An Observational Research Study of the Health of Joints in People With Haemophilia Taking the Medicine Esperoct

Официальное название

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)

Обзор

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.

Вмешательства

• Препарат Esperoct
  Participants will be treated with commercially available Esperoct for a total study duration of 24 months according to the local label and local routine clinical practice at the discretion of the physician. The decision to switch to Esperoct will be made prior to and separate from the decision to enrol in the study.

Первичные конечные точки

• Change in haemophilia joint health score (HJHS) [Срок оценки: From date of switch to end of study (up to 24 months)]

Вторичные конечные точки (6)

• The number of bleeding episodes requiring coagulation Factor VIII (FVIII) treatment [Срок оценки: From baseline to end of study (up to 24 months)]
• Number of target joints [Срок оценки: From baseline to end of study (up to 24 months)]
• Resolution of any target joints (Yes/No) [Срок оценки: From baseline to end of study (up to 24 months)]
• Change in patient reported Problem Joint score [Срок оценки: From baseline to end of study (up to 24 months)]
• Change in patient reported pain scores (Brief Pain Inventory) [Срок оценки: From baseline to end of study (up to 24 months)]
• Change in physical function and activity measured using patient reported outcome questionnaires (Short Form-36) [Срок оценки: From baseline to end of study (up to 24 months)]

Критерии участия

Критерии включения

• 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.

Критерии исключения

• 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.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

Канада · 1 центр

• Novo Nordisk Investigational Site — Hamilton

Идентификаторы

NCT: NCT05621746 · NN7088-4928 · U1111-1271-9209

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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