Efanesoctocog Alfa Prophylaxis in Patients With Hemophilia A With Synovial Hypertrophy

NCT ID: NCT06941870

Last Updated: 2025-11-18

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE4

Total Enrollment

35 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-09-23

Study Completion Date

2027-10-25

Brief Summary

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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.

Detailed Description

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Conditions

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Factor VIII Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Phase 3 in Taiwan, Canada and Australia, Phase 4 in US and Japan.
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Efanesoctocog Alfa

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

Group Type EXPERIMENTAL

Efanesoctocog alfa

Intervention Type DRUG

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

Interventions

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Efanesoctocog alfa

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

Intervention Type DRUG

Other Intervention Names

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BIVV001 ALTUVIIIO®

Eligibility Criteria

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Inclusion Criteria

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).

Exclusion Criteria

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.
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Sanofi

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Orthopedic Institute for Children- Site Number : 8400009

Los Angeles, California, United States

Site Status RECRUITING

Innovative Hematology, Inc.- Site Number : 8400006

Indianapolis, Indiana, United States

Site Status RECRUITING

University of Iowa- Site Number : 8400004

Iowa City, Iowa, United States

Site Status RECRUITING

Ohio State University Hospital East- Site Number : 8400001

Columbus, Ohio, United States

Site Status RECRUITING

Investigational Site Number : 1240003

Hamilton, Ontario, Canada

Site Status RECRUITING

Investigational Site Number : 1240004

Hamilton, Ontario, Canada

Site Status RECRUITING

Investigational Site Number : 3920003

Kawasaki, Kanagawa, Japan

Site Status RECRUITING

Investigational Site Number : 3920002

Hiroshima, , Japan

Site Status RECRUITING

Investigational Site Number : 3920001

Saitama, , Japan

Site Status RECRUITING

Investigational Site Number : 3920004

Tokyo, , Japan

Site Status RECRUITING

Investigational Site Number : 1580003

Kaohsiung City, , Taiwan

Site Status RECRUITING

Investigational Site Number : 1580001

Taichung, , Taiwan

Site Status RECRUITING

Investigational Site Number : 1580002

Taipei, , Taiwan

Site Status RECRUITING

Countries

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United States Canada Japan Taiwan

Central Contacts

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

Role: CONTACT

800-633-1610 ext. option 6

Related Links

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Other Identifiers

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U1111-1311-1701

Identifier Type: REGISTRY

Identifier Source: secondary_id

LPS18168

Identifier Type: -

Identifier Source: org_study_id

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