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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05911763
Recruitment Status : Recruiting
First Posted : June 22, 2023
Last Update Posted : March 8, 2024
Sponsor:
Information provided by (Responsible Party):
Sanofi

Brief 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.


Condition or disease Intervention/treatment
Hemophilia A Drug: Efanesoctocog Alfa BIVV001

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Study Type : Observational
Estimated Enrollment : 200 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective, Observational , Multicenter Study of Effectiveness of Efanesoctocog Alfa on Long-term Joint Health in Patients With Hemophilia A
Actual Study Start Date : June 30, 2023
Estimated Primary Completion Date : June 6, 2028
Estimated Study Completion Date : June 6, 2028

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

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 Name: 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 Name: ALTUVIIIO




Primary Outcome Measures :
  1. 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

  2. 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

  3. 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


Secondary Outcome Measures :
  1. 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)

  2. 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)

  3. 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)

  4. 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)

  5. 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)

  6. 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)

  7. 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)

  8. 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)

  9. 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)

  10. 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)

  11. 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)

  12. 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)

  13. 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

  14. 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

  15. 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

  16. 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

  17. 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

  18. 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

  19. 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

  20. 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

  21. 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

  22. 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

  23. 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

  24. 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

  25. 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

  26. 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

  27. 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

  28. 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

  29. 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

  30. 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

  31. 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

  32. 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

  33. 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.

  34. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
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.
Criteria

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."


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05911763


Contacts
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Contact: Trial Transparency email recommended (Toll free number for US & Canada) 1800633-1610 ext Option 6 contact-us@sanofi.com

Locations
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United States, California
Orthopedic Institute for Children-Site Number: 8400004 Recruiting
Los Angeles, California, United States, 90007
Center for Inherited Blood Disorders (CIBD)-Site Number:8400001 Recruiting
Orange, California, United States, 92868-4306
United States, Colorado
University of Colorado Hemophilia and Thrombosis Center-Site Number: 8400019 Recruiting
Aurora, Colorado, United States, 80045-7202
United States, Illinois
Bleeding and Clotting Disorders Institute-Site Number:8400005 Recruiting
Peoria, Illinois, United States, 61614-2868
United States, Indiana
Indiana Hemophilia and Thrombosis Center-Site Number: 8400013 Recruiting
Indianapolis, Indiana, United States, 46260-1920
United States, Massachusetts
Massachusetts General Hospital-Site Number: 8400017 Recruiting
Boston, Massachusetts, United States, 02114-2621
Dana Farber and Boston Children's Hospital-Site Number: 8400031 Recruiting
Boston, Massachusetts, United States, 02215-5418
United States, Nevada
Alliance for Childhood Diseases-Site Number:8400007 Recruiting
Las Vegas, Nevada, United States, 89135-3011
United States, Tennessee
Vanderbilt University Medical Center Site Number : 8400003 Recruiting
Nashville, Tennessee, United States, 37232-0011
Sponsors and Collaborators
Sanofi
Investigators
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Study Director: Clinical Sciences & Operations Sanofi
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Responsible Party: Sanofi
ClinicalTrials.gov Identifier: NCT05911763    
Other Study ID Numbers: OBS17523
U1111-1281-8840 ( Registry Identifier: ICTRP )
First Posted: June 22, 2023    Key Record Dates
Last Update Posted: March 8, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
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

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn