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

14. januar 2022 oppdatert av: 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.

Studieoversikt

Status

Rekruttering

Forhold

Intervensjon / Behandling

Detaljert beskrivelse

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.

Studietype

Observasjonsmessig

Registrering (Forventet)

30

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

  • Frankrike
      • Lille, Frankrike
        • Rekruttering
        • Centre Hospitalier Universitair de Lille
      • Lyon, Frankrike
        • Rekruttering
        • Hospices Civils de Lyon
      • Paris, Frankrike
        • Rekruttering
        • APHP Paris - Assistance Publique Hopitaux de Paris
  • Nederland
      • Groningen, Nederland
        • Rekruttering
        • University Medical Center Groningen - Beatrix Children's Hospital
  • Storbritannia
      • Birmingham, Storbritannia
        • Rekruttering
        • Birmingham Women's and Children's Hospital
      • Bristol, Storbritannia
        • Rekruttering
        • Bristol Royal
      • Liverpool, Storbritannia
        • Rekruttering
        • Alder Hay Children's Hospital
      • London, Storbritannia
        • Rekruttering
        • Great Ormond Street Hospital

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

12 år til 17 år (Barn)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Prøvetakingsmetode

Sannsynlighetsprøve

Studiepopulasjon

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.

Beskrivelse

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.

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

  • Observasjonsmodeller: Kohort
  • Tidsperspektiver: Potensielle

Hva måler studien?

Primære resultatmål

Resultatmål
Tiltaksbeskrivelse
Tidsramme
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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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.
Tidsramme: 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

Samarbeidspartnere og etterforskere

Det er her du vil finne personer og organisasjoner som er involvert i denne studien.

Sponsor

Kyowa Kirin Pharmaceutical Development Ltd

Studierekorddatoer

Disse datoene sporer fremdriften for innsending av studieposter og sammendragsresultater til ClinicalTrials.gov. Studieposter og rapporterte resultater gjennomgås av National Library of Medicine (NLM) for å sikre at de oppfyller spesifikke kvalitetskontrollstandarder før de legges ut på det offentlige nettstedet.

Studer hoveddatoer

Studiestart (Faktiske)

24. november 2021

Primær fullføring (Forventet)

1. september 2023

Studiet fullført (Forventet)

30. januar 2024

Datoer for studieregistrering

Først innsendt

15. november 2021

Først innsendt som oppfylte QC-kriteriene

5. januar 2022

Først lagt ut (Faktiske)

6. januar 2022

Oppdateringer av studieposter

Sist oppdatering lagt ut (Faktiske)

18. januar 2022

Siste oppdatering sendt inn som oppfylte QC-kriteriene

14. januar 2022

Sist bekreftet

1. januar 2022

Mer informasjon

Begreper knyttet til denne studien

Ytterligere relevante MeSH-vilkår

Andre studie-ID-numre

  • 2020-70-EU-CRY

Plan for individuelle deltakerdata (IPD)

Planlegger du å dele individuelle deltakerdata (IPD)?

Nei

Legemiddel- og utstyrsinformasjon, studiedokumenter

Studerer et amerikansk FDA-regulert medikamentprodukt

Nei

Studerer et amerikansk FDA-regulert enhetsprodukt

Nei

produkt produsert i og eksportert fra USA

Nei

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