A Study to Assess Growth in Children With Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature
- Registration Number
- NCT06309979
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- All
- Target Recruitment
- 600
Inclusion Criteria:<br><br> 1. Participants must be > 2 years old, and = 14 years old (female) or = 16 years old<br> (males) at the time of signing the informed consent.<br><br> 2. A height assessment corresponding to a height Z-score of = -2.25 SDs in reference to<br> the general population of the same age and sex, as calculated using the Centers for<br> Disease Control and Prevention (CDC) growth chart<br> (https://www.cdc.gov/growthcharts/zscore.htm).<br><br> 3. Participants who have either never received hGH, or who are currently receiving hGH<br> treatment.<br><br> 4. Historic stimulation test result with serum or plasma GH level greater than 10 µg/L.<br><br> 5. Parent(s) or guardian(s) are willing and able to provide written, signed informed<br> consent.<br><br>Exclusion Criteria:<br><br> 1. Diagnosis of systemic disease or condition that may cause short stature, eg renal,<br> neoplastic, pulmonary, cardiac, gastrointestinal, immunologic and metabolic disease.<br> Children with such diagnoses can be considered for inclusion if their condition is<br> well controlled, at the discretion of the Medical Monitor.<br><br> 2. Known presence of one or more pituitary hormone deficiencies<br><br> 3. Bone age advanced over chronological age by more than 3 years.<br><br> 4. Known chromosomal imbalance or genetic variant causing short stature syndrome,<br> including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver<br> Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene<br> analysis, or Rasopathy (including Noonan syndrome).<br><br> 5. Have received an investigational product (IP) or investigational medical device for<br> any purpose within 6 months before the Screening visit. .
Not provided
Study & Design
- Study Type
- Observational
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in Height Z-score;Change in Standing Height;Change in BMI;Change in BMI Z-score;Change in annualized growth velocity (AGV)
- Secondary Outcome Measures
Name Time Method Frequency of event rates of medical events of interest;Frequency of Serious Medical Events;Association between specific variants and growth velocity;Association between other health outcomes directly related to short stature