Hemophilia A
Conditions
Interventions
Study drug name: Efanesoctocog alfa
Dose formulation: Injection
Route of administration: IV
Treatment group: single-arm
Treatment will be administered in accordance with protocol.
Sponsors
Tsao Nicole
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to = 12 years old at the time of consent/assent. Participant has existing synovial hypertrophy, defined as at least 1 eligible joint by the HEAD-US score (Synovitis score: 1 or 2) at the time of consent/assent.
Exclusion criteria
Exclusion criteria: Participant has other associated clotting disorders at the time of consent/assent. Participant is already under efanesoctocog alfa treatment. Participant has a current diagnosis of an FVIII inhibitor, defined as inhibitor titer >= 0.60 BU/mL Participant has ITI within the last 2 years prior to the baseline visit (Visit 2).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The proportion of joints with improvement (ie, at least 1 point decrease from baseline) in the HEAD-US synovitis domain score at Week 52. | — |
Secondary
| Measure | Time frame |
|---|---|
| 1) Change in the distribution of joint HEAD-US synovitis score at Week 52 from baseline. 2) The number and percent of joints with the HEADUS synovitis domain score that has remained unchanged or worsened at Week 52 from baseline, in all index joints. 3) The number and percent of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened at Week 52 from baseline, as reported by index joint. 4) The number and percent of patients with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened at Week 52 from baseline. 5) Change in average HEAD-US synovitis domain score across all index joints per patient at Week 52 from baseline. 6) Change in total/domain scores of the HJHS at Week 26 from baseline. 7) Change in total/domain scores of the HJHS at Week 52 from baseline. 8) Change in PROs, as assessed by the EQ-5D-5L, PROMIS-SF (v2.0) Pain Intensity 3a, PROMIS-SF (v1.1) Pain Interference 6a, and PROMIS-SF (v2.0) Physical Function 6b, at Week 52 from baseline. 9) Patient-reported treatment preference and satisfaction at Week 52 (assessed through surveys and an exit interview). 10) Assess the effectiveness of efanesoctocog alfa prophylaxis QW during 12 months. 11) Change in ABR and AjBR (spontaneous, traumatic) at Week 52 from baseline. 12) Change in the number of patients with target joint resolution, recurrence, or development at Week 52 from baseline. 13) Assess the safety and tolerability of efanesoctocog alfa prophylaxis QW. 14) The occurrence of AEs, all AEs leading to treatment discontinuation, SAEs, and AESIs. | — |
Countries
Australia, Japan, Taiwan, U.S.A.
Contacts
Public ContactNicole Tesao
Sanofi
Outcome results
None listed