- ICH GCP
- 미국 임상 시험 레지스트리
- 임상시험 NCT05181839
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
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
연구 개요
상세 설명
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:
- 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.
- 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.
- 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.
- Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.
연구 유형
등록 (예상)
연락처 및 위치
연구 장소
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Groningen, 네덜란드
- 모병
- University Medical Center Groningen - Beatrix Children's Hospital
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Birmingham, 영국
- 모병
- Birmingham Women's and Children's Hospital
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Bristol, 영국
- 모병
- Bristol Royal
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Liverpool, 영국
- 모병
- Alder Hay Children's Hospital
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London, 영국
- 모병
- Great Ormond Street Hospital
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Lille, 프랑스
- 모병
- Centre Hospitalier Universitair de Lille
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Lyon, 프랑스
- 모병
- Hospices Civils de Lyon
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Paris, 프랑스
- 모병
- APHP Paris - Assistance Publique Hopitaux de Paris
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참여기준
자격 기준
공부할 수 있는 나이
건강한 자원 봉사자를 받아들입니다
연구 대상 성별
샘플링 방법
연구 인구
설명
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.
공부 계획
연구는 어떻게 설계됩니까?
디자인 세부사항
- 관찰 모델: 보병대
- 시간 관점: 유망한
연구는 무엇을 측정합니까?
주요 결과 측정
결과 측정 |
측정값 설명 |
기간 |
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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.
기간: 12 Weeks
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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.
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12 Weeks
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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.
기간: 26 weeks
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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.
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26 weeks
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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.
기간: Up to 52 weeks
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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.
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Up to 52 weeks
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Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.
기간: will be completed between weeks 21 and 25
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Explore the supportive care needs and burden on carers at the time the adolescent with XLH reaches end of skeletal growth.
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will be completed between weeks 21 and 25
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공동 작업자 및 조사자
연구 기록 날짜
연구 주요 날짜
연구 시작 (실제)
기본 완료 (예상)
연구 완료 (예상)
연구 등록 날짜
최초 제출
QC 기준을 충족하는 최초 제출
처음 게시됨 (실제)
연구 기록 업데이트
마지막 업데이트 게시됨 (실제)
QC 기준을 충족하는 마지막 업데이트 제출
마지막으로 확인됨
추가 정보
이 연구와 관련된 용어
추가 관련 MeSH 약관
기타 연구 ID 번호
- 2020-70-EU-CRY
개별 참가자 데이터(IPD) 계획
개별 참가자 데이터(IPD)를 공유할 계획입니까?
약물 및 장치 정보, 연구 문서
미국 FDA 규제 의약품 연구
미국 FDA 규제 기기 제품 연구
미국에서 제조되어 미국에서 수출되는 제품
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X-연관 저인산혈증에 대한 임상 시험
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ProgenaBiome모병자폐 스펙트럼 장애 | 자폐증 | 자폐성 | 자폐증, 무력증 | 자폐성; 전형적인 아닌 | 자폐성; 사이코패스 | 자폐증 취약 증후군 X | 인지 능력이 높은 자폐증 | 자폐증 관련 언어 지연 | 자폐증, 감수성, 6 | 자폐 스펙트럼 장애 고기능 | 자폐적 사고 | 자폐증, 감수성, X-Linked 6 | 자폐증 행동미국
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VegaVect, Inc.National Eye Institute (NEI)모집하지 않고 적극적으로
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University of California, DavisNational Institute of Mental Health (NIMH)완전한
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Ramsay Générale de SantéDr Béatrice Daoud알려지지 않은
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University of California, DavisUniversity of Alberta; St. Justine's Hospital모병신경 행동 징후 | 유전 질환, X-연관 | 지적 장애 | 취약 X 증후군 | 성 염색체 장애 | 취약 X 정신 지체 증후군 | 트리뉴클레오티드 반복 확장 | 프라(X) 증후군 | FXS | 정신 지체, X 연계미국, 캐나다
Burosumab에 대한 임상 시험
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University of Alabama at BirminghamUltragenyx Pharmaceutical Inc완전한