Assessing Growth in Children with Short Stature

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

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.

Why it matters

Who can join: the full rules

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 it's running

• Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
  Los Angeles, California, United States Recruiting
• Center Of Excellence in Diabetes and Endocrinology
  Sacramento, California, United States Recruiting
• University of California San Francisco (UCSF)
  San Francisco, California, United States Recruiting
• Connecticut Children's Medical Center
  Hartford, Connecticut, United States Recruiting
• Children's National Health System
  Washington D.C., District of Columbia, United States Recruiting
• Nemours Children's Clinic
  Jacksonville, Florida, United States Recruiting

Study contacts

• Study coordinator: Trial Specialist
• Email: Medinfo@bmrn.com
• Phone: 18009834587

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Long-term Inpegsomatropin Treatment for Idiopathic Short Stature: Extension of Phase III Study

  This extension study tests whether continuing Inpegsomatropin injections helps kids with Idiopathic Short Stature who finished a prior phase 3 trial.

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• Once-weekly lonapegsomatropin versus daily somatropin for children and adolescents with short stature due to growth-hormone-sufficient conditions

  It will test whether a once-weekly injection of lonapegsomatropin helps children and teens with Turner syndrome, SHOX mutation, small-for-gestational-age, or idiopathic short stature grow as well as or better than daily somatropin.

  Recruiting at 56 sites Phase 3 Ascendis Pharma A/S · Phoenix, Arizona and 55 other locations · NCT07221851

• Once-weekly inpegsomatropin versus daily recombinant growth hormone for children with idiopathic short stature

  This trial will test whether a once-weekly inpegsomatropin injection works as well and is as safe as daily recombinant human growth hormone in prepubertal children with idiopathic short stature.

  Recruiting at 38 sites Phase 3 Xiamen Amoytop Biotech Co., Ltd. · Hefei, Anhui and 37 other locations · NCT06927310

• Evaluating Vosoritide for Children with Short Stature

  This study is testing a new treatment called vosoritide to see if it helps children with short stature grow better than the standard growth hormone treatment.

  Recruiting at 43 sites Phase 2 BioMarin Pharmaceutical · California City, California and 46 other locations · NCT06382155

• Multidisciplinary evaluation and whole-genome testing for children with unexplained short stature

  We will try whole-genome sequencing plus a team-based clinical review to find genetic causes of unexplained short stature in children aged 4–18.

  Recruiting at 4 sites University Hospital, Montpellier · Le Kremlin-Bicêtre and 3 other locations · NCT05858606

• Evaluating the long-term safety and effectiveness of Growtropin®-II in children with short stature

  This study is testing whether the Growtropin®-II treatment is safe and effective for children with short stature caused by various conditions, to see how it helps them grow and stay healthy over time.

  Recruiting Dong-A ST Co., Ltd. · Seoul, Seodaemun-gu · NCT04798690

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.