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

• Conditions: Von Willebrand Disease (VWD)

• Registration Number: NCT06173024
• Lead Sponsor: Takeda

Brief 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.

Detailed 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.

Basic Information
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Recruitment & Eligibility
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Study & Design

Study Timeline

• Study First Submitted: 2023-12-07
• Study First Submitted QC: 2023-12-07
• Study First Posted: 2023-12-15
• Status Verified: 2024-01
• Last Update Submitted: 2024-01-03
• Last Update Posted: 2024-01-05

Recruitment & Eligibility

• Status: AVAILABLE
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

1. Participant has completed the treatment period of the SHP677-304 study (at least 12 months on study treatment).
2. Participant had good clinical response to rVWF treatment.
3. Participant does not have access to any comparable or satisfactory alternative replacement 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 the post-trial access program and can provide written informed consent for themselves or the child to participate (with assent from a child when appropriate) before treatment).

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Exclusion Criteria

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

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Study & Design

• Study Type: EXPANDED_ACCESS
• Study Design: Not specified