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A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth

14 de enero de 2022 actualizado por: Kyowa Kirin Pharmaceutical Development Ltd

An Observational, Prospective, European, Multicentre, Mixed Methods Study to Describe the Lived Experience of X-Linked Hypophosphatemia (XLH) for Adolescents at End of Skeletal Growth

An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.

Descripción general del estudio

Estado

Reclutamiento

Condiciones

Intervención / Tratamiento

Descripción detallada

This an observational, prospective, European, multicentre, mixed methods study that will involve the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth. The study will involve two observation periods around the confirmed date of end of skeletal growth (index date)

The purpose of this study is to describe the lived experience of XLH for adolescents who are being treated with burosumab at the end of skeletal growth, with a focus on adolescent reported symptoms, activity duration and intensity, and wider burden, and to describe change over time for those who continue and discontinue burosumab at the end of skeletal growth. The study will also explore the experiences of carers at the time the adolescent reaches the end of skeletal growth.

The key objectives of this study are to:

  1. Describe the lived experience of adolescents with XLH who are being treated with burosumab within the 12 weeks prior to reaching the end of skeletal growth.
  2. Describe the lived experience of adolescents with XLH during the 26 weeks immediately after the end of skeletal growth, overall and according to whether they continue or discontinue burosumab treatment.
  3. Describe within-person changes in the lived experience of adolescents with XLH after reaching end of skeletal growth, in relation to their own pre-end of skeletal growth period.
  4. Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.

Tipo de estudio

De observación

Inscripción (Anticipado)

30

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

  • Francia
      • Lille, Francia
        • Reclutamiento
        • Centre Hospitalier Universitair de Lille
      • Lyon, Francia
        • Reclutamiento
        • Hospices Civils de Lyon
      • Paris, Francia
        • Reclutamiento
        • APHP Paris - Assistance Publique Hopitaux de Paris
  • Países Bajos
      • Groningen, Países Bajos
        • Reclutamiento
        • University Medical Center Groningen - Beatrix Children's Hospital
  • Reino Unido
      • Birmingham, Reino Unido
        • Reclutamiento
        • Birmingham Women's and Children's Hospital
      • Bristol, Reino Unido
        • Reclutamiento
        • Bristol Royal
      • Liverpool, Reino Unido
        • Reclutamiento
        • Alder Hay Children's Hospital
      • London, Reino Unido
        • Reclutamiento
        • Great Ormond Street Hospital

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

12 años a 17 años (Niño)

Acepta Voluntarios Saludables

No

Géneros elegibles para el estudio

Todos

Método de muestreo

Muestra de probabilidad

Población de estudio

The population for this study is adolescents with XLH approaching end of skeletal growth, who are being treated with burosumab for at least 12 months as part of routine clinical care at specialist paediatric centres within the UK, France, Germany, Spain and the Netherlands.

Descripción

Inclusion Criteria:

  • Confirmed diagnosis of XLH (documented diagnosis of XLH in medical records, and evidence of at least one of the following: hypophosphataemia and/or impaired phosphate reabsorption due to elevated FGF23; PHEX mutation).
  • Aged 12 to 17 years at start of study.
  • Has open growth plates at enrolment and is estimated by their treating clinician to reach end of skeletal growth within the next 26 weeks (based on clinician's judgement in accordance with their normal approach used in routine practice).
  • Has been receiving treatment with burosumab for at least study le (52 weeks).
  • Provides informed consent to take part in the study (or provides assent, and carer provides consent, where applicable in accordance with specific country regulations).

Carer Inclusion Criteria:

  • A main carer of a study participant (i.e. a parent or guardian who provides day-today support or care for the adolescent with XLH who is taking part in this study).
  • Provides informed consent to take part in the study (for self and/or on behalf of eligible adolescent, where applicable in accordance with specific country regulations).

Exclusion Criteria:

  • Unwilling and unable to participate in all aspects of the study (i.e. interviews, app, EQ- 5D-Y, wearable data collection) and /or does not agree to the collection of data from medical records.
  • Missed two or more injections of burosumab in the past 6 months.
  • Is planned to have any surgery during the study period.

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

  • Modelos observacionales: Grupo
  • Perspectivas temporales: Futuro

¿Qué mide el estudio?

Medidas de resultado primarias

Medida de resultado
Medida Descripción
Periodo de tiempo
Describe the lived experience of adolescents with XLH who are being treated with burosumab within the 12 weeks prior to reaching the end of skeletal growth.
Periodo de tiempo: 12 Weeks
Describe the lived experience of adolescents with XLH who are being treated with burosumab within the 12 weeks prior to reaching the end of skeletal growth.
12 Weeks
Describe the lived experience of adolescents with XLH during the 26 weeks immediately after the end of skeletal growth, overall and according to whether they continue or discontinue burosumab treatment.
Periodo de tiempo: 26 weeks
Describe the lived experience of adolescents with XLH during the 26 weeks immediately after the end of skeletal growth, overall and according to whether they continue or discontinue burosumab treatment.
26 weeks
Describe within-person changes in the lived experience of adolescents with XLH after reaching end of skeletal growth, in relation to their own pre-end of skeletal growth period.
Periodo de tiempo: Up to 52 weeks
Describe within-person changes in the lived experience of adolescents with XLH after reaching end of skeletal growth, in relation to their own pre-end of skeletal growth period.
Up to 52 weeks
Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.
Periodo de tiempo: will be completed between weeks 21 and 25
Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.
will be completed between weeks 21 and 25

Colaboradores e Investigadores

Aquí es donde encontrará personas y organizaciones involucradas en este estudio.

Patrocinador

Kyowa Kirin Pharmaceutical Development Ltd

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)

24 de noviembre de 2021

Finalización primaria (Anticipado)

1 de septiembre de 2023

Finalización del estudio (Anticipado)

30 de enero de 2024

Fechas de registro del estudio

Enviado por primera vez

15 de noviembre de 2021

Primero enviado que cumplió con los criterios de control de calidad

5 de enero de 2022

Publicado por primera vez (Actual)

6 de enero de 2022

Actualizaciones de registros de estudio

Última actualización publicada (Actual)

18 de enero de 2022

Última actualización enviada que cumplió con los criterios de control de calidad

14 de enero de 2022

Última verificación

1 de enero de 2022

Más información

Términos relacionados con este estudio

Términos MeSH relevantes adicionales

Otros números de identificación del estudio

  • 2020-70-EU-CRY

Plan de datos de participantes individuales (IPD)

¿Planea compartir datos de participantes individuales (IPD)?

No

Información sobre medicamentos y dispositivos, documentos del estudio

Estudia un producto farmacéutico regulado por la FDA de EE. UU.

No

Estudia un producto de dispositivo regulado por la FDA de EE. UU.

No

producto fabricado y exportado desde los EE. UU.

No

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. .

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