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A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura
NCT04683003 · View on ClinicalTrials.gov ↗
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
Conditions Studied
Interventions
- BIOLOGICAL TAK-755
Study Locations (20)
Other
- AKH - Medizinische Universität Wien — Vienna
- Hôpital Saint-Antoine — Paris
- Hôpital Robert Debré- Paris — Paris
- Universitaetsklinikum Hamburg-Eppendorf — Hamburg
- Universitaetsklinikum Jena, Klinik fuerKinder-und Jugendmedizin — Jena
Beijing Municipality
- Beijing Children's Hospital — Beijing
- Peking Union Medical College Hospital — Beijing
Hubei
- Tongji Hospital Affiliated to Tongji Medicine University — Wuhan
- Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII) — Bergamo
Georgia
- Childrens Healthcare of Atlanta — Atlanta
New York
- Roswell Park Comprehensive Cancer Center — Buffalo
North Carolina
- Duke University Medical Center — Durham
Ohio
- Mid Ohio Heart Clinic Inc — Dublin
Oklahoma
- University of Oklahoma — Oklahoma City
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 77 participants |
| Start Date | 2021-04-14 |
| Est. Completion | 2027-03-16 |
| Phase | Phase 3 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT04683003
The ClinicalTrials.gov registry entry for NCT04683003 describes a study currently listed as recruiting. It is categorized as Phase 3, which is the standard way researchers label where a study sits along the investigational pathway from early safety work through later efficacy and post-marketing evaluation. The registered enrollment target is 77 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is Takeda, which has 387 total studies on file at ClinicalTrials.gov, and sponsors are the parties responsible for study design, oversight, and regulatory filings.
The record links to 1 condition, with Thrombotic Thrombocytopenic Purpura (TTP) appearing as the primary indexed condition, and to 1 intervention — of which TAK-755 is the first listed. Interventions can include drugs, devices, procedures, behavioral programs, or observational arms, and each is tracked as a separate registry field so that downstream queries can filter accurately. When a trial lists multiple interventions, it usually reflects a multi-arm design or a comparison protocol rather than a single treatment being tested in isolation. The brief summary published in the registry is the clearest source of protocol intent and should be read before drawing conclusions from any sidebar tags.
Geographic footprint matters for practical reasons: NCT04683003 reports 20 study locations spanning 14 distinct geographic areas — top geographies include Other, Beijing Municipality, Hubei. A larger site network tends to correlate with broader recruitment capacity, but it does not imply anything about study quality, and site-level enrollment status can diverge from the overall registry status shown above. Every data point on this page comes from the public ClinicalTrials.gov dataset and is reproduced here for reference only; it is not a medical recommendation, an endorsement of the sponsor, or an invitation to enroll. Verify current status, eligibility criteria, and contact details directly at ClinicalTrials.gov, and discuss any participation decision with your own healthcare provider.
Frequently Asked Questions
What is clinical trial NCT04683003 about?
NCT04683003 is a clinical study titled "A Study of TAK-755 in Participants With Congenital Thrombotic Thrombocytopenic Purpura". 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 de...
What is the current status of trial NCT04683003?
This trial is currently recruiting. It is a Phase 3 study. The enrollment target is 77 participants. The study started on 2021-04-14. Estimated completion is 2027-03-16.
What conditions does trial NCT04683003 study?
This clinical trial studies the following conditions: Thrombotic Thrombocytopenic Purpura (TTP). These conditions were identified from the trial registry and reflect the primary focus areas of the research.
What interventions are being tested in trial NCT04683003?
The interventions under investigation include: TAK-755 (BIOLOGICAL). Each intervention is being evaluated for safety and efficacy as part of this clinical study.
Who is sponsoring clinical trial NCT04683003?
This trial is sponsored by Takeda, which has 387 total clinical trials registered on ClinicalTrials.gov. The sponsor is responsible for the study's design, funding, and regulatory compliance.
Where is trial NCT04683003 being conducted?
This trial has 20 study locations across Georgia, New York, North Carolina, Ohio, Oklahoma. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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