Study Evaluating The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age
20 de mayo de 2022 actualizado por: Wyeth is now a wholly owned subsidiary of Pfizer
An Open-Label Study To Evaluate The Efficacy And Safety Of Xyntha In Children Less Than 6 Years Of Age In Usual Care Settings
This study will be investigating the safety and efficacy of Xyntha (moroctocog alfa (AF-CC)) in male patients less than 6 years old.
Annualized bleeding rates and physician / caregiver assessments of responses to treatment will be characterized.
FVIII inhibitor levels will be assessed throughout the study.
Descripción general del estudio
Descripción detallada
The study was terminated on 22 Sept 2009 due to competition with another Wyeth study for a similar patient population.
The decision to terminate the trial was not based on any safety issues.
Tipo de estudio
Intervencionista
Inscripción (Actual)
1
Fase
- Fase 3
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
1 segundo a 5 años (Niño)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Masculino
Descripción
Inclusion Criteria:
- Male patients less than 6 years of age with moderately severe to severe hemophilia A (FVIII less than or equal to 2%).
- Treatment history of less than 50 exposure days to prior recombinant or plasma-derived FVIII replacement products.
- Not receiving treatment for HIV or hepatitis infection, or the patient is on a stable antiviral regimen at the time of enrollment in the study.
Exclusion Criteria:
- Presence of any bleeding disorder in addition to hemophilia A.
- Inhibitor titer of greater than or equal to 5 Bethesda Units (BU) at screening.
- Treated with immunomodulatory therapy during the screening period
- Treatment history of more than 5 exposure days (ED) to Xyntha.
- Known hypersensitivity to hamster protein.
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: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Otro: abierto
|
Patients will receive Moroctocog alfa according to their investigator's prescription.
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Percentage of Participants With Factor VIII (FVIII) Inhibitor Development
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
Incidence of inhibitor development was defined as any result determined positive at a central laboratory (Bethesda inhibitor titer of >=0.6 BU/mL) using Nijmegen modification of the Bethesda assay.
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Baseline to 24 months or early withdrawal.
|
|
Percentage of Participants With Less Than Expected Therapeutic Effects (LETE) in the On-Demand Setting
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
LETE in the on-demand setting was based on the response to the treatment of a bleeding episode.
LETE in the on-demand setting occurred if the participant recorded 2 successive "No Response" ratings (indicated there was no improvement at all between infusions or during the 24 hour interval following an infusion, or condition worsened) after 2 successive Xyntha infusions, respectively.
The infusions was to be administered within 24 hours (=<24 hours) of each other for the treatment of the same bleeding event in the absence of confounding factor.
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Baseline to 24 months or early withdrawal.
|
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Percentage of Participants With LETE in the Prophylaxis Setting
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
The LETE in the prophylaxis setting was the occurrence of a bleed.
LETE in the prophylaxis setting occurred if there was a spontaneous bleed within 48 hours (=<48 hours) after a regularly scheduled prophylactic dose of Xyntha (which was not used to treat a bleed) in the absence of confounding factors.
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Baseline to 24 months or early withdrawal.
|
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Percentage of Participants With Low Recovery LETE
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
The LETE could be considered lower than expected recovery of FVIII in the opinion of the investigator following infusion of Xyntha in the absence of confounding factors.
|
Baseline to 24 months or early withdrawal.
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Mean Annualized Bleed Rate (ABR)
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
An annualized bleeding rate (ABR) for each participant was calculated as the number of bleeds requiring administration of FVIII replacement product (taken from the electronic Infusion Log Diary), divided by his total therapy duration (in days), and then multiplied by 365.25.
|
Baseline to 24 months or early withdrawal.
|
|
Number of Xyntha Infusions Needed to Treat Each New Bleed
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
The data from the electronic Infusion Log Diary plus the Test Article case report form (CRF) was used to determine the number of infusions administered to treat a bleed.
This was calculated by adding the initial 'for a new bleed' (on demand) infusion to any subsequent (on demand) infusions for the (same) 'previously treated bleed'.
An on-demand infusion for a 'previously treated bleed' was counted toward the bleed with the most recent start time prior to that infusion.
|
Baseline to 24 months or early withdrawal.
|
|
Response to First On-demand Xyntha Treatment for All New Bleeds as Assessed by the Caregiver
Periodo de tiempo: Baseline to 24 months or early withdrawal
|
A 4-point response scale to be completed is as defined as follows: (Excellent: definite pain relief/improvement in signs of bleeding starting within 8 hrs after an infusion, with no additional infusion; Good: definite pain relief/improvement in signs of bleeding starting within 8 hrs or following the infusion; Moderate: probable/slight improvement starting after 8 hours following the infusion; No Response: no improvement at all between infusions).
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Baseline to 24 months or early withdrawal
|
|
Mean Number of Breakthrough (Spontaneous/Non-traumatic) Bleeds
Periodo de tiempo: Baseline to 24 months or early withdrawal.
|
The number of breakthrough (spontaneous/non-traumatic) bleeds within 48 hours following a prophylaxis dose of Xyntha was summarized.
The data from the electronic Infusion Log Diary plus the Test Article CRF was used to determine the number of infusions administered to treat a new bleed, counting only those infusions administered =<48 hours after an infusion marked as 'prophylaxis' (which had no associated bleed).
|
Baseline to 24 months or early withdrawal.
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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
1 de junio de 2009
Finalización primaria (Actual)
1 de diciembre de 2009
Finalización del estudio (Actual)
1 de diciembre de 2009
Fechas de registro del estudio
Enviado por primera vez
23 de septiembre de 2008
Primero enviado que cumplió con los criterios de control de calidad
23 de septiembre de 2008
Publicado por primera vez (Estimar)
25 de septiembre de 2008
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
15 de junio de 2022
Última actualización enviada que cumplió con los criterios de control de calidad
20 de mayo de 2022
Última verificación
1 de mayo de 2022
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- 3082B2-3315
- B1831002 (Otro identificador: Pfizer)
- 3082B2-3315-WW
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Ensayos clínicos sobre Moroctocog alfa
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PfizerTerminado
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PfizerTerminadoHemofilia AEstados Unidos, Argentina, Austria, Colombia, Croacia, Jordán, México, Nueva Zelanda, Omán, Perú, Polonia, Rumania, Pavo
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PfizerTerminado
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PfizerTerminadoHemofilia AReino Unido, Hungría, Bulgaria
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Wyeth is now a wholly owned subsidiary of PfizerTerminado
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PfizerTerminadoHemofilia APaíses Bajos, España, Grecia, Italia, Francia, Bélgica, Alemania, Austria, Suecia, Hungría, Finlandia, Reino Unido, Dinamarca, Rumania
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PfizerTerminado
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PfizerTerminadoHemofilia AEspaña, Finlandia, Serbia, Georgia, Italia, Rumania, Suecia, Pavo, Ucrania
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PfizerTerminadoHemofilia AEstados Unidos, Nueva Zelanda
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National Heart, Lung, and Blood Institute (NHLBI)TerminadoEnfermedad cardiovascularEstados Unidos