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New enzyme replacement therapy aims to stop dangerous blood clots in rare disease

NCT ID NCT04683003

Ongoing Disease control Sponsor: Takeda Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a medicine called TAK-755 in people with a rare inherited blood disorder called congenital thrombotic thrombocytopenic purpura (cTTP). The condition causes dangerous blood clots and bleeding. TAK-755 replaces a missing enzyme to help prevent or treat these episodes. About 77 participants will receive the drug either regularly to prevent flare-ups or as needed during an acute event, and researchers will monitor side effects over about three years.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance

TAK-755 (a replacement for the missing ADAMTS13 enzyme)

What this could lead to

If successful, this could provide a safe, long-term treatment option to prevent or control TTP flare-ups, reducing hospitalizations and serious complications.

What could go wrong

This is an open-label study with no placebo group, so results may be less definitive. Long-term side effects are still being monitored, and the treatment requires regular infusions.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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Conditions

The condition(s) this trial relates to.

congenital thrombotic thrombocytopenic purpura thrombotic thrombocytopenic purpura

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

THROMBOTIC THROMBOCYTOPENIC PURPURA (TTP)

Contacts and locations

Locations

  • AKH - Medizinische Universität Wien

    Vienna, 1090, Austria

  • Azienda Socio Sanitaria Territoriale Papa Giovanni XXIII (Presidio Papa Giovanni XXIII)

    Bergamo, Hubei, 24127, Italy

  • Beijing Children's Hospital

    Beijing, Beijing Municipality, 100045, China

  • CHU Saint Etienne - Hôpital Nord

    Saint-Priest-en-Jarez Cedex, Pays de la Loire Region, 42270, France

  • Childrens Healthcare of Atlanta

    Atlanta, Georgia, 30322, United States

  • Complejo Hospitalario Universitario A Coruña

    A Coruña, La Coruña, 15006, Spain

  • Duke University Medical Center

    Durham, North Carolina, 27705, United States

  • Hospital de Cruces

    Barakaldo, Vizcaya, 48903, Spain

  • Hyogo College of Medicine Hospital

    Nishinomiya, Hyōgo, 663-8501, Japan

  • Hôpital Necker - Enfants Malades

    Paris, Paris, 75015, France

  • Hôpital Robert Debré- Paris

    Paris, 75935, France

  • Hôpital Saint-Antoine

    Paris, 75012, France

  • Inselspital -Universitaetsspital Bern

    Bern, 3010, Switzerland

  • Institute of Hematology and Hospital of Blood Disease

    Tianjin, Tianjin Municipality, 300021, China

  • Instytut Hematologii i Transfuzjologii

    Warsaw, 02-776, Poland

  • Kyushu University Hospital

    Fukuoka, Fukuoka, 812-8582, Japan

  • Medical Hospital,Tokyo Medical and Dental University

    Bunkyo City, Tokyo, 113-8519, Japan

  • Mid Ohio Heart Clinic Inc

    Dublin, Ohio, 43017, United States

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100730, China

  • Roswell Park Comprehensive Cancer Center

    Buffalo, New York, 14203, United States

  • Samodzielny Publiczny Dzieciecy Szpital Kliniczny

    Warsaw, 02-091, Poland

  • The First Affiliated Hospital of Soochow University

    Suzhou, Jiangsushe, 215006, China

  • Tongji Hospital Affiliated to Tongji Medicine University

    Wuhan, Hubei, 430030, China

  • Universitaetsklinikum Hamburg-Eppendorf

    Hamburg, 20246, Germany

  • Universitaetsklinikum Jena, Klinik fuerKinder-und Jugendmedizin

    Jena, 07747, Germany

  • University College London Hospitals

    London, Greater London, NW12PG, United Kingdom

  • University of Oklahoma

    Oklahoma City, Oklahoma, 73104, United States