What is this study trying to find out about Idiopathic Short Stature?

This observational study aims to gather detailed growth data in children with Idiopathic Short Stature to better understand their growth patterns over time. Sponsored by BioMarin Pharmaceutical, it focuses on tracking height, growth velocity, and related health outcomes in affected children. Participants can help researchers learn more about how ISS affects growth and health development.

Who may be eligible for this Idiopathic Short Stature clinical trial?

Children between 2 and 16 years old diagnosed with Idiopathic Short Stature may be eligible. Participants must meet specific height criteria and may or may not have received human growth hormone treatment. People with certain systemic diseases or hormone deficiencies may be excluded. Please review eligibility criteria for more details.

Is there any cost to participate in this Idiopathic Short Stature clinical trial?

Study-related care, including all evaluations and data collection, is provided at no cost. However, personal travel and other expenses are not guaranteed to be covered and may vary. Participants should consider these factors when deciding to join the study.

Actively Recruiting

Age: 2Years - 16Years

All Genders

ID06309979

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

Led by BioMarin Pharmaceutical · Updated on 2025-11-14

300

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are observing children with Idiopathic Short Stature ISS to collect baseline growth data and other important health information. This observational study aims to better understand growth patterns in children diagnosed with ISS, focusing on how their height and body measurements change over time. The study is sponsored by BioMarin Pharmaceutical and involves children aged 2 to 16 years. The study does not involve any specific treatments but monitors participants regularly to track growth and related health variables. Data such as height, body mass index BMI, and growth velocity will be collected every six months. The observation period can last up to 15 years, allowing researchers to gather long-term information about growth changes and possible medical events. Participants will have their height, BMI, and growth velocity measured every six months throughout the study. Researchers will also collect information on medical events and health outcomes related to short stature. The study includes regular evaluations to monitor safety and growth progression, with total participation lasting up to 15 years. This long follow-up helps to understand how children with ISS grow over time.

CONDITIONS

Brief Title

A Study to Assess Growth in Children With Idiopathic Short Stature

Who Can Participate

Age: 2Years - 16Years

All Genders

Eligibility Criteria

You may qualify if you...

Participants must be older than 2 years and no older than 14 years for females or 16 years for males at consent signing.
Height must correspond to a height Z-score of -2.25 or lower compared to peers of the same age and sex.
Participants may have never received human growth hormone (hGH) or may currently be receiving hGH treatment.
A historic growth hormone stimulation test must show serum or plasma GH level greater than 10 µg/L.
Parent(s) or guardian(s) must be willing and able to provide signed informed consent.

You will not qualify if you...

Diagnosis of systemic diseases that may cause short stature, such as kidney, cancer, lung, heart, digestive, immune, or metabolic diseases, unless well controlled as decided by the Medical Monitor.
Presence of one or more pituitary hormone deficiencies.
Bone age advanced more than 3 years beyond chronological age.
For hGH-naive participants, historic stimulation test with serum or plasma GH level above 10 µg/L or serum IGF-1 in the normal range (-1.00 SD to +2.00 SD).
For participants currently on hGH, historic pre-treatment results with stimulation test showing serum or plasma GH level above 10 µg/L or serum IGF-1 between -1.00 SD and +2.00 SD.
Received any investigational product or medical device within 6 months before screening.

Research Team

Trial Specialist

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