Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors

Running, not enrolling · Phase 3

Conditions studied: Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors

In brief

This study will test how well a new medicine called concizumab works in the body of people with haemophilia A or B without inhibitors. The purpose is to show that concizumab can prevent bleeds in the body and is safe to use. Participants who usually only take medicine to treat bleeds (on-demand) will be placed in one of two groups. In one group participants will get study medicine from the start of the study. In the other group participants will continue with their normal medicine and get study medicine after 6 months. Which treatment the participant gets is decided by chance. Participants who usually take medicine to prevent bleeds (prophylaxis treatment) or who are already being treated with concizumab (study medicine) will receive the study medicine from the start of the study. Participants will have to inject themselves with the study medicine 1 time every day under the skin. This can be done at home. The study doctor will hand out the medicine in the form of a pen-injector. The pen-injector will contain the study medicine. The study will last for up to 8 years. The length of time the participant will be in the study depends on when they agreed to take part and when the medicine is available for purchase in their country (or 31 December 2027 at the latest). The time between visits will be approximately 4 weeks for the first 6 to 12 months depending on the group participants are in, and approximately 8 weeks for the rest of the study. If the participant attends extra visits due to the prescription medicine not being available for purchase in their country, these will be 14 weeks apart. Participants will be asked to record information in an electronic diary during the study and may also be asked to wear an activity tracker.

Key facts

Study ID

NCT04082429

Run by

Novo Nordisk A/S

People needed

156

Starts

2019-11-13

Expected to finish

2028-02-21

Last updated by the study team

2026-06-12

Who can join

Age: 12 and older. Sex: male. Healthy volunteers: not accepted.

You may qualify if…

• Informed consent obtained before any trial-related activities. Trial-related activities are any procedures that are carried out as part of the trial, including activities to determine suitability for the trial.
• Male aged 12 years or older at the time of signing informed consent.
• Congenital severe haemophilia A (FVIII below 1%) or B (FIX equal to or below 2%).

You may not qualify if…

• Known or suspected hypersensitivity to any constituent of the trial product or related products.
• Known inherited or acquired coagulation disorder other than congenital haemophilia.
• Presence of confirmed inhibitors 0.6 BU or greater at screening.
• History of thromboembolic disease (includes arterial and venous thrombosis including myocardial infarction, pulmonary embolism, cerebral infarction/thrombosis, deep vein thrombosis, other clinically significant thromboembolic events and peripheral artery occlusion). Current clinical signs of, or treatment for thromboembolic disease. Patients who in the judgement of the investigator are considered at high risk of thromboembolic events (thromboembolic risk factors could include, but are not limited to, hypercholesterolemia, diabetes mellitus, hypertension, obesity, smoking, family history of thromboembolic events, arteriosclerosis, other conditions associated with increased risk of thromboembolic events.)

Where it is running

• Center for Inherited Blood Disorders — Orange, California, United States
• Center for Blood Disorders Augusta University — Augusta, Georgia, United States
• Indiana Hemophilia-Thromb Ctr — Indianapolis, Indiana, United States
• University of Iowa_Iowa City — Iowa City, Iowa, United States
• Children's Hospital of Michigan — Detroit, Michigan, United States
• Michigan State University — Lansing, Michigan, United States
• Novant Hlth Vasc Ins Charlotte — Charlotte, North Carolina, United States
• M.S. Hershey Medical Center — Hershey, Pennsylvania, United States
• Vanderbilt University Medical Center_Nashville_0 — Nashville, Tennessee, United States
• University of Texas San Antonio — San Antonio, Texas, United States
• Versiti, CCBD — Milwaukee, Wisconsin, United States
• Haematology and Blood Bank Department — Algiers, Algeria
• CHU Constantine BEN BADIS/ Hematology department — Constantine, Algeria
• The Alfred — Melbourne, Victoria, Australia
• Royal Children's Hospital — Parkville, Victoria, Australia
• The Royal Children's Hospital — Parkville, Victoria, Australia
• Fiona Stanley Hospital - Haemophilia and Haemostasis Centre — Murdoch, Western Australia, Australia
• University Clinical Center of Republic Srpska (205) — Banja Luka, Bosnia and Herzegovina
• UMHAT Tsaritsa Yoanna - ISUL EAD, Pediatric clinical hematology and oncology — Sofia, Bulgaria
• UMHAT Sveta Marina EAD, Clinic of Pediatric Clinical Hematology and Oncology — Varna, Bulgaria
• Eastern Health Authority — St. John's, Newfoundland and Labrador, Canada
• Hamltn Hth Sci/McMstr Child Hosp — Hamilton, Ontario, Canada
• KBC Zagreb_Hematology — Zagreb, Croatia
• Copenhagen Center for Heamatology — Copenhagen, Denmark
• Children's Hospital Los Angeles - Endocrinology — Los Angeles, California, United States

Full record on ClinicalTrials.gov

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