Profilaxis con Fitusiran en sujetos pediátricos masculinos de 1 a menos de 12 años con hemofilia A o B (ATLAS-PEDS)
ATLAS-PEDS: un estudio multinacional abierto de profilaxis con fitusirán en sujetos pediátricos masculinos de 1 a menos de 12 años con hemofilia A o B
Primary Objective:
- To confirm appropriate dose levels of fitusiran when administered to male pediatric participants (ages 1 to <12 years of age) with severe hemophilia A or B
Secondary Objectives:
- To characterize the safety and tolerability
- To determine fitusiran plasma concentrations at selected time points
Descripción general del estudio
Descripción detallada
The estimated total time on study is up to 256 weeks for participants who roll over into the extension study and up to 280 weeks for participants who do not roll over into the extension study (due to the requirement for up to an additional 6 months of follow-up for monitoring of AT levels).
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 2
- Fase 3
Contactos y Ubicaciones
Ubicaciones de estudio
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Ontario
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Hamilton, Ontario, Canadá, L8N 3Z5
- Investigational Site Number : 1240001
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Ottawa, Ontario, Canadá, K1H 8L1
- Investigational Site Number : 1240002
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Madrid, Comunidad de
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Madrid, Madrid, Comunidad de, España, 28046
- Investigational Site Number : 7240002
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California
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Los Angeles, California, Estados Unidos, 90027
- Children's Hospital Los Angeles Site Number : 8400002
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New Jersey
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Hackensack, New Jersey, Estados Unidos, 07601
- Hackensack University Medical Center- Site Number : 8400008
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Ohio
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Cleveland, Ohio, Estados Unidos, 44106
- University Hospitals of Cleveland- Site Number : 8400007
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Pennsylvania
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Hershey, Pennsylvania, Estados Unidos, 17033
- Penn State Milton S. Hershey Medical Center- Site Number : 8400006
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Bangalore, India, 560034
- Investigational Site Number : 3560006
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Mumbai, India, 400 022
- Investigational Site Number : 3560002
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Pune-411011, India, 411 011
- Investigational Site Number : 3560001
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Vellore, India, 632004
- Investigational Site Number : 3560004
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Florence, Italia, 50134
- Investigational Site Number : 3800002
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Milan, Italia, 20122
- Investigational Site Number : 3800001
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Adana, Turquía (Türkiye), 01130
- Investigational Site Number : 7920001
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Istanbul, Turquía (Türkiye), 34093
- Investigational Site Number : 7920002
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Izmir, Turquía (Türkiye), TR-35100
- Investigational Site Number : 7920003
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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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Male, aged 1 to <12 years at the time of enrollment.
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Severe hemophilia A or B (Factor VIII (FVIII) <1% or Factor IX (FIX) ≤2%)
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Participants must have inhibitory antibodies to FVIII or FIX and must meet one of the following Nijmegen-modified Bethesda assay results criteria:
- Inhibitor titer of ≥0.6 BU/mL at screening, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, OR
- Inhibitor titer of <0.6 BU/mL at screening with medical record evidence of 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response or severe allergic reaction (anaphylaxis or nephrotic syndrome)
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Adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol
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Weight requirements at the time of enrollment: 8 to <45 kg
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Willing and able to comply with the study requirements and to provide signed written informed consent obtained from parent(s)/legal guardian (hereinafter the "parent") and written or oral assent obtained from participant, per local and national requirements
Exclusion Criteria:
Participants are excluded from the study if any of the following criteria apply:
- Known co-existing bleeding disorders other than hemophilia A or B
- Antithrombin (AT) activity <60% at Screening
- Co-existing thrombophilic disorder
- Clinically significant liver disease
- Active Hepatitis C virus infection
- Acute or chronic Hepatitis B virus infection
- Acute Hepatitis A or hepatitis E infection
- HIV positive with a CD4 count of <400 cells/μL
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Inadequate renal function
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or N-Acetylgalactosamine (GalNAc)
- Subjects with central or peripheral indwelling catheters, with history of venous access complications leading to hospitalization and/or systemic anticoagulation therapy.
- History of intolerance to subcutaneous (SC) injection(s)
- Use of emicizumab (Hemlibra®) within 6 months prior to screening
- Any other conditions or comorbidities that would make the patient unsuitable for enrollment or could interfere with participation in or completion of the study, per Investigator judgment
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: N / A
- 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: Fitusiran
Participants will receive a selected dose of fitusiran at regular intervals, as per study protocol |
Pharmaceutical form:Solution for injection-Route of administration:Subcutaneous
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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Plasma antithrombin (AT) activity levels
Periodo de tiempo: Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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Characterize the AT activity at the optimal therapeutic dose |
Day 1 to the AT analysis time point at the optimal therapeutic dose (approximately 256 weeks)
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Number of participants reported with adverse events
Periodo de tiempo: Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Number of participants reported with treatment-emergent adverse events (TEAEs) |
Up to 280 weeks (up to 256 weeks of treatment + up to 24 weeks of AT follow up)
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Fitusiran plasma concentrations
Periodo de tiempo: Day 1 and Day 85
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Plasma samples will be collected for measurement of plasma concentrations of fitusiran at specific time points post dose on Day 1 and pre dose on Day 85 |
Day 1 and Day 85
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Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Clinical Sciences & Operations, Sanofi
Publicaciones y enlaces útiles
Enlaces Útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Actual)
Finalización del estudio (Actual)
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
- Fitusiran
Otros números de identificación del estudio
- EFC15467
- 2019-000679-18 (Número EudraCT)
- U1111-1223-4368 (Identificador de registro: ICTRP)
- 2024-512501-76 (Identificador de registro: CTIS)
- 2019 (Subvención/contrato del NIH de EE. UU.)
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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