Recruiting NCT06309979

A Study to Assess Growth in Children With Idiopathic Short Stature

— Observational Idiopathic Short Stature

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied: This is an observational study: the protocol does not assign a study treatment.
Who it may be relevant to: Registry conditions: Idiopathic Short Stature. Basic parameters: 2 years — 16 years · All.
What needs checking: Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place: United States
Next step: Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Official title

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

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

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

Secondary outcome measures (4)

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

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.

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. .

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

United States · 38 centers

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
… and 30 more centers

NCT: NCT06309979 · 111-903