Haemophilia A
Conditions
Brief summary
The purpose of this study is to assess the safety and effectiveness of Esperoct® for long-term routine use in patients with Haemophilia A. Participants will get Esperoct® as prescribed by their doctor. The study will last for about 2 years for each participant.
Interventions
Patients will be treated with commercially available Esperoct® according to routine clinical practice at the discretion of the treating physician. The decision to initiate treatment with commercially available Esperoct® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study.
Sponsors
Novo Nordisk A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
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 Esperoct® has been made by the patient/Legally Acceptable Representative (LAR) and the treating physician before and independently from the decision to include the patient in this study. * Diagnosis of haemophilia A in males or females, no age limitation. * New patients who have not previously been exposed to Esperoct®.
Exclusion criteria
* Previous participation in this study. Participation is defined as having given informed consent in this study. * Known or suspected hypersensitivity to study product or related products. * Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of adverse reactions (ARs) reported during the observation period | From baseline (week 0) to end of study (week 104) | Count |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of serious adverse events (SAEs) reported during the observation period | From baseline (week 0) to end of study (week 104) | Count |
| Number of serious adverse reactions (SARs) reported during the observation period | From baseline (week 0) to end of study (week 104) | Count |
| Number of patients who have confirmed inhibitory antibodies against FVIII during the observation period | From baseline (week 0) to end of study (week 104) | Count |
| Number of bleeding episodes requiring treatment for patients using Esperoct® during the observation period assessed by annual bleeding rate (ABR) | From baseline (week 0) to end of study (week 104) | Count |
| Evaluation of the haemostatic response of Esperoct® measured as number of successes for treatment requiring bleeds | From baseline (week 0) to end of study (week 104) | Count, assessed based on a four-point scale (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure |
| Evaluation of the haemostatic response of Esperoct® measured as number of successes in treatment of bleeds in perioperative management during surgical procedures | From baseline (week 0) to end of study (week 104) | Count, assessed as success/failure based on a four-point scale for haemostatic response (excellent, good, moderate and poor) by counting excellent and good as success and moderate and poor as failure |
Countries
Japan
Contacts
STUDY_DIRECTORClinical Reporting Anchor & Disclosure (1452)
Novo Nordisk A/S
Outcome results
None listed