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

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05911763
• Other study ID #: OBS17523
• Secondary ID: U1111-1281-8840
• Status: Recruiting
• Start date: June 30, 2023
• Est. completion date: June 6, 2028

Study information

• Verified date: March 2024
• Source: Sanofi
• Contact: Trial Transparency email recommended (Toll free number for US &
• Phone: 1800633-1610
• Email: contact-us[@]sanofi.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 6, 2028
• Est. primary completion date: June 6, 2028
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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."

Related Conditions & MeSH terms

• Hemophilia A

Intervention

Drug:
• Efanesoctocog Alfa BIVV001
Given per investigator's discretion

Locations

Country	Name	City	State
United States	University of Colorado Hemophilia and Thrombosis Center-Site Number: 8400019	Aurora	Colorado
United States	Dana Farber and Boston Children's Hospital-Site Number: 8400031	Boston	Massachusetts
United States	Massachusetts General Hospital-Site Number: 8400017	Boston	Massachusetts
United States	Indiana Hemophilia and Thrombosis Center-Site Number: 8400013	Indianapolis	Indiana
United States	Alliance for Childhood Diseases-Site Number:8400007	Las Vegas	Nevada
United States	Orthopedic Institute for Children-Site Number: 8400004	Los Angeles	California
United States	Vanderbilt University Medical Center Site Number : 8400003	Nashville	Tennessee
United States	Center for Inherited Blood Disorders (CIBD)-Site Number:8400001	Orange	California
United States	Bleeding and Clotting Disorders Institute-Site Number:8400005	Peoria	Illinois

Sponsors (1)

Lead Sponsor	Collaborator
Sanofi

Country where clinical trial is conducted

United States, 

Outcome

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