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

Active:	Yes
Cancer Type:	Unknown Primary	NCT ID:	NCT04683003
Trial Phases:	Phase III	Protocol IDs:	TAK-755-3002 (primary) NCI-2021-13466 2020-003348-10
Eligibility:	0 - 70 Years, Male and Female	Study Type:	Treatment
Study Sponsor:	Takeda
NCI Full Details:	http://clinicaltrials.gov/show/NCT04683003

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.

Objectives

This is a follow-up study to the Phase 3 pivotal study (281102 [NCT03393975]) and will be
comprised of two treatment cohorts (Prophylactic and On-demand) consisting of naïve and
non-naïve participants who were born with TTP, with a total duration of approximately 6
years. A maximum of approximately 77 participants will be enrolled in this study
(approximately 57 participants who have completed study 281102 [NCT03393975] [non-naïve
participants] and at least 20 naïve participants (participants who are naïve to TAK-755).
Participants from Expanded Access Programs, participants from study 281102 (NCT03393975)
who had an allergic reaction to standard of care treatment, and participants who
completed the Phase 1 study (281101 [NCT02216084]) but did not participate in Study
281102, will also be eligible for enrollment in this continuation study as naïve
participants.

TAK-755 is given slowly through a vein (intravenous infusion). Participants in the
prophylactic cohort will be able to opt for treatment in a home setting by caregiver or
self-infusion subject to conditions and to local regulatory approval.

Treatment Sites in Georgia

Winship Cancer Institute of Emory University
1365 Clifton Road NE
Building C
Atlanta, GA 30322
winshipcancer.emory.edu

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