NCT05770219

Expanded Access Program of TAK-755 for Congenital Thrombotic Thrombocytopenic Purpura (cTTP)

Expanded Access Program: TAK-755 (rADAMTS13) for the Prophylaxis and Treatment of Severe Congenital or Hereditary Thrombotic Thrombocytopenic Purpura

• Status: Approved For Marketing
• Phase: —
• Study type: Expanded Access
• Enrollment: —
• Sponsor: Takeda · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

The expanded access program allows people to gain access to unlicensed treatment on compassionate grounds. TAK-755 also known as rADAMTS13, is a medicine that treats people born with severe congenital or hereditary thrombotic thrombocytopenic purpura (cTTP). This expanded access program enables continued access to those participants who have no other treatment options available for cTTP.

Detailed description

This is an expanded access program in which the drug being given is called TAK-755. This study will provide access to TAK-755 prior to marketing authorization for eligible participants with severe congenital or hereditary TTP who cannot adequately be treated via current standard of care and who cannot enter a clinical trial. All participants will receive TAK-755 as intravenous (IV) injection based on their weight as measured at each dosing visit. This is a multi-center, international program. Participants will continue treatment until benefit is no longer derived from the treatment (or treatment is no longer tolerable), sponsor decision, the participant chooses to discontinue the treatment, or TAK-755 becomes commercially available.

Conditions

• Thrombotic Thrombocytopenic Purpura (TTP)

Interventions

Type	Name	Description
DRUG	TAK-755	TAK-755 as IV injection at dose calculated based on the participant's body weight as measured at each dosing visit. For most participants, the final volume of infusion will be between 5 and 14 mL.

Timeline

• First posted: 2023-03-15
• Last updated: 2025-02-24