An 18-month study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa
Key facts
- Where
- Budapest XIII, MagyarországSee the site
- Phase
- 4. fázis
Where in Magyarország
- Medical Centre Hungarian Defence ForcesBudapest XIII, MagyarországNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Swedish Orphan Biovitrum AB (publ)
- Trial code
- EU 2022-502921-16-00
- Record updated
- 2023. október 16.
- Original description
To evaluate the overall joint status as detected by ultrasound (US) in haemophilia A and B patients treated with efmoroctocog alfa (rFVIIIFc) or eftrenonacog alfa (rFIXFc) prophylaxis over the 18-month study period.Read full original descriptionShow less
An 18-month low-interventional prospective, multicentre study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa
To evaluate the overall joint status as detected by ultrasound (US) in haemophilia A and B patients treated with efmoroctocog alfa (rFVIIIFc) or eftrenonacog alfa (rFIXFc) prophylaxis over the 18-month study period.
Inclusion: Age ≥ 6 years; Diagnosis of haemophilia A or B; Having at least 6 months documented pre-study treatment data regarding treatment prescriptions and bleeding episodes prior to the baseline visit; Previous treatment for haemophilia A or B with any marketed recombinant and/or plasma-derived FVIII or FIX concentrate for at least 6 months; Start of prophylactic treatment with rFVIIIFc or rFIXFc prior to study enrolment or latest at the baseline visit, in accordance with local regulations; Signed and dated informed consent provided by the patient, or the patient’s legally authorized representative for patients under the legal age. Assent should be obtained from paediatric patients in accordance with local regulations; To be eligible for the florio HAEMO sub-study a patient should have used florio HAEMO (a CE marked medical device used in routine clinical practice) for at least 3 months and must agree to have data collected from the florio HAEMO app by providing a separate informed consent or assent.
Exclusion: Prophylactic treatment with non-factor therapy during the 6 months prior to enrolment; Presence of factor VIII or FIX inhibitory antibodies (inhibitors) (≥0.60 Bethesda Units [BU]/mL) at the latest available inhibitor test; Enrolment in a concurrent clinical interventional study, or intake of an Investigational medicinal product, within 3 months prior to inclusion in this study; Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion; Foreseeable inability to cooperate with given instructions or study procedures.