Efanesoctocog Alfa -profilaxis en pacientes con hemofilia A con hipertrofia sinovial (SYNOVIIIUS)
Estudio intervencionista prospectivo de la efectividad de la profilaxis alfa de Efanesoctocog en la hipertrofia sinovial en pacientes con hemofilia A
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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 4
Contactos y Ubicaciones
Estudio Contacto
- Nombre: Trial Transparency email recommended (Toll free for US & Canada)
- Número de teléfono: option 6 800-633-1610
- Correo electrónico: Contact-US@sanofi.com
Ubicaciones de estudio
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Western Australia
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Murdoch, Western Australia, Australia, 6150
- Terminado
- Investigational Site Number : 0360001
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Alberta
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Calgary, Alberta, Canadá, T2N 1N4
- Reclutamiento
- Investigational Site Number : 1240002
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Newfoundland and Labrador
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St. John's, Newfoundland and Labrador, Canadá, A1B 3V6
- Reclutamiento
- Investigational Site Number : 1240001
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Ontario
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Hamilton, Ontario, Canadá, L8N 3Z5
- Reclutamiento
- Investigational Site Number : 1240003
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Hamilton, Ontario, Canadá, L8N 3Z5
- Terminado
- Investigational Site Number : 1240004
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California
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Los Angeles, California, Estados Unidos, 90007
- Terminado
- Orthopedic Institute for Children- Site Number : 8400009
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Sacramento, California, Estados Unidos, 95817-1399
- Reclutamiento
- University of California Davis Medical Center-Site Number: 8400005
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Indiana
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Indianapolis, Indiana, Estados Unidos, 46260
- Reclutamiento
- Indiana Hemophilia and Thrombosis Center- Site Number : 8400006
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Iowa
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Iowa City, Iowa, Estados Unidos, 52242
- Reclutamiento
- University of Iowa- Site Number : 8400004
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Ohio
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Columbus, Ohio, Estados Unidos, 43203
- Terminado
- The Ohio State University Wexner Medical Center - CarePoint East- Site Number : 8400001
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Oregon
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Portland, Oregon, Estados Unidos, 97239-3011
- Reclutamiento
- Oregon Health and Science University-Site Number: 8400002
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Washington
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Seattle, Washington, Estados Unidos, 98101-3932
- Reclutamiento
- Washington Center for Bleeding Disorders- Site Number : 8400008
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Hiroshima, Japón, 734-8551
- Terminado
- Investigational Site Number : 3920002
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Saitama, Japón, 330-8777
- Reclutamiento
- Investigational Site Number : 3920001
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Tokyo, Japón, 160-0023
- Terminado
- Investigational Site Number : 3920004
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Kanagawa
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Kawasaki, Kanagawa, Japón, 216-8511
- Reclutamiento
- Investigational Site Number : 3920003
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Kaohsiung City, Taiwán, 80756
- Reclutamiento
- Investigational Site Number: 1580003
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Taichung, Taiwán, 407
- Reclutamiento
- Investigational Site Number: 1580001
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Taipei, Taiwán, 114
- Reclutamiento
- Investigational Site Number: 1580002
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
Inclusion Criteria:
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Participant is diagnosed with moderate to severe hemophilia A (defined as less than or equal to (≤)5 percent (%) of endogenous FVIII clotting activity) at the time of consent/assent.
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Participant is more than or equal to (≥)12 years old at the time of consent/assent.
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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.
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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
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Participant has received prophylactic treatment with hemophilia prophylaxis prescriptions in the last 12 months prior to the baseline visit (Visit 2).
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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.
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If male, no contraceptive measures are required for this study.
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If female, is not pregnant or breastfeeding, and one of the following conditions applies:
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Is a women of nonchildbearing potential (WONCBP)
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Is a 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).
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A WOCBP must have a negative serum pregnancy test at the screening visit (Visit 1)
Exclusion Criteria:
- Participants are excluded from the study if any of the following criteria apply:
- 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.
