An 18-month Low-interventional Study to Assess Joint Health in Haemophilia A and B Patients on Prophylaxis With Efmoroctocog Alfa or Eftrenonacog Alfa

NCT ID: NCT05856266

Last Updated: 2025-01-16

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 10 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2023-08-24
• Study Completion Date: 2023-12-06

Brief Summary

The goal of this low-interventional study is to describe the overall joint health in patients with haemophilia A or haemophilia B prophylactically treated with rFVIIIFc or rFIXFc.

The main question it aims to answer is the:

• Evaluation of the overall joint status as detected by ultrasound in haemophilia A and B patients treated with rFVIIIFc or rFIXFc prophylaxis over the 18-month study period.

Participants will come to 6-monthly visits during the 18-month long study period and will perform an ultrasound with the Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) protocol at each visit. At baseline and end of study visits, the patients will be assessed with the clinical scoring system Haemophilia Joint Health Score (HJHS) and complete patient questionnaires. Retrospective data from patient medical records will also be collected for at least 6 months before enrolment in the study.

Detailed Description

The main purpose of this study is to prospectively describe the joint health over an 18-month period of prophylactic treatment with rFVIIIFc or rFIXFc in patients with haemophilia A or haemophilia B in a real-world setting in Europe.

This is a low-interventional, multicentre study using point-of-care US examination for regular monitoring of joint health and detection of hypertrophic synovium, cartilage and bone damage. At the same time, clinical joint status will also be examined by HJHS. This study will evaluate the presence, resolution, recurrence, and new development of target joints. It will also describe bleeding episodes and evaluate the quality of life and physical activity with PROs. If patients are using the CE marked Florio HAEMO app in their routine clinical practice, they will be offered to participate in an optional sub-study that aims to explore possible correlations between the levels of physical activity, the estimated FVIII/FIX levels and bleeding occurrence.

Conditions

Hemophilia A; Hemophilia B

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: OTHER
• Blinding Strategy: NONE

Study Groups

Efmoroctocog alfa or eftrenonacog alfa

Efmoroctocog alfa or eftrenonacog alfa is prescribed and used according to usual clinical practice.

Group Type: OTHER

Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)

Intervention Type: PROCEDURE

Ultrasound examination

Haemophilia Joint Health Score (HJHS)

Intervention Type: OTHER

Functional joint examination

Interventions

Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US)

Ultrasound examination

Intervention Type: PROCEDURE

Haemophilia Joint Health Score (HJHS)

Functional joint examination

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. Age ≥ 6 years

2. Diagnosis of haemophilia A or B

3. Having at least 6 months documented pre-study treatment data regarding treatment prescriptions and bleeding episodes prior to the baseline visit

4. Previous treatment for haemophilia A or B with any marketed recombinant and/or plasma-derived FVIII or FIX concentrate for at least 6 months

5. Start of prophylactic treatment with rFVIIIFc or rFIXFc prior to study enrollment or latest at the baseline visit, in accordance with local regulations

6. 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

Exclusion Criteria

1. Any medical condition which in the opinion of the investigator makes the subject patient unsuitable for inclusion

2. Prophylactic treatment with non-factor therapy during the 6 months prior to enrolment

3. Presence of factor VIII or FIX inhibitory antibodies (inhibitors) (≥0.60 Bethesda Units [BU]/mL) at the latest available inhibitor test

4. Enrolment in a concurrent clinical interventional study, or intake of an IMP, within three months prior to inclusion in this study

5. Foreseeable inability to cooperate with given instructions or study procedures

• Minimum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

IQVIA Pvt. Ltd

INDUSTRY

Sponsor Role: collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Stefan Lethagen, MD, PhD

Role: STUDY_DIRECTOR

Swedish Orphan Biovitrum AB (publ)

Locations

Study site 100

Sofia, , Bulgaria

Study site 101

Sofia, , Bulgaria

Study site 102

Zagreb, , Croatia

Study site 103

Zagreb, , Croatia

Study site 108

Brno, , Czechia

Study site 107

Liberec, , Czechia

Study site 104

Olomouc, , Czechia

Study site 105

Prague, , Czechia

Study site 106

Prague, , Czechia

Study site 115

Bordeaux, , France

Study site 129

Caen, , France

Study site 125

Chambéry, , France

Study site 110

Clermont-Ferrand, , France

Study site 123

Dijon, , France

Study site 124

Lille, , France

Study site 111

Lyon, , France

Study site 126

Marseille, , France

Study site 120

Nancy, , France

Study site 121

Nantes, , France

Study site 113

Nîmes, , France

Study site 119

Paris, , France

Study site 130

Paris, , France

Study site 118

Poitiers, , France

Study site 117

Reims, , France

Sudy site 122

Rennes, , France

Study site 109

Rouen, , France

Study site 112

Strasbourg, , France

Study site 128

Toulouse, , France

Study site 114

Tours, , France

Study site 131

Budapest, , Hungary

Study site 132

Dublin, , Ireland

Study site 133

Dublin, , Ireland

Study site 136

Bologna, , Italy

Study site 134

Brescia, , Italy

Study site 138

Catania, , Italy

Study site 140

Milan, , Italy

Study site 137

Palermo, , Italy

Study site 135

Pisa, , Italy

Study site 139

Vicenza, , Italy

Study site 145

Baia Mare, , Romania

Study site 141

Bucharest, , Romania

Study site 143

Craiova, , Romania

Study site 146

Iași, , Romania

Study site 142

Timișoara, , Romania

Study site 147

Ljubljana, , Slovenia

Study site 151

A Coruña, , Spain

Study site 150

Alicante, , Spain

Study site 152

Barcelona, , Spain

Study site 149

Madrid, , Spain

Study site 154

Málaga, , Spain

Study site 156

Oviedo, , Spain

Study site 153

Salamanca, , Spain

Study site 157

Valencia, , Spain

Study site 155

Valladolid, , Spain

Study site 158

Zaragoza, , Spain

Study site 159

Belfast, , United Kingdom

Countries

Bulgaria; Croatia; Czechia; France; Hungary; Ireland; Italy; Romania; Slovenia; Spain; United Kingdom

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

2022-502921-16-00

Identifier Type: OTHER

Identifier Source: secondary_id

Sobi.HAEM89-007

Identifier Type: -

Identifier Source: org_study_id