Un estudio para evaluar el impacto de efanesoctocog alfa en la salud articular a largo plazo en participantes con hemofilia A
Estudio prospectivo, observacional y multicéntrico de la eficacia de efanesoctocog alfa en la salud articular a largo plazo en pacientes con hemofilia 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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Contactos y Ubicaciones
Ubicaciones de estudio
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Arizona
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Gilbert, Arizona, Estados Unidos, 85234-2165
- Banner MD Anderson Cancer Center- Site Number : 8400008
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California
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Loma Linda, California, Estados Unidos, 92354-3450
- Loma Linda University- Site Number : 8400015
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Los Angeles, California, Estados Unidos, 90007
- Orthopedic Institute for Children- Site Number : 8400004
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Orange, California, Estados Unidos, 92868
- The Center for Inherited Blood Disorders- Site Number : 8400001
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Colorado
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Aurora, Colorado, Estados Unidos, 80045-7202
- University of Colorado Hemophilia and Thrombosis Center- Site Number : 8400019
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Florida
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Gainesville, Florida, Estados Unidos, 32610-3003
- William A. Shands Hospital at the University of Florida- Site Number : 8400032
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St. Petersburg, Florida, Estados Unidos, 33701-4804
- Johns Hopkins All Childrens Hospital - Outpatient Care Center - PIN- Site Number : 8400025
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Illinois
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Chicago, Illinois, Estados Unidos, 60612-3863
- Rush University Medical Center -1725 W Harrison St- Site Number : 8400014
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Peoria, Illinois, Estados Unidos, 61615
- The Bleeding and Clotting Disorders Institute- Site Number : 8400005
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Indiana
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Indianapolis, Indiana, Estados Unidos, 46260-1920
- Indiana Hemophilia and Thrombosis Center- Site Number : 8400013
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Iowa
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Iowa City, Iowa, Estados Unidos, 52242-1009
- University Of Iowa Hospitals And Clinics- Site Number : 8400026
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02114
- ~Massachusetts General Hospital- Site Number : 8400017
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Boston, Massachusetts, Estados Unidos, 02215-5418
- Dana Farber and Boston Children's Hospital- Site Number : 8400031
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48109-5000
- University of Michigan Hospital - 1500 E Medical Center Dr- Site Number : 8400023
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Minnesota
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Minneapolis, Minnesota, Estados Unidos, 55455-4800
- M Health Fairview- Masonic Cancer Clinic - Clinics- Site Number : 8400010
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Rochester, Minnesota, Estados Unidos, 55905-0001
- Mayo Clinic - PPDS- Site Number : 8400035
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Mississippi
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Madison, Mississippi, Estados Unidos, 39110-6115
- Mississippi Center For Advanced Medicine - 7731 Old Canton Rd- Site Number : 8400016
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Nebraska
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Omaha, Nebraska, Estados Unidos, 68114-4113
- University of Nebraska Medical Center - 985400 Nebraska Medical Center- Site Number : 8400009
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Nevada
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Las Vegas, Nevada, Estados Unidos, 89109
- Children's Specialty Center - Las Vegas- Site Number : 8400007
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New York
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New York, New York, Estados Unidos, 10065-4870
- Weill Cornell Medicine-New York Presbyterian Hospital- Site Number : 8400021
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45229-3026
- Cincinnati Children's Hospital Medical Center - PIN- Site Number : 8400006
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Pennsylvania
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Pittsburgh, Pennsylvania, Estados Unidos, 15213-4306
- Hemophilia Center of Western Pennsylvania- Site Number : 8400012
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Tennessee
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Nashville, Tennessee, Estados Unidos, 37232-0011
- Vanderbilt University Medical Center- Site Number : 8400003
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Texas
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Dallas, Texas, Estados Unidos, 75390-7202
- University of Texas Southwestern Medical Center-2001 Inwood Rd- Site Number : 8400020
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Dallas, Texas, Estados Unidos, 75390-8565
- University of Texas Southwestern Medical Center- Site Number : 8400011
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Houston, Texas, Estados Unidos, 77030
- Gulf States Hemophilia and Thrombophilia Center- Site Number : 8400029
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Washington
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Seattle, Washington, Estados Unidos, 98101-3932
- Washington Institute for Coagulation- Site Number : 8400022
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Wisconsin
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Milwaukee, Wisconsin, Estados Unidos, 53233-2121
- Blood Center of Wisconnsin- Site Number : 8400027
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
- Niño
- Adulto
- Adulto Mayor
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Método de muestreo
Población de estudio
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.
Descripción
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."
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Modelos observacionales: COHORT
- Perspectivas temporales: PROSPECTIVE
Cohortes e Intervenciones
Grupo / Cohorte |
Intervención / Tratamiento |
|---|---|
|
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 |
Given per investigator's discretion
Otros nombres:
|
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Cohort B (On-Demand treatment)
Participants receiving on-demand treatment with efanesoctocog alfa who fulfil the overall study inclusion/exclusion criteria |
Given per investigator's discretion
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Change from baseline in Annualized joint bleeding rate (AjBR) for treated bleeds
Periodo de tiempo: Up to 5 years
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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
Periodo de tiempo: 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
Periodo de tiempo: Up to 5 years
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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
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Change from baseline in the Hemophilia Joint Health Score (HJHS v2.1) total/domains scores
Periodo de tiempo: 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
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Annual Bleeding Rate (ABR) by type for treated bleeds
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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ABR by type all (treated and untreated) bleeds
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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ABR by location for treated bleeds
Periodo de tiempo: At 1, 2, 3, 4, 5 years
|
Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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ABR by location for all (treated and untreated) bleeds
Periodo de tiempo: 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)
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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Percentage of patients with zero joint bleeds
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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Annualized factor consumption per participant (IU/kg) assessed by prescription during the follow-up period.
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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Annualized injection frequency per participant (assessed by prescription) during the follow- up period
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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Treatment adherence (%) as judged by the physician during the follow-up period
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for prophylactic cohorts (A1 and A2) |
At 1, 2, 3, 4, 5 years
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Number of injections of efanesoctocog alfa to treat a bleeding episode.
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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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.
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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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
Periodo de tiempo: 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
Periodo de tiempo: At 1, 2, 3, 4, 5 years
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Data will be reported for On-demand cohort |
At 1, 2, 3, 4, 5 years
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|
Percentage of bleeding episodes treated with a single injection of efanesoctocog alfa.
Periodo de tiempo: 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.
Periodo de tiempo: 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
Periodo de tiempo: 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
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|
Change from baseline in Hemophilia Early Arthropathy Detection with Joint Tissue Activity and Damage exam (JADE) musculoskeletal ultrasound (MSKUS) (JADE MSKUS)
Periodo de tiempo: At 1, 2, 3, 4 and 5 years
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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
Periodo de tiempo: 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)
Periodo de tiempo: 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.
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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.
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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)
Periodo de tiempo: 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
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Clinical Sciences & Operations, Sanofi
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Términos MeSH relevantes adicionales
- Enfermedades Genéticas Congénitas
- Enfermedades hematológicas
- Trastornos de la coagulación de la sangre
- Trastornos hemorrágicos
- Trastornos de la coagulación de la sangre, hereditarios
- Trastornos de proteínas de coagulación
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Enfermedades hemic y linfáticas
- Hemofilia A
Otros números de identificación del estudio
- OBS17523
- U1111-1281-8840 (Identificador de registro: ICTRP)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Descripción del plan IPD
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
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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