Actively Recruiting
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
40
Research Sites
N/A
Total Duration
On this page
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
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.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 15 years
Participants are monitored to observe changes in growth and health outcomes related to idiopathic short stature over time.
Visits every 6 months
Trial Site Locations
Total: 40 locations
1
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona, United States, 85016
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
Los Angeles, California, United States, 90502
Actively Recruiting
4
Children's Hospital of Orange County Main Campus - Orange
Orange, California, United States, 92868
Actively Recruiting
5
Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, United States, 95821
Actively Recruiting
6
Rady Children's Hospital - San Diego
San Diego, California, United States, 92123
Actively Recruiting
7
University of California San Francisco (UCSF)
San Francisco, California, United States, 94143
Actively Recruiting
8
Connecticut Children's Medical Center
Hartford, Connecticut, United States, 06106-3322
Actively Recruiting
9
Yale School of Medicine
New Haven, Connecticut, United States, 06511
Actively Recruiting
10
Children's National Health System
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
11
Nemours Children's Clinic
Jacksonville, Florida, United States, 32207
Actively Recruiting
12
Nicklaus Children's Hospital
Miami, Florida, United States, 33155
Actively Recruiting
13
Nemours Children's Health System - Corporate Headquarters
Pensacola, Florida, United States, 32514
Actively Recruiting
14
Centricity Research
Columbus, Georgia, United States, 31904
Actively Recruiting
15
St. Luke's Children's Endocrinology
Boise, Idaho, United States, 83712
Actively Recruiting
16
Rocky Mountain Diabetes Center - Idaho Falls
Idaho Falls, Idaho, United States, 83404-7596
Actively Recruiting
17
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, United States, 83404
Actively Recruiting
18
University of Louisville School Of Medicine - Norton Children's Hospital
Louisville, Kentucky, United States, 40202
Actively Recruiting
19
Ochsner Health Center for Children - New Orleans
New Orleans, Louisiana, United States, 70121
Actively Recruiting
20
University of Michigan
Ann Arbor, Michigan, United States, 48109-4000
Actively Recruiting
21
University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic
Minneapolis, Minnesota, United States, 55454
Actively Recruiting
22
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, United States, 03756
Actively Recruiting
23
University of Iowa Stead Family Children's Hospital
Princeton, New Jersey, United States, 08540
Actively Recruiting
24
UBMD Pediatrics
Buffalo, New York, United States, 14203
Actively Recruiting
25
ACGC Research, NYU Ambulatory Care Garden City
Garden City, New York, United States, 11530
Actively Recruiting
26
New York University Grossman School of Medicine
New York, New York, United States, 10016
Actively Recruiting
27
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates
New York, New York, United States, 10029
Actively Recruiting
28
Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
The Bronx, New York, United States, 10467
Actively Recruiting
29
WakeMed Children's - Pediatric Endocrinology - North Raleigh
Raleigh, North Carolina, United States, 27615
Actively Recruiting
30
Cincinnati Children's Hospital Medical Center Burnet Campus
Cincinnati, Ohio, United States, 45229
Actively Recruiting
31
Nationwide Children's Hospital
Columbus, Ohio, United States, 43205
Actively Recruiting
32
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, United States, 29203
Actively Recruiting
33
Vanderbilt University Medical Center
Nashville, Tennessee, United States, 37232-2578
Actively Recruiting
34
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
35
Cook Children's Endocrinology
Fort Worth, Texas, United States, 76104
Actively Recruiting
36
Baylor College of Medicine
Houston, Texas, United States, 77030
Actively Recruiting
37
Seattle Children's Hospital
Seattle, Washington, United States, 98105
Actively Recruiting
38
Charleston Area Medical Center (CAMC) Health System
Charleston, West Virginia, United States, 25304
Actively Recruiting
39
Charleston Area Medical Center (CAMC) Health System
Charleston, West Virginia, United States, 25304
Actively Recruiting
40
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
Research Team
T
Trial Specialist
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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