A Study to Assess Growth in Children With Idiopathic Short Stature

Launched by BIOMARIN PHARMACEUTICAL · Mar 7, 2024

Keywords

ClinConnect Summary

This clinical trial, titled "A Study to Assess Growth in Children With Idiopathic Short Stature," is looking at how children who are shorter than average grow over time. Specifically, it aims to collect important growth measurements from children diagnosed with idiopathic short stature (ISS), which means their short height doesn’t have an obvious medical cause. The study is currently recruiting children aged between 2 and 14 years for girls and up to 16 years for boys. Eligible participants need to have a height that is significantly below average according to growth charts and must be willing to get their height checked and share other relevant information.

If a child is selected to participate, their growth will be monitored, and they may be required to undergo a few tests to gather more data. It's important to know that children with certain medical conditions or specific genetic syndromes that cause short stature may not be eligible for this study. The goal is to better understand growth patterns in children with ISS, which could help doctors provide better care in the future. If you have a child who might qualify, it's a good idea to talk to your healthcare provider for more information.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• • 1. Participants must be \> 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.
• • 2. 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).
• • 3. Participants who have either never received hGH, or who are currently receiving hGH treatment.
• • 4. Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
• • 5. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.
• Exclusion Criteria:
• • 1. 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.
• • 2. Known presence of one or more pituitary hormone deficiencies
• • 3. Bone age advanced over chronological age by more than 3 years.
• • 4. 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).
• • 5. 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.
• • 6. Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .