Prospective Clinical Assessment Study in Children With Hypochondroplasia

Официальное название

Prospective Clinical Assessment Study in Children With Hypochondroplasia: ACCEL

Обзор

This is a long-term, multicenter, non-interventional study of children ages 2.5 to \<17 years with hypochondroplasia (HCH).

Подробное описание

The objective is to evaluate growth, HCH-related medical complications, health-related quality of life, functional abilities and cognitive functions of study participants. Data collected will contribute to the characterization of the natural history of children with HCH. No study medication will be administered.

Первичные конечные точки

• Annualized height velocity [Срок оценки: up to 3 years]

Вторичные конечные точки (5)

• Change over time in height Z-score, upper arm to forearm ratio, and upper leg to lower leg ratio [Срок оценки: up to 3 years]
• Cognitive functions [Срок оценки: up to 3 years]
• HCH-related medical events reported as medical history or NT-AEs [Срок оценки: up to 3 years]
• HCH-related surgical procedures [Срок оценки: up to 3 years]
• Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form. [Срок оценки: up to 3 years]

Критерии участия

Критерии включения

Signed informed consent.

Aged 2.5 to <17 years at study entry.

Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.

Participants are ambulatory and able to stand without assistance.

Study participants and parent(s), guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.

Критерии исключения

Have ACH or short stature condition other than HCH.

In females, having had their menarche. Annualized height growth velocity ≤1.5 cm/year over a period ≥6 months prior to screening.

Having a clinically significant disease or condition that in view of the investigator or Sponsor will interfere with the evaluation of growth, with study participation or not be in the best interest of the participant.

Clinically significant abnormality in any laboratory test result at screening

Current evidence of corneal or retinal disorders.

Have used any other investigational or approved product or medical device for the treatment of HCH or short stature for ≥ 30 days or with the last dose <6 months before screening.

Have had regular long-term treatment (>1 month) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable).

Previous limb-lengthening surgery or guided growth surgery with plates still in place or removed within the 6 months prior to screening.

Having had a fracture of the long bones or spine within 12 months of screening.

History and/or current evidence of extensive ectopic tissue calcification.

History of malignancy.

Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation, and/or would place the participant at high risk for poor compliance with study activities or for not completing the study.

Current participation in any other ongoing clinical study with another sponsor.

Критерии приведены из реестра в оригинале (на английском). Окончательную оценку соответствия проводит исследовательский центр.

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Только случаи

Центры проведения

США · 8 центров

• UCSF Benioff Children's Hospital — Oakland
• Childrens Hospital Colorado — Aurora
• Children's National Hospital — Washington D.C.
• Johns Hopkins School of Medicine — Baltimore
• University of Missouri — Columbia
• Cincinnati Children's Hospital Medical Center — Cincinnati
• Vanderbilt University Medical Center — Nashville
• University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic — Madison

Великобритания · 4 центра

• The Portland Hospital for Women and Children — London
• Manchester University — Manchester
• Sheffield Children's NHS Foundation Trust — Sheffield
• Glasgow Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow

Канада · 3 центра

• London Health Sciences Centre - Children's Hospital of Western Ontario — London
• Children's Hospital of Eastern Ontario Research Institute — Ottawa
• Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine — Montreal

Франция · 3 центра

• Hôpital Femme Mère Enfant HCL — Bron
• Hôpital Necker-Enfants Malades — Paris
• Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants — Toulouse

Норвегия · 2 центра

• Haukeland University Hospital — Bergen
• Paediatric Clinical Research Unit at Oslo University Hospital — Oslo

Австралия · 1 центр

• Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne — Parkville

Португалия · 1 центр

• Hospital Pediátrico de Coimbra — Coimbra

Сингапур · 1 центр

• KK Women's and Children's Hospital — Singapore

Испания · 1 центр

• Unidad de Cirugía Artroscopica, Hopsital MIKS — Vitoria-Gasteiz

Швеция · 1 центр

• Astrid Lindgren Children's Hospital — Solna

Идентификаторы

NCT: NCT06410976 · QBGJ398-004

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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