Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

NCT06941870 | Recruiting Phase 4 FDA Drug

• 2 other identifiers: LPS18168U1111-1311-1701
• Study Type: interventional
• Target: 35
• Locations: 4 countries
• Sites: 13

Brief Summary

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.

Conditions

Factor VIII Deficiency

Outcome Measures

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.

  From Baseline up to Week 52

Secondary Outcomes (18)

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

  From Baseline up to Week 52

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

  From Baseline up to Week 52

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

  From Baseline up to Week 52

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

  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

  From Baseline up to Week 52

Study Arms (1)

Efanesoctocog Alfa

EXPERIMENTAL

Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW

Drug: Efanesoctocog alfa

Interventions

Efanesoctocog alfa DRUG

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

Also known as: BIVV001, ALTUVIIIO®

Efanesoctocog Alfa

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Sanofi: lead

Study Sites (13)

Orthopedic Institute for Children- Site Number : 8400009

Los Angeles, California, 90007, United States

RECRUITING

Innovative Hematology, Inc.- Site Number : 8400006

Indianapolis, Indiana, 46260, United States

RECRUITING

University of Iowa- Site Number : 8400004

Iowa City, Iowa, 52242, United States

RECRUITING

Ohio State University Hospital East- Site Number : 8400001

Columbus, Ohio, 43203-1779, United States

RECRUITING

Investigational Site Number : 1240003

Hamilton, Ontario, L8N 3Z5, Canada

RECRUITING

Investigational Site Number : 1240004

Hamilton, Ontario, L8N 3Z5, Canada

RECRUITING

Investigational Site Number : 3920003

Kawasaki, Kanagawa, 216-8511, Japan

RECRUITING

Investigational Site Number : 3920002

Hiroshima, 734-8551, Japan

RECRUITING

Investigational Site Number : 3920001

Saitama, 330-8777, Japan

RECRUITING

Investigational Site Number : 3920004

Tokyo, 160-0023, Japan

RECRUITING

Investigational Site Number : 1580003

Kaohsiung City, 80756, Taiwan

RECRUITING

Investigational Site Number : 1580001

Taichung, 407, Taiwan

RECRUITING

Investigational Site Number : 1580002

Taipei, 114, Taiwan

RECRUITING

Related Links

• LPS18168 Plain Language Results Summary

MeSH Terms

Conditions

Hemophilia A

Interventions

BIVV001

Condition Hierarchy (Ancestors)

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

Central Study Contacts

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

CONTACT

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

Study Design

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

Model Details: Phase 3 in Taiwan, Canada and Australia, Phase 4 in US and Japan.

Study Record Dates

• First Submitted: April 16, 2025
• First Posted: April 24, 2025
• Study Start: September 23, 2025
• Primary Completion (Estimated): October 25, 2027
• Study Completion (Estimated): October 25, 2027
• Last Updated: November 18, 2025

Record last verified: 2025-11

Data Sharing

• IPD Sharing: Will share

Locations

US (4); JP (4); TW (3); CA (2)