Clinical Trials in the USA / NCT06309979
Recruiting
Observational
A Study to Assess Growth in Children With Idiopathic Short Stature
NCT06309979 · tracked via the Priya Life Science USA tracker
Condition(s) studied
Idiopathic Short Stature
Study summary
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
Eligibility
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. .
Primary outcome measure(s)
- Change in annualized growth velocity (AGV) — Every 6 months through end of study, up to 15 years
- Change in Height Z-score — Every 6 months through end of study, up to 15 years
- Change in Standing Height — Every 6 months through end of study, up to 15 years
- Change in BMI — Every 6 months through end of study, up to 15 years
- Change in BMI Z-score — Every 6 months through end of study, up to 15 years
Trial sites (38)
| Facility | City | Region | Status |
|---|---|---|---|
| Phoenix Children's Hospital - Thomas Campus (Main) | Phoenix | Arizona | Withdrawn |
| Children's Hospital Los Angeles | Los Angeles | California | Not Yet Recruiting |
| Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed) | Los Angeles | California | Recruiting |
| Children's Hospital of Orange County Main Campus - Orange | Orange | California | Not Yet Recruiting |
| Center Of Excellence in Diabetes and Endocrinology | Sacramento | California | Recruiting |
| Rady Children's Hospital - San Diego | San Diego | California | Not Yet Recruiting |
| University of California San Francisco (UCSF) | San Francisco | California | Recruiting |
| Connecticut Children's Medical Center | Hartford | Connecticut | Recruiting |
| Yale School of Medicine | New Haven | Connecticut | Withdrawn |
| Children's National Health System | Washington D.C. | District of Columbia | Recruiting |
| Nemours Children's Clinic | Jacksonville | Florida | Recruiting |
| Nicklaus Children's Hospital | Miami | Florida | Recruiting |
| Nemours Children's Health System - Corporate Headquarters | Pensacola | Florida | Recruiting |
| Centricity Research | Columbus | Georgia | Recruiting |
| St. Luke's Children's Endocrinology | Boise | Idaho | Recruiting |
| Rocky Mountain Clinical Research - Idaho Falls | Idaho Falls | Idaho | Recruiting |
| University of Louisville School Of Medicine - Norton Children's Hospital | Louisville | Kentucky | Recruiting |
| Ochsner Health Center for Children - New Orleans | New Orleans | Louisiana | Withdrawn |
| University of Michigan | Ann Arbor | Michigan | Recruiting |
| University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic | Minneapolis | Minnesota | Withdrawn |
| Dartmouth-Hitchcock Medical Center | Lebanon | New Hampshire | Recruiting |
| University of Iowa Stead Family Children's Hospital | Princeton | New Jersey | Recruiting |
| UBMD Pediatrics | Buffalo | New York | Recruiting |
| ACGC Research, NYU Ambulatory Care Garden City | Garden City | New York | Recruiting |
| New York University Grossman School of Medicine | New York | New York | Not Yet Recruiting |
| Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates | New York | New York | Not Yet Recruiting |
| Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM) | The Bronx | New York | Recruiting |
| WakeMed Children's - Pediatric Endocrinology - North Raleigh | Raleigh | North Carolina | Withdrawn |
| Cincinnati Children's Hospital Medical Center Burnet Campus | Cincinnati | Ohio | Recruiting |
| Nationwide Children's Hospital | Columbus | Ohio | Not Yet Recruiting |
| Prisma Health Children's Hospital - Midlands | Columbia | South Carolina | Recruiting |
| Vanderbilt University Medical Center | Nashville | Tennessee | Recruiting |
| University of Texas Southwestern Medical Center | Dallas | Texas | Withdrawn |
| Cook Children's Endocrinology | Fort Worth | Texas | Withdrawn |
| Baylor College of Medicine | Houston | Texas | Not Yet Recruiting |
| Seattle Children's Hospital | Seattle | Washington | Not Yet Recruiting |
| Charleston Area Medical Center (CAMC) Health System | Charleston | West Virginia | Withdrawn |
| Medical College of Wisconsin, Children's Hospital | Milwaukee | Wisconsin | Not Yet Recruiting |
More BioMarin Pharmaceutical trials in the USA
A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema
Active, not recruiting
A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)
Active, not recruiting
Other trials for the same condition
Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06309979 on ClinicalTrials.gov ↗ ← All trials in the USA