Study of Safety And Efficacy Of ReFacto AF In Previously Untreated Hemophilia A Patients In The Usual Care Setting
10 de mayo de 2019 actualizado por: Pfizer
AN OPEN-LABEL STUDY OF THE SAFETY AND EFFICACY OF REFACTO AF IN PREVIOUSLY UNTREATED PATIENTS IN USUAL CARE SETTINGS
Study to evaluate the safety and effectiveness of ReFacto AF for the treatment of severe hemophilia A in patients who have not yet received treatment for their hemophilia.
Study subjects will be males less than 6 years old who have not taken any clotting factor or other blood products before the study.
The safety and effectiveness of ReFacto AF will be determined in this study by tests and procedures done at the doctor's office.
Descripción general del estudio
Descripción detallada
Regulatory Commitment
Tipo de estudio
Intervencionista
Inscripción (Actual)
23
Fase
- Fase 4
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
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Bremen, Alemania, 28177
- Klinikum Bremen-Mitte gGmbH, Professor Hess Kinderklinik
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Niedersachsen
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Hannover, Niedersachsen, Alemania, 30625
- Medizinische Hochschule, Hannover
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Madrid, España, 28046
- Hospital Universitario La Paz
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Valencia, España, 46009
- Hospital Universitario La Fe
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Nantes, Francia, 44093
- CHU de Nantes
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Firenze, Italia, 50134
- Agenzia per L'emofilia e Centro Regionale Riferimento Coagulopatie Congenite
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Adana, Pavo, 01330
- Cukurova Universitesi Tip Fakultesi
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Istanbul, Pavo, 34098
- Istanbul Universitesi Cerrahpasa Tip Fakultesi
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Izmir
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Bornova, Izmir, Pavo, 35100
- Ege Universitesi Tip Fakultesi
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Groningen, Países Bajos, 9713 GZ
- University Medical Center Groningen
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Lviv, Ucrania, 79044
- Derzhavna ustanova "Instytut patolohii krovi ta transfuziinoi medytsyny Natsionalnoi akademii
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
No mayor que 6 años (Niño)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Masculino
Descripción
Inclusion Criteria:
- Male subjects <6 years of age with severe hemophilia A (FVIII:C <1%) based on clinical records, including newborns.
- No prior exposure to factor products or any blood products.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Treatment with any investigational agent or device within the past 30 days.
- Any condition(s) that compromises the ability to collect study-related observations, or that poses a contraindication to study participation (these conditions include, but are not limited to, inadequate medical history to assure study eligibility; and expectation of poor adherence to study requirements).
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Otro
- 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: 1
The investigator treats subjects with ReFacto AF in the usual care setting.
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Blood draws to determine the level of factor VIII activity before and after dosing at appropriate visits, and blood draws to determine levels of factor VIII inhibitor (antibody to factor VIII).
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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Percentage of Participants Who Developed Clinically Significant Factor VIII (FVIII) Inhibitors During the Course of the Study
Periodo de tiempo: 2 years
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Percentage of participants who developed clinically significant FVIII inhibitors: those persistent over a defined period with clinically impactful effects like breakthrough bleed, low recovery, etc., during the course of the study.
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2 years
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Annualized Bleeding Rate (ABR)
Periodo de tiempo: 2 years
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Annualized bleeding rate was calculated as the number of bleeds divided by the treatment interval duration (enrollment visit to final visit) and then multiplied by 365.25.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Total Number of Infusions to Treat a New Bleed Classified on Basis of Response to First On-Demand Treatment With Refacto AF
Periodo de tiempo: 2 years
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Number of infusions of Refacto AF required to treat a new bleed were classified on basis of the response to at 4-point response scale of assessment (excellent, good, moderate and no response).
Assessment was completed each time a participant experienced a new bleed requiring an 'on-demand' IV infusion.
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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2 years
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Total Number of Infusions Needed for Resolution of Bleeding Episodes Classified on Basis of Response to Study Drug Infusion
Periodo de tiempo: Within 48 hours after infusion, up to 2 years treatment duration
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Number of infusions of Refacto AF required for resolution of a bleeding episodes were classified on basis of the response at 4-point response scale of assessment (excellent, good, moderate and no response).
Excellent: definite pain relief and/or improvement in bleeding signs within 8 hours (hr) after infusion, no additional infusion administered; Good: definite pain relief and/or improvement in bleeding signs within 8 hr after infusion, at least 1 additional infusion administered for complete resolution or with no additional infusion administered; Moderate: probable or slight improvement starting after 8 hr following infusion, at least 1 additional infusion administered for complete resolution; No Response: no improvement at all between infusions or during 24 hr interval following infusion or condition worsen.
Bleeds for which response not recorded, reported as: Data Not Recorded.
