NCT05135559

A Research Study on How Well Concizumab Works for You if You Have Haemophilia A or B With or Without Inhibitors

Open-label Study Investigating Efficacy, Safety and Pharmacokinetics of Concizumab Prophylaxis in Children Below 12 Years With Haemophilia A or B With or Without Inhibitors

• Status: Active Not Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 153 (estimated)
• Sponsor: Novo Nordisk A/S · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

This study will test how well a new medicine called concizumab works for participants who have haemophilia A or B with or without inhibitors. The purpose is to show that concizumab can prevent bleeds and is safe to use. Participants will have to inject the study medicine every day under the skin with a pen-injector. The study will last for at least 2 years and up to about 4 years. The length of time the participant will be in the study depends on if the study medicine will be available for purchase in their country.

Conditions

• Haemophilia A and B With and Without Inhibitors

Interventions

Type	Name	Description
DRUG	Concizumab	Participants in Arm 1 will be assigned to concizumab prophylaxis starting with a loading dose on treatment day 0 followed by daily injections of an individual maintenance dose. Participants in Arm 2 will be assigned to concizumab prophylaxis with daily injections of an individual maintenance dose.

Timeline

• Start date: 2022-03-24
• Primary completion: 2026-04-21
• Completion: 2029-11-02
• First posted: 2021-11-26
• Last updated: 2025-12-30

Locations

91 sites across 24 countries: United States, Algeria, Bosnia and Herzegovina, Bulgaria, Canada, Estonia, France, Greece, India, Italy, Japan, Lebanon, Lithuania, Malaysia, North Macedonia, Norway, Poland, Romania, South Africa, Spain, Sweden, Thailand, Turkey (Türkiye), United Kingdom

Regulatory

• FDA-regulated drug study