Clinical trial NCT05621746: An Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine Esperoct
Official title: Non-Interventional Study of the Change in Joint Health in Adult Patients With Haemophilia A After Switching to Prophylaxis With Turoctocog Alfa Pegol (N8-GP)
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Haemophilia A
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2022-11-18
Enrollment: 100
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Participants with Haemophilia A

Brief summary: This study will collect information on the long term health of joints in people with haemophilia A who have started treatment with Esperoct within twelve months prior to participation to the study. This study is conducted to look at how joint health of people with haemophilia changes over time when they are receiving the medicine Esperoct. The participants will get Esperoct as prescribed to the participants by the study doctor. The participant's treatment will not be affected by their involvement in the study. Every six months, the participants will be asked to answer some questionnaires about their joints, their pain and their ability to be physically active. Their participation in the study will last for no more than 2 years. The participants are free to leave the study at any time and for any reason. This will not affect their current and future medical care.


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).
* Male, greater than or equal to 18 years of age at the time of signing informed consent, diagnosed with severe (FVIII activity below 1%) or moderate congenital haemophilia A (FVIII activity 1-5%).
* The decision to initiate treatment with commercially available Esperoct has been made by the patient and the treating physician before and independently from the decision to include the patient in this study.
* Switched, within two months prior to enrolment, OR planned to switch, within one month post enrolment, to prophylaxis treatment with Esperoct from previous therapy; the decision to initiate treatment with Esperoct must be made prior to and independently from the decision to enrol in the study.
* Must have baseline data (HJHS, target joints, and medical history) collected in routine clinical practice within two months prior or up to one month post switch to Esperoct therapy.

Exclusion Criteria:

* Previous participation in this study. Participation is defined as having given informed consent in this study.
* Previous terminated treatment regimen with Esperoct prophylaxis.
* Current or previously terminated treatment regimen with Esperoct on-demand.
* Mental incapacity, unwillingness or language barriers precluding adequate understanding or cooperation.
* Previous participation in a clinical trial within the 30 days prior to switching to Esperoct.

Primary outcomes:
measure: Change in haemophilia joint health score (HJHS)
timeFrame: From date of switch to end of study (up to 24 months)
Secondary outcomes:
measure: The number of bleeding episodes requiring coagulation Factor VIII (FVIII) treatment
timeFrame: From baseline to end of study (up to 24 months)
measure: Number of target joints
timeFrame: From baseline to end of study (up to 24 months)
measure: Resolution of any target joints (Yes/No)
timeFrame: From baseline to end of study (up to 24 months)
measure: Change in patient reported Problem Joint score
timeFrame: From baseline to end of study (up to 24 months)
measure: Change in patient reported pain scores (Brief Pain Inventory)
timeFrame: From baseline to end of study (up to 24 months)
measure: Change in physical function and activity measured using patient reported outcome questionnaires (Short Form-36)
timeFrame: From baseline to end of study (up to 24 months)
Other outcomes:
Locations:
city: Hamilton
country: Canada
facility: Novo Nordisk Investigational Site
state: Ontario
Study officials:
affiliation: Novo Nordisk A/S'
name: Clinical Transparency' dept. 2834
role: STUDY_DIRECTOR
References: