Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo

Special Use-results Surveillance on Use of Alhemo in Patients With Haemophilia A or Haemophilia B Without Inhibitors A Multi-centre, Open-label, Non-interventional Post-marketing Study to Investigate the Long-term Safety and Clinical Parameters of Treatment With Alhemo in Patients With Haemophilia A or Haemophilia B Without Inhibitors in Routine Clinical Practice Conditions in Japan

The purpose of the study is to investigate the safety and effectiveness of Alhemo in participants under real-world clinical practice in Japan. Total duration of this study is about 6 years. Participants enrolment will be completed in the first 4 years. The observation period of this study will last for about 2 years for each enrolled participant.

Study Overview

• Status: Enrolling by invitation
• Conditions: Haemophilia A, Haemophilia B
• Intervention / Treatment: Drug: Concizumab

• Study Type: Observational
• Enrollment (Estimated): 30

Contacts and Locations

Study Locations

• Japan
  • Chiba, Japan, 260-8677
    • Chiba university hospital_Pediatrics
  • Kanagawa, Japan, 216-8511
    • St. Marianna University School of Medicine Hospital_Pediatrics
  • Kawagoe, Japan
    • Saitama Medical Univ. Hospital Saitama medical center_Department of Transfusion Medicine and Cell Therapy
  • Nahano, Japan, 380-8582
    • Nagano red cross hospital_Pediatrics
  • Okinawa, Japan, 901-1193
    • Nanbu Medical Center & Children's Medical Center
  • Osaka, Japan, 573-1191
    • Kansai Medical University Hospital_Pediatrics
  • Saitama, Japan, 350-8550
    • Saitama Medical Univ. Hospital Saitama medical center_Department of Transfusion Medicine and Cell Therapy
  • Shimane, Japan, 690-8506
    • Matsue red cross hospital_Pediatrics
  • Tokyo, Japan, 157-8535
    • National Center for Child Health and Development_Hematology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: N/A

• Sampling Method: Non-Probability Sample

Study Population

Participants with haemophilia A or haemophilia B without inhibitors will be be treated with commercially available Alhemo according to routine clinical practice at the discretion of the treating physician.

Description

Inclusion Criteria:

• Signed consent obtained before any study-related activities (study-related activities are any procedure related to recording of data according to the protocol).
• The decision to initiate treatment with commercially available Alhemo has been made by the participant/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the participant in this study.
• Male or female, age above or equal to 12 years at the time of signing informed consent.
• Diagnosis with Haemophilia A (HA) or Haemophilia B (HB).
• Participant who has never been exposed to concizumab or who starts treatment with concizumab within the past 12 weeks at registration.

Exclusion Criteria:

• Previous participation in this study. Participation is defined as having given informed consent in this study.
• Treatment with any investigational drug within 30 days prior to baseline (the starting date of Alhemo treatment).
• Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
• A history of hypersensitivity to any ingredients of Alhemo.

Study Plan

How is the study designed?

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	Intervention / Treatment
Concizumab; Participants with haemophilia A or haemophilia B without inhibitors will be be treated with commercially available Alhemo according to routine clinical practice at the discretion of the treating physician.	Drug: Concizumab; Participants will be treated with commercially available Alhemo according to routine clinical practice at the discretion of the treating physician. Administration will be according to the approved product labelling. The decision to treat a participant with Alhemo is made at the physician's discretion before and independently from this study.; Other Names: Alhemo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse reaction (AR)	Count of adverse reaction.	From baseline (week 0) to end of study (week 104)

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of serious adverse reaction (SAR)	Count of serious adverse reaction.	From baseline (week 0) to end of study (week 104)
Number of serious adverse event (SAE)	Count of serious adverse event.	From baseline (week 0) to end of study (week 104)
Number of thromboembolic adverse event (AE)	Count of thromboembolic adverse event.	From baseline (week 0) to end of study (week 104)
Number of shock/anaphylaxis adverse event (AE)	Count of shock/anaphylaxis adverse event.	From baseline (week 0) to end of study (week 104)
Number of treated spontaneous and traumatic bleeding episodes	Count of treated spontaneous and traumatic bleeding episodes.	From baseline (week 0) to end of study (week 104)
Number of treated spontaneous and traumatic target joint bleeding episodes	Count of treated spontaneous and traumatic target joint bleeding episodes.	From baseline (week 0) to end of study (week 104)
Number of all treatment requiring bleeding episodes	Count of all treatment requiring bleeding episodes.	From baseline (week 0) to end of study (week 104)

Collaborators and Investigators

• Sponsor: Novo Nordisk A/S
• Investigators: Study Director: Clinical Transparency (dept. 2834), Novo Nordisk A/S

Study record dates

Study Major Dates

• Study Start (Actual): May 2, 2025
• Primary Completion (Estimated): January 31, 2031
• Study Completion (Estimated): January 31, 2031

Study Registration Dates

• First Submitted: February 12, 2025
• First Submitted That Met QC Criteria: February 12, 2025
• First Posted (Actual): February 18, 2025

Study Record Updates

• Last Update Posted (Estimated): August 29, 2025
• Last Update Submitted That Met QC Criteria: August 28, 2025
• Last Verified: August 1, 2025

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Genetic Diseases, Inborn; Hematologic Diseases; Blood Coagulation Disorders; Hemorrhagic Disorders; Genetic Diseases, X-Linked; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Hemic and Lymphatic Diseases; Hemophilia A; Hemophilia B; concizumab
• Other Study ID Numbers: NN7415-7690; U1111-1286-2960 (Other Identifier: Universal trial number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: According to the Novo Nordisk disclosure commitment on novonordisk-trials.com

Drug and device information, study documents

• Studies a U.S. FDA-regulated device product: No