An 18-month study to assess joint outcomes in patients with haemophilia A or B on prophylaxis with efmoroctocog alfa or eftrenonacog alfa

Phase 1

Recruiting

• Conditions: Hemophilia A and B; MedDRA version: 20.0Level: LLTClassification code: 10018939Term: Haemophilia B (Factor IX) Class: 10010331; MedDRA version: 20.0Level: LLTClassification code: 10018937Term: Haemophilia A Class: 10010331; Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]

• Registration Number: CTIS2022-502921-16-00
• Lead Sponsor: Swedish Orphan Biovitrum AB (publ)

Brief Summary

ow Intervention Clinical Trial Report Summary

Detailed Description

Not available

Study Timeline

• Study Start: 2023-02-17
• Last Update Posted: 21 August 2024

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 250

Inclusion Criteria

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 Criteria

Any medical condition which in the opinion of the investigator makes the patient unsuitable for inclusion, 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, Foreseeable inability to cooperate with given instructions or study procedures

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified