A Study to Evaluate Impact of Efanesoctocog Alfa on Long-term Joint Health in Participants With Hemophilia A

March 7, 2024 updated by: Sanofi

Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A

This is a prospective, observational, multi-center longitudinal cohort study to describe the real-world effectiveness, safety and treatment usage of efanesoctocog alfa in patients with hemophilia A treated per standard of care in the US and Japan.

Patients will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each study country. Decision to initiate treatment with commercially available efanesoctocog alfa will be made by the treating physician independently from the decision to include patients in the study. No study medication is provided. The data related to efanesoctocog alfa effectiveness, safety and usage will be collected prospectively during routine visits (expected annual/semi-annual visits) for up to 5 years following enrollment /treatment initiation.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

  • Name: Trial Transparency email recommended (Toll free number for US & Canada)
  • Phone Number: Option 6 1800633-1610
  • Email: contact-us@sanofi.com

Study Locations

  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Recruiting
        • Orthopedic Institute for Children-Site Number: 8400004
      • Orange, California, United States, 92868-4306
        • Recruiting
        • Center for Inherited Blood Disorders (CIBD)-Site Number:8400001
    • Colorado
      • Aurora, Colorado, United States, 80045-7202
        • Recruiting
        • University of Colorado Hemophilia and Thrombosis Center-Site Number: 8400019
    • Illinois
      • Peoria, Illinois, United States, 61614-2868
        • Recruiting
        • Bleeding and Clotting Disorders Institute-Site Number:8400005
    • Indiana
      • Indianapolis, Indiana, United States, 46260-1920
        • Recruiting
        • Indiana Hemophilia and Thrombosis Center-Site Number: 8400013
    • Massachusetts
      • Boston, Massachusetts, United States, 02114-2621
        • Recruiting
        • Massachusetts General Hospital-Site Number: 8400017
      • Boston, Massachusetts, United States, 02215-5418
        • Recruiting
        • Dana Farber and Boston Children's Hospital-Site Number: 8400031
    • Nevada
      • Las Vegas, Nevada, United States, 89135-3011
        • Recruiting
        • Alliance for Childhood Diseases-Site Number:8400007
    • Tennessee
      • Nashville, Tennessee, United States, 37232-0011
        • Recruiting
        • Vanderbilt University Medical Center Site Number : 8400003

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Approximately 200 patients from sites in the US and Japan will be enrolled in the study after the introduction of efanesoctocog alfa in the hemophilia treatment landscape in each of these two countries. To generate data for the broad real-world population of individuals with hemophilia A, eligible patients will include all ages, sexes and hemophilia A severities, whether on prophylactic or on-demand therapy or if efanesoctocog alfa is taken perioperatively.

Description

Inclusion Criteria:

  • Have a diagnosis of hemophilia A
  • Patients starting efanesoctocog alfa treatment as per standard of care no more than one month prior to the enrollment date, for either on demand or prophylaxis. Patients starting efanesoctocog alfa treatment for a surgery event may also be enrolled only if the treatment is prescribed at enrollment.
  • Physician's decision to treat the patient with efanesoctocog alfa is made prior to and independently of participation in the study.
  • Signed and dated informed consent provided by the patient, or by the patient's legally acceptable representative for patients under the legal age before any study-related activities are undertaken. Assent should be obtained for pediatric patients according to local regulations.

Exclusion Criteria:

Diagnosed with other known bleeding disorder

  • Participation in an investigational medicinal product trial at enrollment visit, or intake of an Investigational Medicinal Product within 3 months prior to inclusion in this study
  • Current diagnosis of a FVIII inhibitor, defined as inhibitor titer ≥0.60 BU/mL

"The above information is not intended to contain all considerations relevant to a potential participation in a clinical trial."

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Cohort A (Prophylactic treatment)

All participants on efanesoctocog alfa prophylactic treatment fulfilling the overall study inclusion/exclusion criteria. The prophylactic cohort will include the following sub-cohorts:

Sub-cohort A1 (Joint imaging): Participants with severe hemophilia A and joint imaging by Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) or Joint Tissue Activity and Damage Exam (JADE) protocol performed within 6 months of initiating treatment with efanesoctocog alfa or within 3 months after initiating treatment with efanesoctocog alfa available.

Sub-cohort A2 (Children with no prior joint damage):Participants with severe hemophilia A who have no prior joint damage
Drug: Efanesoctocog Alfa BIVV001
Given per investigator's discretion
Other Names:
  • ALTUVIIIO
Cohort B (On-Demand treatment)
Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria
Drug: Efanesoctocog Alfa BIVV001
Given per investigator's discretion
Other Names:
  • ALTUVIIIO