The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: No aleatorizado
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: Efanesoctocog Alfa
Participants will receive Efanesoctocog Alfa at 50 international unit per kilogram (IU/kg) intravenous (IV) QW |
Pharmaceutical form:Lyophilized powder in a sterile vial that requires reconstitution with sterile water for injection (diluent)-Route of administration:Intravenous
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Proportion of joints with improvement in the Hemophilia Early Arthropathy Detection with Ultrasound (HEAD-US) synovitis domain score
Periodo de tiempo: From Baseline up to Week 52
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HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Change from baseline in the distribution of joint HEAD-US synovitis score
Periodo de tiempo: From Baseline up to Week 52
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HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Number of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Percentage of joints with the HEAD-US synovitis domain score that has remained unchanged or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Number of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Percentage of index joint type with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Number of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Percentage of participants with at least 1 joint with the HEAD-US synovitis domain score that has improved, remained unchanged, or worsened
Periodo de tiempo: From Baseline up to Week 52
|
HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Change from baseline in average HEAD-US synovitis domain score across all index joints
Periodo de tiempo: From Baseline up to Week 52
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HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 52
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Change from baseline in total/ domain scores of the HEAD-US
Periodo de tiempo: From Baseline up to Week 26
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HEAD-US scoring system is a simplified ultrasound scanning procedure to image the elbow, knee, and ankle (index joints) and a scoring system based on an additive scale to define the joint status from disease activity and disease damage parameters to evaluate disease progression and monitor the results of treatment. |
From Baseline up to Week 26
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Change from baseline in total/domain scores of the HJHS
Periodo de tiempo: From Baseline up to Week 26
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HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health. |
From Baseline up to Week 26
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Change from baseline in total/domain scores of the HJHS
Periodo de tiempo: From Baseline up to Week 52
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HJHS comprises an assessment of specific features, or items, of the 6 index joints and an assessment of global gait. For each of the 6 joints, the following items are scored: swelling (0 to 3), duration of swelling (0 to 1), muscle atrophy (0 to 2), crepitus on motion (0 to 2), flexion loss (0 to 3), extension loss (0 to 3), joint pain (0 to 2), and strength (0 to 4). The maximum score for an individual index joint is 20. Gait is scored from 0 to 4. The maximum HJHS total score is 124, with a higher score indicating worse joint health. |
From Baseline up to Week 52
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Change from baseline in total/ domain scores of magnetic resonance imaging (MRI)
Periodo de tiempo: From Baseline up to Week 52
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MRI will be used to detect changes in synovial hypertrophy in index joints of participants. |
From Baseline up to Week 52
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Change from baseline in patient-reported outcome (PRO)
Periodo de tiempo: From Baseline up to Week 52
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Change from baseline in PRO will be assessed by the EQ-5D 5-level (EQ-5D-5L), Patient-Reported Outcomes Measurement Information System - Short Form (PROMIS-SF) version 2.0 Pain Intensity 3a, PROMIS-SF version 1.1 Pain Interference 6a, and PROMIS-SF version 2.0 Physical Function 6b. |
From Baseline up to Week 52
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Patient reported treatment preference and satisfaction
Periodo de tiempo: At Week 52
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Patient reported treatment preference and satisfaction will be assessed through surveys and exit interviews. |
At Week 52
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Change from baseline in annualized bleeding rate (ABR) (spontaneous, traumatic)
Periodo de tiempo: At Week 52
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The ABR will be defined as (number of bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25. |
At Week 52
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Change from baseline in annualized joint bleeding rate (AjBR) (spontaneous, traumatic)
Periodo de tiempo: At Week 52
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The AjBR will be defined as (number of joint bleeding episodes occurring during the treatment period)/(duration of the treatment period [days]) × 365.25. |
At Week 52
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Change from baseline in target joint resolution, recurrence, or development
Periodo de tiempo: At Week 52
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Change from baseline in target joint resolution, recurrence, or development will be assessed. |
At Week 52
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The occurrence of Adverse Events (AEs), all AEs leading to treatment discontinuation, Serious Adverse Events (SAEs), and Adverse Event of Special Interest (AESIs)
Periodo de tiempo: Up to Week 52
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The safety and tolerability of efanesoctocog alfa evaluated in all participants |
Up to Week 52
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Colaboradores e Investigadores
Patrocinador
Publicaciones y enlaces útiles
Enlaces Útiles
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
- Bivv001
Otros números de identificación del estudio
- LPS18168
- U1111-1311-1701 (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.
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .