Clinical trial NCT06309979: A Study to Assess Growth in Children With Idiopathic Short Stature
Official title: A Multicenter, Observational Study to Characterize Growth in Children With Idiopathic Short Stature
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Idiopathic Short Stature
Lead sponsor: BioMarin Pharmaceutical
Sponsor class: INDUSTRY
First posted: 2024-03-13
Enrollment: 300
Sex: ALL
Minimum age (years): 2
Maximum age (years): 16
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include up to 600 pediatric individuals with Idiopathic Short Stature.

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


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. .

Primary outcomes:
measure: Change in annualized growth velocity (AGV)
timeFrame: Every 6 months through end of study, up to 15 years
measure: Change in Height Z-score
timeFrame: Every 6 months through end of study, up to 15 years
measure: Change in Standing Height
timeFrame: Every 6 months through end of study, up to 15 years
measure: Change in BMI
timeFrame: Every 6 months through end of study, up to 15 years
measure: Change in BMI Z-score
timeFrame: Every 6 months through end of study, up to 15 years
Secondary outcomes:
measure: Frequency of event rates of medical events of interest
timeFrame: Every 6 months through end of study, up to 15 years
measure: Frequency of Serious Medical Events
timeFrame: Every 6 months through end of study, up to 15 years
measure: Association between specific variants and growth velocity
timeFrame: Every 6 months through end of study, up to 15 years
measure: Association between other health outcomes directly related to short stature
timeFrame: Every 6 months through end of study, up to 15 years
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
state: California
city: Sacramento
country: United States
facility: Center Of Excellence in Diabetes and Endocrinology
state: California
city: San Francisco
country: United States
facility: University of California San Francisco (UCSF)
state: California
city: Washington
country: United States
facility: Children's National Health System
state: District of Columbia
city: Jacksonville
country: United States
facility: Nemours Children's Clinic
state: Florida
city: Miami
country: United States
facility: Nicklaus Children's Hospital
state: Florida
city: Pensacola
country: United States
facility: Nemours Children's Health System - Corporate Headquarters
state: Florida
city: Columbus
country: United States
facility: Centricity Research
state: Georgia
city: Boise
country: United States
facility: St. Luke's Children's Endocrinology
state: Idaho
city: Idaho Falls
country: United States
facility: Rocky Mountain Clinical Research - Idaho Falls
state: Idaho
city: Louisville
country: United States
facility: University of Louisville School Of Medicine - Norton Children's Hospital
state: Kentucky
city: Minneapolis
country: United States
facility: University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic
state: Minnesota
city: Bronx
country: United States
facility: Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
state: New York
city: Buffalo
country: United States
facility: UBMD Pediatrics
state: New York
city: Garden City
country: United States
facility: ACGC Research, NYU Ambulatory Care Garden City
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates
state: New York
city: Cincinatti
country: United States
facility: Cincinnati Children's Hospital Medical Center Burnet Campus
state: Ohio
city: Fort Worth
country: United States
facility: Cook Children's Endocrinology
state: Texas
city: Charleston
country: United States
facility: Charleston Area Medical Center (CAMC) Health System
state: West Virginia
Study officials:
References: