efanesoctocog alfa for Haemophilia A

Inclusion criteria

• {"criterion_text":"- Capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and protocol. Parents’ or legally designated representatives’ consent is required for patients who are <18 years of age or unable to give consent, or as applicable per local laws. Patients who are <18 years of age should provide assent in addition to the parents’/legally designated representatives’ consent, if appropriate.\n- Male or female patients who are ≥12 years of age and diagnosed with moderate or severe haemophilia A (defined as ≤5% of normal FVIII clotting activity) at the time of signing the ICF.\n- A female patient is eligible to participate if she is not pregnant at enrolment and does not plan to become pregnant during the study. A woman of child-bearing potential (WOCBP) must have a negative highly sensitive serum pregnancy test at the Screening Visit.\n- Must have received prophylactic treatment per local label with any marketed FVIII product or emicizumab for ≥12 months prior to the Baseline Visit.\n- Have at least one eligible index joint (ankle, elbow, knee).\n- Have 12 months of documented pre-study treatment data on haemophilia prescriptions and on treated bleeding episodes prior to the Baseline Visit.\n- Willingness and the ability of the patient or their legally designated representative to complete training in the use of the study patient diary and to complete the diary throughout the study."}

Exclusion criteria

• {"criterion_text":"- Blood clotting disorders other than haemophilia A\n- Patient not suitable for participation, whatever the reason, as judged by the Investigator, including medical or clinical conditions, or patients potentially at risk of noncompliance to study procedures.\n- Enrolment in a concurrent clinical interventional study, or intake of an investigational medicinal product (IMP), within 3 months prior to inclusion in the study.\n- Already on efanesoctocog alfa treatment\n- Positive inhibitor result (assessed by local laboratory) from the Screening Visit, defined as ≥0.6 Bethesda units (BU)/mL.\n- History of inhibitors without successful immune tolerance induction (ITI) • Successful ITI is defined as: •\tNegative inhibitor titer (<0.6 BU/mL) • FVIII recovery > 66% of expected • FVIII half-life ≥ 6 hours\n- ITI performed within the last 2 years prior to the Baseline Visit.\n- Currently receiving treatment with any of the prohibited concomitant medications, as specified by the protocol.\n- Planned major orthopaedic procedure in any eligible index joint during the course of the study.\n- Patients are not eligible for participation in the study if they cannot undergo MRI assessments at the Baseline Visit.\n- Patients with known hypersensitivity to the active substance or to any of the excipients."}

Primary endpoints

• {"endpoint_text":"- Joints with synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score of 1 or 2) and at least 1 point decrease in HEAD-US synovial hypertrophy domain score at Month 12 (Yes/No)","definition_or_measurement_approach":"Assessment using HEAD-US synovial hypertrophy domain scoring per joint; baseline score of 1 or 2 and improvement defined as at least 1 point decrease at Month 12; endpoint assessed as Yes/No per joint."}

