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A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

NCT04683003 · Status: ACTIVE_NOT_RECRUITING · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 77

Last updated 2026-04-02

No results posted yet for this study

Summary

Thrombotic thrombocytopenic purpura (or TTP for short) is a condition where blood clots form in small blood vessels throughout the body. The clots can limit or block the flow of oxygen-rich blood to the body's organs, such as the brain, kidneys, and heart. As a result, serious health problems can develop. The increased clotting that occurs in TTP uses up the cells that help the blood to clot, called platelets. With fewer platelets available in the blood, bleeding problems can also occur. People who have TTP may bleed underneath the skin forming purple bruises, or purpura. TTP also can cause anemia, a condition in which red blood cells break apart faster than the body can replace them, leading to fewer red blood cells than in normal.

TTP is caused by a lack of activity in the ADAMTS13 enzyme, a protein in the blood involved in controlling clotting of the blood. The ADAMTS13 enzyme breaks up another blood protein called von Willebrand factor that forms blood clots by clumping together with platelets. Some people are born with this condition, while others develop the condition during their life. Many people who are born with TTP experience frequent flare-ups that need to be treated right away. TAK-755 is a medicine that replaces ADAMTS13 and may prevent or control TTP flare-ups, called acute TTP events.

The main aim of the study is to check for side effects of long-term treatment with TAK-755. Treatment will be given in 2 ways:

1. TAK-755 treatment given either every week or every other week to prevent acute TTP events from happening (the "prophylactic" cohort).
2. TAK-755 treatment given to control an acute TTP event when it happens (the "on-demand" cohort).

Participants in the prophylactic cohort will receive treatment in the clinic or at home for up to approximately 3 years. They will visit the clinic at least every 12 weeks. Participants in the on-demand cohort will receive daily treatment for the acute TTP event until the flare-up has gotten better. They will have a follow-up visit at the clinic 4 weeks later.

Conditions

  • Thrombotic Thrombocytopenic Purpura (TTP)

Interventions

BIOLOGICAL

TAK-755

TAK-755 IV infusion

Sponsors & Collaborators

  • Takeda Development Center Americas, Inc.

    collaborator INDUSTRY

  • Shire

    collaborator INDUSTRY

  • Takeda

    lead INDUSTRY

Principal Investigators

  • Study Director · Takeda

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
0 Years
Max Age
70 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-04-14
Primary Completion
2027-03-16
Completion
2027-03-16
FDA Drug
Yes

Countries

  • United States
  • Austria
  • China
  • France
  • Germany
  • Italy
  • Japan
  • Poland
  • Spain
  • Switzerland
  • United Kingdom

Study Locations

More Related Trials

Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04683003 on ClinicalTrials.gov