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Assessing Growth in Children with Short Stature

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

Observational BioMarin Pharmaceutical · NCT06309979

This study is trying to gather information on how children with unexplained short stature grow to help improve future treatments for them.

Quick facts

Study typeObservational
Enrollment300 (estimated)
Ages2 Years to 16 Years
SexAll
SponsorBioMarin Pharmaceutical Industry-sponsored
Locations40 sites (Phoenix, Arizona and 39 other locations)
Trial IDNCT06309979 on ClinicalTrials.gov

What this trial studies

This observational study aims to collect baseline growth data in children diagnosed with idiopathic short stature (ISS). It will involve measuring growth parameters and other relevant variables to better understand growth patterns in this population. Participants will include children aged 2 to 16 years who meet specific height criteria and may or may not have received growth hormone treatment. The data gathered will help inform future research and treatment approaches for children with ISS.

Who should consider this trial

Good fit: Ideal candidates for this study are children aged 2 to 16 years with a height Z-score of -2.25 or lower.

Not a fit: Patients with short stature due to systemic diseases or conditions that are not well controlled may not benefit from this study.

Why it matters

Potential benefit: If successful, this study could provide valuable insights into the growth patterns of children with idiopathic short stature, potentially leading to improved treatment strategies.

How similar studies have performed: While this study focuses on observational data collection, similar studies assessing growth in children with ISS have shown promise in understanding growth patterns and treatment outcomes.

Eligibility criteria

Show full inclusion / exclusion 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. .

Where this trial is running

Phoenix, Arizona and 39 other locations

Study contacts

How to participate

  1. Review the eligibility criteria above with your treating physician.
  2. Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
  3. Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.
View on ClinicalTrials.gov → Find more trials like this →
Conditions Idiopathic Short Stature
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.