Secondary endpoints

• {"endpoint_text":"- Joints with no synovial hypertrophy at baseline (HEAD-US synovial hypertrophy domain score 0) and at least 1 point increase in HEAD-US synovial hypertrophy domain score at Month 6 or Month 12 (Yes/No)","definition_or_measurement_approach":"HEAD-US synovial hypertrophy domain score assessed at baseline and at Months 6 and 12; deterioration defined as ≥1 point increase; binary Yes/No per joint at Month 6 or 12."}
• {"endpoint_text":"- Distribution of joint HEAD-US synovial hypertrophy domain scores 0/1/2 at baseline and at Months 6 and 12","definition_or_measurement_approach":"Descriptive distribution of HEAD-US synovial hypertrophy domain scores (0/1/2) per joint at baseline, Month 6 and Month 12."}
• {"endpoint_text":"- Change from baseline in total/domain scores of HEAD-US per patient and per joint at Months 6 and 12","definition_or_measurement_approach":"Change in total and domain HEAD-US scores calculated per patient and per joint at Months 6 and 12 versus baseline."}
• {"endpoint_text":"- Change from baseline in total/domain scores of International Prophylaxis Study Group (IPSG) MRI per patient and per joint at Month 12","definition_or_measurement_approach":"IPSG MRI total and domain scores assessed at baseline and Month 12; change from baseline reported per patient and per joint."}
• {"endpoint_text":"- Change from baseline in total/domain scores of HJHS per patient and per joint at Month 12","definition_or_measurement_approach":"Haemophilia Joint Health Score (HJHS) total and domain scores assessed at baseline and Month 12; change from baseline reported per patient and per joint."}
• {"endpoint_text":"- Patients with improved, unchanged, or worsened total/domain HEAD-US/MRI/HJHS scores at Month 12 from baseline","definition_or_measurement_approach":"Categorical assessment (improved/unchanged/worsened) of total and domain scores for HEAD-US, MRI (IPSG) and HJHS at Month 12 compared with baseline."}
• {"endpoint_text":"- Changes in PROs from baseline to Month 6 and Month 12 (assessed by 5- level EuroQol-5 dimensions [EQ-5D-5L], Patient-Reported Outcomes Measurement Information System [PROMIS] pain intensity, PROMIS pain interference, and PROMIS physical function)","definition_or_measurement_approach":"Patient-reported outcomes measured using EQ-5D-5L and PROMIS instruments at baseline, Month 6 and Month 12; changes from baseline reported."}
• {"endpoint_text":"- Patient-reported treatment preference at Month 12 (assessed with a questionnaire and exit interview)","definition_or_measurement_approach":"Patient treatment preference assessed at Month 12 using a questionnaire and exit interview; descriptive reporting of preference."}
• {"endpoint_text":"- Change in ABR and annualized joint bleeding rate (AjBR) (spontaneous, traumatic) based on treated bleeds from the retrospective data collection period to on-study period","definition_or_measurement_approach":"Annualised bleeding rates (ABR) and AjBR for spontaneous and traumatic treated bleeds compared between retrospective pre-study period and on-study period."}
• {"endpoint_text":"- Patients with target joint resolution or development from baseline to Month 12","definition_or_measurement_approach":"Assessment of resolution or development of target joints between baseline and Month 12; categorical reporting per patient."}
• {"endpoint_text":"- The occurrence of treatment-emergent adverse events (TEAEs) leading to treatment discontinuation, serious TEAEs, and adverse events of special interest (AESIs)","definition_or_measurement_approach":"Safety monitoring of TEAEs including those leading to treatment discontinuation, serious TEAEs, and AESIs; reported per standard safety reporting."}

Spain

Earliest CTIS Part Ii Submission Date

30-10-2024

Latest Decision Or Authorization Date

17-11-2025

Processing Time Days

383

Number Of Sites

4

Number Of Participants

11

Italy

Earliest CTIS Part Ii Submission Date

28-10-2024

Latest Decision Or Authorization Date

14-11-2025

Processing Time Days

382

Number Of Sites

3

Number Of Participants

10

Sweden

Earliest CTIS Part Ii Submission Date

28-10-2024

Latest Decision Or Authorization Date

11-11-2025

Processing Time Days

379

Number Of Sites

2

Number Of Participants

7

Norway

Earliest CTIS Part Ii Submission Date

08-11-2024

Latest Decision Or Authorization Date

14-11-2025

Processing Time Days

371

Number Of Sites

1

Number Of Participants

12

Primary sponsor

Full Name

Swedish Orphan Biovitrum AB (publ)

Organisation Type

Pharmaceutical company

Country Of Registered Address

Sweden

Contract research organisations

Name

Psi Cro AG

Responsibilities

sponsorDuties codes: 1,10,11,12,15 (translations; vendor management),2,5,6

Name

4G Clinical B.V.

Responsibilities

sponsorDuties codes: 3

Name

Almac Clinical Services Limited

Responsibilities

sponsorDuties codes: 14

Third parties

• {"country":"Switzerland","full_name":"Psi Cro AG","duties_or_roles":"sponsorDuties codes: 1,10,11,12,15 (translations; vendor management),2,5,6; contact: contact@psi-cro.com","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"4G Clinical B.V.","duties_or_roles":"sponsorDuties codes: 3; contact: info@4gclinical.com","organisation_type":"Non-Pharmaceutical company"}
• {"country":"Sweden","full_name":"SliceVault AB","duties_or_roles":"sponsorDuties codes: 15 (Imaging portal); contact: contact@slicevault.com","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Veeva Systems Inc.","duties_or_roles":"sponsorDuties codes: 15 (eTMF),7; contact: pr@veeva.com","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Clinical Services Limited","duties_or_roles":"sponsorDuties codes: 14; contact: clinicalservices@almacgroup.com","organisation_type":"Pharmaceutical company"}