A Study to Assess Growth in Children With Idiopathic Short Stature

Официальное название

A Multicenter, Observational Study to Characterize Growth in Children With Idiopathic Short Stature

Обзор

Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.

Первичные конечные точки

• Change in annualized growth velocity (AGV) [Срок оценки: Every 6 months through end of study, up to 15 years]
• Change in Height Z-score [Срок оценки: Every 6 months through end of study, up to 15 years]
• Change in Standing Height [Срок оценки: Every 6 months through end of study, up to 15 years]
• Change in BMI [Срок оценки: Every 6 months through end of study, up to 15 years]
• Change in BMI Z-score [Срок оценки: Every 6 months through end of study, up to 15 years]

Вторичные конечные точки (4)

• Frequency of event rates of medical events of interest [Срок оценки: Every 6 months through end of study, up to 15 years]
• Frequency of Serious Medical Events [Срок оценки: Every 6 months through end of study, up to 15 years]
• Association between specific variants and growth velocity [Срок оценки: Every 6 months through end of study, up to 15 years]
• Association between other health outcomes directly related to short stature [Срок оценки: Every 6 months through end of study, up to 15 years]

Критерии участия

Критерии включения

• Participants must be > 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.
• A height assessment corresponding to a height Z-score of ≤ -2.25 SDs in reference to the general population of the same age and sex, as calculated using the Centers for Disease Control and Prevention (CDC) growth chart (https://www.cdc.gov/growthcharts/zscore.htm).
• Participants who have either never received hGH, or who are currently receiving hGH treatment.
• Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.

Критерии исключения

• Diagnosis of systemic disease or condition that may cause short stature, eg renal, neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease. Children with such diagnoses can be considered for inclusion if their condition is well controlled, at the discretion of the Medical Monitor.
• Known presence of one or more pituitary hormone deficiencies
• Bone age advanced over chronological age by more than 3 years.
• For hGH naïve participants, historic stimulation test result with serum or plasma GH level greater than 10 μg/L or serum IGF-1 in the normal range for age (between -1.00 SDs and +2.00 SDs).
• For participants currently on hGH treatment, historic results before GH treatment of stimulation test with serum or plasma GH level greater than 10 μg/L or serum IGF-1 test between -1.00 SDs and +2.00 SDs.
• Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .

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

Здоровые добровольцы: Нет

Дизайн исследования

Модель наблюдения

Когортное

Центры проведения

США · 38 центров

• Phoenix Children's Hospital - Thomas Campus (Main) — Phoenix
• Children's Hospital Los Angeles — Los Angeles
• Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Inst — Los Angeles
• Children's Hospital of Orange County Main Campus - Orange — Orange
• Center Of Excellence in Diabetes and Endocrinology — Sacramento
• Rady Children's Hospital - San Diego — San Diego
• University of California San Francisco (UCSF) — San Francisco
• Connecticut Children's Medical Center — Hartford
• … и ещё 30 центров

Идентификаторы

NCT: NCT06309979 · 111-903

Первоисточники (государственные реестры)

• ClinicalTrials.gov (NIH, США)

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