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Time Frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Up to 5 years
Change from baseline in Annualized joint bleeding rate (AjBR) for all (treated and untreated) bleeds
Time Frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic cohort
Up to 5 years
Number of Target joint development, resolution and/or recurrence
Time Frame: Up to 5 years
Data will be reported for the effectiveness of efanesoctocog alfa prophylaxis on clinical joint status over 5-years for prophylactic coho
Up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Time Frame: At 1, 2, 3, 4, 5 years
HJHS total/domain scores will be reported for prophylactic cohorts (A1 ad A2)
At 1, 2, 3, 4, 5 years
Annual Bleeding Rate (ABR) by type for treated bleeds
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by type all (treated and untreated) bleeds
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for treated bleeds
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR by location for all (treated and untreated) bleeds
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
ABR for all bleeding episodes (including untreated bleeding episodes)
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Percentage of patients with zero joint bleeds
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Treatment adherence (%) as judged by the physician during the follow-up period
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode.
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode.
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for prophylactic cohorts (Sub Cohorts A1 and A2)
At 1, 2, 3, 4, 5 years
Number of injections of efanesoctocog alfa to treat a bleeding episode
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Total dose of efanesoctocog alfa to treat a bleeding episode
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Occurrence of a change in treatment regimen (on-demand to prophylactic or prophylactic to on-demand) at baseline and follow-up.
Time Frame: At 1, 2, 3, 4, 5 years
Data will be reported for On-demand cohort
At 1, 2, 3, 4, 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) total/domain scores
Time Frame: At 1, 2, 3, 4 and 5 years
HEAD-US total/domain scores will be reported for joint imaging sub cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Time Frame: At 1, 2, 3, 4 and 5 years
Data will be reported for consenting participants from the joint imaging sub-cohort
At 1, 2, 3, 4 and 5 years
Change from baseline in synovial hypertrophy by change in mm thickness AND/OR HEAD-US synovitis domain, AND/OR by JADE MSKUS synovial hypertrophy +/- power doppler signal
Time Frame: At 6 months, 1, 2, 3, 4 and 5 years
Data will be reported from the consenting participants from the joint imaging sub-cohort
At 6 months, 1, 2, 3, 4 and 5 years
Occurrence of adverse events (AEs) and serious adverse events (SAEs)
Time Frame: Over 5 year period
The safety and tolerability of efanesoctocog alfa over 5 years evaluated in all participants
Over 5 year period
Development of inhibitors (neutralizing antibodies directed against factor FVIII as determined via the Nijmegen modified Bethesda assay.
Time Frame: Over 5 year period
The safety and tolerability of efanesoctocog alfa over 5 years assessed in all participants
Over 5 year period
Change from baseline in Patient-Reported Outcomes Measurement Information System (PROMIS) pain intensity 3a questionnaire (adults)
Time Frame: At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 3 months, 6 months, 1, 2, 3, 4 and 5 years
Change from baseline in Canadian Hemophilia Outcomes-Kids' Life Assessment Tool (CHO-KLAT) 3.0 (boys ≤18 years old) questionnaire (including Parent Proxy)
Time Frame: At 1, 2, 3, 4 and 5 years
Effectiveness of efanesoctocog alfa assessed per participants' PROs in all participants
At 1, 2, 3, 4 and 5 years
Change from baseline in the occurrence of inpatient and outpatient visits, and length of hospital stay related to hemophilia A
Time Frame: At 1, 2, 3, 4 and 5 years
Healthcare resource use (HCRU) in efanesoctocog alfa related to hemophilia A over a 5-year period
At 1, 2, 3, 4 and 5 years
Hemostatic response/physician reported during peri-operative period for surgery (major, minor) with efanesoctocog alfa.
Time Frame: Over a 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over a 5 year period
Number of injections required to maintain hemostasis during perioperative period for surgery (major, minor)
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Total efanesoctocog alfa consumption (IU) during perioperative period for surgery (major, minor)
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of blood component transfusions used during perioperative period for surgery
Time Frame: Over 5 year Period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year Period
Type of blood component transfusions used during perioperative period for surgery
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Estimated blood loss (ml) (intraoperative and post-operative period) for major surgery
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Number of transfusions required for surgery (intraoperative and post-operative period)
Time Frame: Over 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period
Over 5 year period
Duration of hospitalization (major, minor)
Time Frame: Over 5 years period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period.
Over 5 years period
Dose per injection required to maintain hemostasis during perioperative period for surgery (major, minor)
Time Frame: Over a 5 year period
Data reported to describe the usage and effectiveness of efanesoctocog alfa during the perioperative period for On-demand Cohort
Over a 5 year period

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sanofi

Investigators

  • Study Director: Clinical Sciences & Operations, Sanofi

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

June 30, 2023

Primary Completion (Estimated)

June 6, 2028

Study Completion (Estimated)

June 6, 2028

Study Registration Dates

First Submitted

May 12, 2023

First Submitted That Met QC Criteria

June 19, 2023

First Posted (Actual)

June 22, 2023

Study Record Updates

Last Update Posted (Actual)

March 8, 2024

Last Update Submitted That Met QC Criteria

March 7, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • OBS17523
  • U1111-1281-8840 (Registry Identifier: ICTRP)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, blank case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of trial participants. Further details on Sanofi's data sharing criteria, eligible studies, and process for requesting access can be found at: https://vivli.org

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Hemophilia A

Clinical Trials on Efanesoctocog Alfa BIVV001

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Montenegro

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe