Open-Label, Long-Term, Extension Study of Infigratinib in Children With Hypochondroplasia

Официальное название

Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE

Обзор

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

Вмешательства

• Препарат Infigratinib
  Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3.

Первичные конечные точки

• Incidence of treatment emergent adverse events (TEAE) and serious TEAE [Срок оценки: 10 years]
• Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts [Срок оценки: 10 years]

Вторичные конечные точки (12)

• Changes over time in AHV Z-score [Срок оценки: 10 years]
• Changes over time in body proportions [Срок оценки: 10 years]
• Changes over time in weight Z-score [Срок оценки: 10 years]
• Changes overtime in BMI [Срок оценки: 10 years]
• Age of puberty onset and time to Tanner stage ≥4 [Срок оценки: 10 years]
• Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans [Срок оценки: 10 years]
• Changes over time in bone morphology/density by x-ray and DXA [Срок оценки: 10 years]
• Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test) [Срок оценки: 10 years]
• Change in attention assessed by age-appropriate computerized tests (Identification Test) [Срок оценки: 10 years]
• Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test) [Срок оценки: 10 years]
• Change in working memory assessed by age-appropriate computerized tests (One Back Test) [Срок оценки: 10 years]
• Changes over time in severity of epilepsy measured by frequency and adverse event grading [Срок оценки: 10 years]

Критерии участия

Критерии включения

• Inclusion Criteria for Participants Rolling Over from ACCEL 2/3

• Pediatric participants with HCH who have completed ACCEL 2/3
• Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche

Критерии исключения

• Exclusion Criteria for Participants Rolling Over from ACCEL 2/3

• Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations
• Participants who developed a medical condition that requires the initiation of treatment with a prohibited medication
• Participants who prematurely discontinued ACCEL 2/3
• Participants who have reached final height or near final height
• Current participation in an ongoing clinical study with a sponsor other than QED

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

Здоровые добровольцы: Нет

Дизайн исследования

Распределение

Не применимо

Модель

Одна группа

Маскирование

Открытое

Основная цель

Лечение

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

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

Великобритания · 3 центра

• Manchester University — Manchester
• Sheffield Children's Hospital — Sheffield
• Glasgow Clinical Research Facility, Queen Elizabeth University Hospital — Glasgow

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: NCT07393373 · QBGJ398-205

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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