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Within 48 hours after infusion, up to 2 years treatment duration
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Total Number of Breakthrough Bleeding Episodes Occurring Within 48 Hours After a Prophylaxis Infusion of ReFacto AF
Periodo de tiempo: 2 years
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The number of breakthrough bleeds (spontaneous or traumatic) within 48 hours following a prophylaxis dose of ReFacto AF are summarized.
If there was more than 1 bleed location (like ankle and joint) with identical bleed start date and time, it was treated as 1 bleed occurrence.
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2 years
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Consumption of Total International Units of Factor VIII
Periodo de tiempo: 2 years
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2 years
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Consumption of Total International Units of Factor VIII Per Year
Periodo de tiempo: 2 years
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Consumption of total international units of Factor VIII per year was calculated for a participant: dividing the total consumption of factor VIII by participant's treatment interval duration (in days), then multiplying by 365.25.
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion
Periodo de tiempo: 2 years
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Mean dose for each participant was calculated as participant's total factor VIII consumption (in IU) divided by the number of infusions administered.
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2 years
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Consumption of Total International Units of Factor VIII by Weight
Periodo de tiempo: 2 years
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Consumption of total international units of Factor VIII by weight was calculated for a participant: dividing the total consumption of factor VIII by participant's weight (the most recently recorded).
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2 years
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Consumption of Total International Units of Factor VIII Per Year by Weight
Periodo de tiempo: 2 years
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Consumption of total international units of Factor VIII per year by weight was calculated for a participant: the total consumption of factor VIII divided by participant's treatment interval duration (in days), then multiplying by 365.25 and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean Dose (IU) of Study Drug Consumed Per Infusion by Weight
Periodo de tiempo: 2 years
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Mean dose for each participant was calculated as participant's total factor consumption (in IU) divided by the number of infusions administered and then dividing by participant's weight (the most recently recorded).
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2 years
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Mean of Total Number of Infusions of Study Drug Received
Periodo de tiempo: 2 years
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2 years
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Mean of Total Number of Days Participants Exposed to Study Drug
Periodo de tiempo: 2 years
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2 years
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Number of Participants Who Required Dose Escalation of Their Prescribed Prophylaxis Regimen During Their Participation in This Study
Periodo de tiempo: 2 years
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The number of participants who met the dose escalation criteria were prescribed a higher dose and/or were prescribed more frequent doses.
When dose escalation was required, the specific dose and dosing schedule was at the investigator's discretion.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in On-Demand (OD) Setting
Periodo de tiempo: 2 years
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LETE occurs in OD setting if participant recorded 2 successive "No Response" (no improvement at all between infusions, or condition worsens) ratings after 2 successive infusions of study drug.
Infusions must have been given within 24 hours (hr) of each other for treatment of same bleeding event in absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate dose for type and/or severity of bleed in opinion of investigator, delay of greater than (>) 4 hr between onset of bleed to infusion, delay of >24 hr before administration of a follow-up infusion, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for bleed in opinion of investigator, ongoing trauma responsible for continued bleeding.
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2 years
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Percentage of Bleeding Episodes With Less-Than-Expected Therapeutic Effect (LETE) in the Prophylaxis Setting
Periodo de tiempo: 2 years
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LETE in prophylaxis setting if there was a spontaneous bleed within 48 hours after a regularly scheduled prophylactic dose of study drug (which was not used to treat a bleed) in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known inadequate prophylactic dose [a dose less than that prescribed in participant's regimen], known lack of adherence to the prescribed prophylaxis regimen, known compromised study drug, faulty administration of study drug, participant had an underlying, predisposing condition responsible for the bleed in the opinion of the investigator, traumatic injury responsible for bleeding.
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2 years
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Total Number of Events of Potential Less-Than-Expected Therapeutic Effect (LETE) in the Low Recovery Setting
Periodo de tiempo: 2 years
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LETE was lower than expected recovery of FVIII in the opinion of the investigator following infusion of study drug in the absence of confounding factors (known presence or subsequent identification of a FVIII inhibitor, known compromised study drug, faulty administration of study drug including inadequate dosing).
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2 years
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio (Actual)
10 de febrero de 2010
Finalización primaria (Actual)
24 de noviembre de 2016
Finalización del estudio (Actual)
24 de noviembre de 2016
Fechas de registro del estudio
Enviado por primera vez
29 de julio de 2009
Primero enviado que cumplió con los criterios de control de calidad
30 de julio de 2009
Publicado por primera vez (Estimar)
31 de julio de 2009
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
19 de julio de 2019
Última actualización enviada que cumplió con los criterios de control de calidad
10 de mayo de 2019
Última verificación
1 de mayo de 2019
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- 3082B2-4434
- B1831006 (Otro identificador: Alias Study Number)
- 2008-008436-93 (Número EudraCT)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
SÍ
Descripción del plan IPD
Pfizer will provide access to individual de-identified participant data and related study documents (e.g.
protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions.
Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests.
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. .