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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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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

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