Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy
ConditionFactor VIII Deficiency
Trial statusRecruiting
Trial phasePhase 4
Trial typeInterventional
Biological sexAll
Age12+
SponsorSanofi
About this trial
The objective of the study is to assess the improvement of synovial hypertrophy during the 12 months of efanesoctocog alfa prophylaxis once per week (QW) in joints with existing evidence of synovial hypertrophy in participants with hemophilia A.
The study duration for each participant is approximately 12 months.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
None
Trial design
Design model
Single group
Treatments tested in this trial
Efanesoctocog alfa
DrugPharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous
Treatment groups
35 Participants
are divided into 1 treatment groupGroup A: Efanesoctocog AlfaExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Time frame
From Baseline up to Week 52Secondary outcomes
1
Change from baseline in the distribution of joint HEAD-US synovitis score
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Time frame
From Baseline up to Week 522
Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Time frame
From Baseline up to Week 523
Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Time frame
From Baseline up to Week 524
Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment.
Time frame
From Baseline up to Week 52 Source ClinicalTrials.gov