A Study to Evaluate the Real-world Effectiveness and Usage of Alprolix in Patients With Haemophilia B in France

Completed

• Conditions: Haemophilia B
• Interventions: Drug: Alprolix

• Registration Number: NCT03655340
• Lead Sponsor: Swedish Orphan Biovitrum

Brief Summary

Alprolix (rFIXFc) is a recombinant extended half-life coagulation factor product. The purpose of this non-interventional study is to describe the real-world usage and effectiveness of Alprolix in the on-demand and prophylactic treatment of haemophilia B.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-08-30
• Study First Submitted QC: 2018-08-30
• Study First Posted: 2018-08-31
• Study Start: 2018-09-12
• Primary Completion: 2022-03-16
• Study Completion: 2022-03-16
• Status Verified: 2024-07
• Last Update Submitted: 2024-07-02
• Last Update Posted: 2024-07-03

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 91

Inclusion Criteria

• Have a diagnosis of haemophilia B and been treated previously with factor IX Product
• Have started Alprolix treatment prior to enrolment visit, or at enrolment prescribed treatment with Alprolix, regardless of participation in the study
• Signed and dated informed consent provided by the patient, or the patient's legally acceptable representative for patients under the legal age, before any study-related activities are undertaken. Assent should be obtained from paediatric patients according to local regulations

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Exclusion Criteria

• Participation in an investigational medicinal product trial at enrolment visit

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Prophylactic patients	Alprolix	Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for prophylactic treatment
On demand patients	Alprolix	Alprolix will be prescribed according to local practice and administered by patients with haemophilia B for on-demand treatment

Primary Outcome Measures

Name	Time	Method
Amount of factor Product used to treat a bleed (on-demand treatment)	24 months	Assessed by diary
Annualised bleeding rate (ABR) (prophylactic treatment)	24 months	Assessed by diary
Annualised injection frequency (prophylactic treatment)	24 months	Assessed by diary
Number of injections to treat a bleed (on-demand treatment)	24 months	Assessed by diary
Annualised factor consumption (International Unit [IU]) (prophylactic treatment)	24 months	Assessed by diary

Trial Locations

Locations (3)

Swedish Orphan Biovitrum Research Site; 🇫🇷 Toulouse, France

Swedish Orphan Biovitrum Research Site (CHU de Bordeaux); 🇫🇷 Bordeaux, France

Swedish Orphan Biovitrum research site; 🇫🇷 Strasbourg, France