Post-Marketing Surveillance (All Case Surveillance) on Treatment With Alhemo® in Patients With Haemophilia A or Haemophilia B With Inhibitors

A Multi-centre, Open-label, Single-arm, Non-interventional Post-marketing Study to Investigate Safety and Clinical Parameters of Alhemo® Under Routine Clinical Practice in Japan

The purpose of the study is to investigate the safety and effectiveness of Alhemo® in all participants under real-world clinical practice in Japan. Participants will get Alhemo® as prescribed by the study doctor. The study will last for about 2 years.

Study Overview

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

• Study Type: Observational
• Enrollment (Estimated): 23

Contacts and Locations

• Study Contact: Name: Novo Nordisk; Phone Number: (+1) 866-867-7178; Email: clinicaltrials@novonordisk.com

Study Locations

• Japan
  • Tokyo
    • Toshima, Tokyo, Japan
      • Novo Nordisk Investigational Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Participants with haemophilia A or haemophilia B with inhibitors will be treated with commercially available concizumab 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 patient/LAR and the treating physician before and independently from the decision to include the patient in this study
• Male or female patients, regardless of age
• Diagnosis with HAwI/HBwI

Exclusion Criteria:

- Previous participation in this study. Participation is defined as having given informed consent in this study

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 with inhibitors will be treated with commercially available Alhemo (Concizumab) according to routine clinical practice at the discretion of the treating physician. Recruitment will be completed after 4.5 years from the launch of Concizumab. The observation period for each participant is 2 years. Total duration of this study is about 6.5 years.

Drug: Concizumab; Participants will be treated with commercially available Alhemo (Concizumab) according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available Concizumab 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.; Other Names: Alhemo

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of adverse reaction (AR)	Measured as count of ARs.	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)	Measured as count of SARs.	From baseline (week 0) to end of study (week 104)
Number of serious adverse event (SAE)	Measured as count of SAEs.	From baseline (week 0) to end of study (week 104)
Number of thromboembolic adverse event (AE)	Measured as count of thromboembolic AEs.	From baseline (week 0) to end of study (week 104)
Number of shock/anaphylaxis AE	Measured as count of shock/anaphylaxis AEs.	From baseline (week 0) to end of study (week 104)
Number of treated spontaneous and traumatic bleeding episodes	Measured as count of bleeding episodes.	From baseline (week 0) to end of study (week 104)
Number of treated spontaneous and traumatic target joint bleeding episodes	Measured as count of bleeding episodes.	From baseline (week 0) to end of study (week 104)
Number of all treatment requiring bleeding episode	Measured as count of 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 (Estimated): April 30, 2024
• Primary Completion (Estimated): April 30, 2030
• Study Completion (Estimated): April 30, 2030

Study Registration Dates

• First Submitted: February 13, 2024
• First Submitted That Met QC Criteria: February 26, 2024
• First Posted (Actual): February 29, 2024

Study Record Updates

• Last Update Posted (Actual): April 1, 2024
• Last Update Submitted That Met QC Criteria: March 29, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: NN7415-7557; U1111-1274-4740 (Other Identifier: World Health Organization (WHO))

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.