Clinical trial NCT06831734: Post-marketing Surveillance (Special Use-results Surveillance) on Treatment With Alhemo
Official title: 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
Study type: OBSERVATIONAL
Overall status: NOT_YET_RECRUITING
Conditions: Haemophilia A, Haemophilia B
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2025-02-18
Enrollment: 30
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
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.

Brief summary: 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Number of adverse reaction (AR)
timeFrame: From baseline (week 0) to end of study (week 104)
Secondary outcomes:
measure: Number of serious adverse reaction (SAR)
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of serious adverse event (SAE)
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of thromboembolic adverse event (AE)
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of shock/anaphylaxis adverse event (AE)
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of treated spontaneous and traumatic bleeding episodes
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of treated spontaneous and traumatic target joint bleeding episodes
timeFrame: From baseline (week 0) to end of study (week 104)
measure: Number of all treatment requiring bleeding episodes
timeFrame: From baseline (week 0) to end of study (week 104)
Other outcomes:
Locations:
city: Chiba
country: Japan
facility: Chiba university hospital_Pediatrics
city: Kanagawa
country: Japan
facility: St. Marianna University School of Medicine Hospital_Pediatrics
city: Kawagoe
country: Japan
facility: Saitama Medical Univ. Hospital Saitama medical center_Department of Transfusion Medicine and Cell Therapy
city: Okinawa
country: Japan
facility: Nanbu Medical Center & Children's Medical Center
city: Saitama
country: Japan
facility: Saitama Medical Univ. Hospital Saitama medical center_Department of Transfusion Medicine and Cell Therapy
city: Tokyo
country: Japan
facility: National Center for Child Health and Development_Hematology
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency (dept. 2834)
role: STUDY_DIRECTOR
References: