A Study to Assess Growth in Children With Idiopathic Short Stature
- Conditions
- Idiopathic Short Stature
- Registration Number
- NCT06309979
- Lead Sponsor
- BioMarin Pharmaceutical
- Brief Summary
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 300
- 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. .
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Change in BMI Every 6 months through end of study, up to 15 years Change in annualized growth velocity (AGV) Every 6 months through end of study, up to 15 years Change in BMI Z-score Every 6 months through end of study, up to 15 years Change in Standing Height Every 6 months through end of study, up to 15 years Change in Height Z-score Every 6 months through end of study, up to 15 years
- Secondary Outcome Measures
Name Time Method Association between specific variants and growth velocity Every 6 months through end of study, up to 15 years Association between other health outcomes directly related to short stature Every 6 months through end of study, up to 15 years Frequency of event rates of medical events of interest Every 6 months through end of study, up to 15 years Frequency of Serious Medical Events Every 6 months through end of study, up to 15 years
Trial Locations
- Locations (19)
Center Of Excellence in Diabetes and Endocrinology
🇺🇸Sacramento, California, United States
University of California San Francisco (UCSF)
🇺🇸San Francisco, California, United States
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Nemours Children's Health System - Corporate Headquarters
🇺🇸Pensacola, Florida, United States
Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
🇺🇸Bronx, New York, United States
ACGC Research, NYU Ambulatory Care Garden City
🇺🇸Garden City, New York, United States
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates
🇺🇸New York, New York, United States
Cook Children's Endocrinology
🇺🇸Fort Worth, Texas, United States
Charleston Area Medical Center (CAMC) Health System
🇺🇸Charleston, West Virginia, United States
Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
🇺🇸Los Angeles, California, United States
Children's National Health System
🇺🇸Washington, District of Columbia, United States
Nemours Children's Clinic
🇺🇸Jacksonville, Florida, United States
Centricity Research
🇺🇸Columbus, Georgia, United States
St. Luke's Children's Endocrinology
🇺🇸Boise, Idaho, United States
Rocky Mountain Clinical Research - Idaho Falls
🇺🇸Idaho Falls, Idaho, United States
University of Louisville School Of Medicine - Norton Children's Hospital
🇺🇸Louisville, Kentucky, United States
University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic
🇺🇸Minneapolis, Minnesota, United States
UBMD Pediatrics
🇺🇸Buffalo, New York, United States
Cincinnati Children's Hospital Medical Center Burnet Campus
🇺🇸Cincinatti, Ohio, United States