A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
Public ClinicalTrials.gov record NCT04683003. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 3b, Prospective, Open-label, Multicenter, Single Treatment Arm, Continuation Study of the Safety and Efficacy of TAK-755 (rADAMTS13, Also Known as BAX 930/SHP655) in the Prophylactic and On-demand Treatment of Subjects With Severe Congenital Thrombotic Thrombocytopenic Purpura (cTTP; Upshaw-Schulman Syndrome, or Hereditary Thrombotic Thrombocytopenic Purpura)
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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.
Study identification
- NCT ID
- NCT04683003
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 77 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 0 Years to 70 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Apr 13, 2021
- Primary completion
- Mar 15, 2027
- Completion
- Mar 15, 2027
- Last update posted
- Apr 1, 2026
2021 – 2027
United States locations
- U.S. sites
- 5
- U.S. states
- 5
- U.S. cities
- 5
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Childrens Healthcare of Atlanta | Atlanta | Georgia | 30322 | — |
| Roswell Park Comprehensive Cancer Center | Buffalo | New York | 14203 | — |
| Duke University Medical Center | Durham | North Carolina | 27705 | — |
| Mid Ohio Heart Clinic Inc | Dublin | Ohio | 43017 | — |
| University of Oklahoma | Oklahoma City | Oklahoma | 73104 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 22 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04683003, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Apr 1, 2026 · Synced Sep 7, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04683003 live on ClinicalTrials.gov.