Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

Phase 4RecruitingNCT06941870

Sanofi35 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Factor VIII Deficiency

Interventions

Efanesoctocog alfaDRUG

Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous

Eligibility

Sex: ALLMin age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent. Participant is more 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. Participant has at least 1 eligible\* joint without planned future major orthopedic procedures (example, arthroscopic synovectomy, radioisotopic or chemical synoviorthesis), or major orthopedic procedures in the past 3 months prior to the screening visit (Visit 1). \*An eligible joint is a joint with existing synovial hypertrophy, as defined by a HEAD-US synovitis score of 1 or 2, considering hypertrophic synovium as an indication of the presence of synovitis Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2). Participant is capable of understanding the written Informed Consent Form (ICF)/assent form, provides a signed and witnessed written ICF/assent form, and agrees to comply with the protocol requirements. If male, no contraceptive measures are required for this study. If female, is not pregnant or breastfeeding, and one of the following conditions applies: Is a women of nonchildbearing potential (WONCBP) Is a women of childbearing potential (WOCBP) and agrees to use a highly effective contraceptive method, with a failure rate of less than (\<)1 percent during the study treatment period (at least until the Week 52/ End of Treatment \[EoT\] visit). A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1) Exclusion Criteria: Participants are excluded from the study if any of the following criteria apply: Has other associated clotting disorders at the time of consent/assent. Is already under efanesoctocog alfa treatment. Has a current diagnosis of an factor VIII (FVIII) inhibitor, defined as inhibitor titer ≥0.60 BU/mL. Has ITI within the last 2 years prior to the baseline visit (Visit 2). Has been enrolled in a concurrent clinical interventional study or exposed to other investigational drug(s) within 3 months prior to screening for this study. Is currently in an institution because of regulatory or legal order (that is, is a prisoner or a patient who is legally institutionalized). Is 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. Is an employee or family member of the investigator or site personnel. Is involved in a specific situation during study implementation or the course of the study that may raise ethics considerations. Has hypersensitivity to efanesoctocog alfa or its components or any of its excipients that, in the opinion of the investigator, contraindicates participation in the study.

Locations (13)

Orthopedic Institute for Children- Site Number : 8400009

Los Angeles, California, United States

RECRUITING

Innovative Hematology, Inc.- Site Number : 8400006

Indianapolis, Indiana, United States

Outcomes

Primary Outcomes

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 52

Secondary Outcomes

Change from baseline in the distribution of joint HEAD-US synovitis score

Time frame: From Baseline up to Week 52

Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened

Time frame: From Baseline up to Week 52

Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened

Time frame: From Baseline up to Week 52

Central Contacts

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610 Contact-US@sanofi.com

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.

University of Iowa- Site Number : 8400004

Iowa City, Iowa, United States

RECRUITING

Ohio State University Hospital East- Site Number : 8400001

Columbus, Ohio, United States

RECRUITING

Investigational Site Number : 1240003

Hamilton, Ontario, Canada

RECRUITING

Investigational Site Number : 1240004

Hamilton, Ontario, Canada

RECRUITING

Investigational Site Number : 3920003

Kawasaki, Kanagawa, Japan

RECRUITING

Investigational Site Number : 3920002

Hiroshima, Japan

RECRUITING

Investigational Site Number : 3920001

Saitama, Japan

RECRUITING

Investigational Site Number : 3920004

Tokyo, Japan

RECRUITING

...and 3 more locations

Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened

Time frame: From Baseline up to Week 52

Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened

Time frame: From Baseline up to Week 52

Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened

Time frame: From Baseline up to Week 52

Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened

Time frame: From Baseline up to Week 52

Change from baseline in average HEAD-US synovitis domain score across all index joints

Time frame: From Baseline up to Week 52

Change from baseline in total/ domain scores of the HEAD-US

Time frame: From Baseline up to Week 26

Change from baseline in total/domain scores of the HJHS

HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health.

Time frame: From Baseline up to Week 26

Change from baseline in total/domain scores of the HJHS

Time frame: From Baseline up to Week 52

Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)

MRI will be used to detect changes in synovial hypertrophy in index joints of participants.

Time frame: From Baseline up to Week 52

Change from baseline in patient-reported outcome (PRO)

Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b.

Time frame: From Baseline up to Week 52

Patient reported treatment preference and satisfaction

Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews.

Time frame: At Week 52

Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)

The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period \[days\]) × 365.25.

Time frame: At Week 52

Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)

The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period \[days\]) × 365.25.

Time frame: At Week 52

Change from baseline in target joint resolution, recurrence, or development

Change from baseline in target joint resolution, recurrence, or development will be assessed.

Time frame: At Week 52

The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)

The safety and tolerability of efanesoctocog alfa evaluated in all participants

Time frame: Up to Week 52