued access to children and adults who are benefitting from treatment on study SHP677-304 (NCT03879135) study.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  1. Participant has completed the treatment period of the SHP677-304 study (at least 12months on study treatment).

  2. Participant had good clinical response to rVWF treatment.

  3. Participant does not have access to any comparable or satisfactory alternativereplacement therapy available at country level.

  4. Participant will be/has been negatively impacted by discontinuation of rVWF.

  5. Participant and/or a parent(s)/legal guardian is informed of the nature of thepost-trial access program and can provide written informed consent for themselves orthe child to participate (with assent from a child when appropriate) beforetreatment).

Exclusion

Exclusion Criteria:

  1. Participants with known hypersensitivity/intolerance to the study drug will not be eligible for this study

Study Design

Treatment Group(s): 1
Primary Treatment: TAK-577
Phase:
Study Start date:
Estimated Completion Date:

Study Description

This is a post-trial access program in which the drug being given is called TAK-577. This study will provide access to TAK-577 before marketing authorization for eligible participants with severe VWD who are benefitting from treatment on study SHP677-304 and cannot adequately be treated via the current standard of care and cannot enter a clinical trial.

All participants will receive TAK-577 as a sequential intravenous infusion based on their weight.

This is a multi-center, international program. Participants will continue treatment until a benefit is no longer derived from the treatment (or treatment is no longer tolerable), the sponsor decision to end the program, the participant chooses to discontinue the treatment, or TAK-577 becomes commercially available for children.