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ACTIVE NOT RECRUITING
NCT04683003
PHASE3

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

Sponsor: Takeda

View on ClinicalTrials.gov

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.

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)

Key Details

Gender

All

Age Range

0 Years - 70 Years

Study Type

INTERVENTIONAL

Enrollment

77

Start Date

2021-04-14

Completion Date

2027-03-16

Last Updated

2026-04-02

Healthy Volunteers

No

Interventions

BIOLOGICAL

TAK-755

TAK-755 IV infusion

Locations (27)

Childrens Healthcare of Atlanta

Atlanta, Georgia, United States

Roswell Park Comprehensive Cancer Center

Buffalo, New York, United States

Duke University Medical Center

Durham, North Carolina, United States

Mid Ohio Heart Clinic Inc

Dublin, Ohio, United States

University of Oklahoma

Oklahoma City, Oklahoma, United States

AKH - Medizinische Universität Wien

Vienna, Austria

Beijing Children's Hospital

Beijing, Beijing Municipality, China

Peking Union Medical College Hospital

Beijing, Beijing Municipality, China

Tongji Hospital Affiliated to Tongji Medicine University

Wuhan, Hubei, China

The First Affiliated Hospital of Soochow University

Suzhou, Jiangsushe, China

Institute of Hematology and Hospital of Blood Disease

Tianjin, Tianjin Municipality, China

Hôpital Necker - Enfants Malades

Paris, Paris, France

CHU Saint Etienne - Hôpital Nord

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

Hôpital Saint-Antoine

Paris, France

Hôpital Robert Debré- Paris

Paris, France

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, Germany

Universitaetsklinikum Jena, Klinik fuerKinder-und Jugendmedizin

Jena, Germany

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

Bergamo, Hubei, Italy

Kyushu University Hospital

Fukuoka, Fukuoka, Japan

Hyogo College of Medicine Hospital

Nishinomiya, Hyōgo, Japan

Medical Hospital,Tokyo Medical and Dental University

Bunkyo City, Tokyo, Japan

Samodzielny Publiczny Dzieciecy Szpital Kliniczny

Warsaw, Poland

Instytut Hematologii i Transfuzjologii

Warsaw, Poland

Complejo Hospitalario Universitario A Coruña

A Coruña, La Coruña, Spain

Hospital de Cruces

Barakaldo, Vizcaya, Spain

Inselspital -Universitaetsspital Bern

Bern, Switzerland

University College London Hospitals

London, Greater London, United Kingdom