Summary

Eligibility
for people ages 2-16 (full criteria)
Location
at Los Angeles, California and other locations
Dates
study started
completion around

Description

Summary

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

Official Title

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

Keywords

Idiopathic Short Stature, Dwarfism

Eligibility

You can join if…

Open to people ages 2-16

  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.

You CAN'T join if...

  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. .

Locations

  • Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed) accepting new patients
    Los Angeles California 90502 United States
  • University of California San Francisco (UCSF) accepting new patients
    San Francisco California 94143 United States

Details

Status
accepting new patients
Start Date
Completion Date
(estimated)
Sponsor
BioMarin Pharmaceutical
ID
NCT06309979
Study Type
Observational
Participants
Expecting 300 study participants
Last Updated