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

NCT05856266 | Terminated Phase 4 Results

• 2 other identifiers: Sobi.HAEM89-0072022-502921-16-00
• Study Type: interventional
• Target: 10
• Locations: 11 countries
• Sites: 56

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.

Conditions

Hemophilia A; Hemophilia B

Keywords

Blood coagulation disorder; Coagulation protein disorder; Haemophilia; FVIII; FIX; Efmoroctocog alfa; Eftrenonacog alfa

Outcome Measures

Primary Outcomes (1)

• Change From Baseline in Total HEAD-US Score up to Month 18 (End of Study - EOS)

  HEAD-US is a standardized, fast, repeatable, and simple ultrasound scoring protocol for hemophiliac joints. The HEAD-US joint score will be calculated for the six index joints (elbows, knees, and ankles). The specific joint score is made up of three item scores: disease activity (synovitis), disease damage of articular surfaces, and disease damage of subchondral bone. The specific joint score is the sum of the three item scores for each specific joint. Its values range from 0 (minimum) to 8 (maximum). The total score represents the sum of item scores for abnormalities detected.

  Baseline and month 18

Secondary Outcomes (14)

• Change From Baseline in HEAD-US Score for Hypertrophic Synovium

  Baseline, month 6, 12 and 18

• Change From Baseline in HEAD-US Score for Cartilage

  Baseline, month 6, 12 and 18

• Change From Baseline in HEAD-US Score for Bone

  Baseline, month 6, 12 and 18

• Change From Baseline in Total Hemophilia Joint Health Score (HJHS) at Month 18 (EOS)

  Baseline and month 18

• Number of Target Joints

  Baseline, month 6, 12 and 18

Study Arms (1)

Efmoroctocog alfa or eftrenonacog alfa

OTHER

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

Procedure: Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US); Other: Haemophilia Joint Health Score (HJHS)

Interventions

Haemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) PROCEDURE

Ultrasound examination

Efmoroctocog alfa or eftrenonacog alfa

Haemophilia Joint Health Score (HJHS) OTHER

Functional joint examination

Efmoroctocog alfa or eftrenonacog alfa

Eligibility Criteria

• Age: 6 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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 enrollment 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

You may not qualify if:

• 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
• Foreseeable inability to cooperate with given instructions or study procedures

Sponsors & Collaborators

• Swedish Orphan Biovitrum: lead
• IQVIA Pvt. Ltd: collaborator

Study Sites (56)

Study site 100

Sofia, Bulgaria

Location

Study site 101

Sofia, Bulgaria

Location

Study site 102

Zagreb, Croatia

Location

Study site 103

Zagreb, Croatia

Location

Study site 108

Brno, Czechia

Location

Study site 107

Liberec, Czechia

Location

Study site 104

Olomouc, Czechia

Location

Study site 105

Prague, Czechia

Location

Study site 106

Prague, Czechia

Location

Study site 115

Bordeaux, France

Location

Study site 129

Caen, France

Location

Study site 125

Chambéry, France

Location

Study site 110

Clermont-Ferrand, France

Location

Study site 123

Dijon, France

Location

Study site 124

Lille, France

Location

Study site 111

Lyon, France

Location

Study site 126

Marseille, France

Location

Study site 120

Nancy, France

Location

Study site 121

Nantes, France

Location

Study site 113

Nîmes, France

Location

Study site 119

Paris, France

Location

Study site 130

Paris, France

Location

Study site 118

Poitiers, France

Location

Study site 117

Reims, France

Location

Sudy site 122

Rennes, France

Location

Study site 109

Rouen, France

Location

Study site 112

Strasbourg, France

Location

Study site 128

Toulouse, France

Location

Study site 114

Tours, France

Location

Study site 131

Budapest, Hungary

Location

Study site 132

Dublin, Ireland

Location

Study site 133

Dublin, Ireland

Location

Study site 136

Bologna, Italy

Location

Study site 134

Brescia, Italy

Location

Study site 138

Catania, Italy

Location

Study site 140

Milan, Italy

Location

Study site 137

Palermo, Italy

Location

Study site 135

Pisa, Italy

Location

Study site 139

Vicenza, Italy

Location

Study site 145

Baia Mare, Romania

Location

Study site 141

Bucharest, Romania

Location

Study site 143

Craiova, Romania

Location

Study site 146

Iași, Romania

Location

Study site 142

Timișoara, Romania

Location

Study site 147

Ljubljana, Slovenia

Location

Study site 151

A Coruña, Spain

Location

Study site 150

Alicante, Spain

Location

Study site 152

Barcelona, Spain

Location

Study site 149

Madrid, Spain

Location

Study site 154

Málaga, Spain

Location

Study site 156

Oviedo, Spain

Location

Study site 153

Salamanca, Spain

Location

Study site 157

Valencia, Spain

Location

Study site 155

Valladolid, Spain

Location

Study site 158

Zaragoza, Spain

Location

Study site 159

Belfast, United Kingdom

Location

MeSH Terms

Conditions

Hemophilia A; Hemophilia B; Blood Coagulation Disorders; Coagulation Protein Disorders

Interventions

High-Energy Shock Waves

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Hematologic Diseases; Hemic and Lymphatic Diseases; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Ultrasonic Waves; Sound; Radiation, Nonionizing; Radiation; Physical Phenomena

Limitations and Caveats

Study was terminated early. Only 10 patients had baseline assessments.

Results Point of Contact

• Title: CPL
• Organization: Biovitrum

rolf.schlosshauer@sobi.com

0795914760

Study Officials

• Stefan Lethagen, MD, PhD

  Swedish Orphan Biovitrum AB (publ)

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: This is a low-interventional study, with the drugs being prescribed and used according to clinical practice. The interventions in the study are the addition of the mandatory joint examinations HEAD-US (ultrasound) and HJHS (functional joint assessment).

Study Record Dates

• First Submitted: April 24, 2023
• First Posted: May 12, 2023
• Study Start: August 24, 2023
• Primary Completion: December 6, 2023
• Study Completion: December 6, 2023
• Last Updated: January 16, 2025
• Results First Posted: January 16, 2025

Record last verified: 2025-01

Data Sharing

• IPD Sharing: Will not share

Locations

ES (10); IE (2); RO (5); GB (1); CR (2); CZ (5); HU (1); BU (2); FR (20); IT (7); SL (1)