A Study to Assess Growth in Children With Idiopathic Short Stature

Recruiting

• Conditions: Idiopathic Short Stature

• Registration Number: NCT06309979
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2024-03-07
• Study First Submitted QC: 2024-03-07
• Study First Posted: 2024-03-13
• Study Start: 2024-08-08
• Status Verified: 2025-03
• Last Update Submitted: 2025-04-14
• Last Update Posted: 2025-04-17
• Primary Completion: 2040-12-31
• Study Completion: 2040-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 300

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. .

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Change in BMI	Every 6 months through end of study, up to 15 years
Change in annualized growth velocity (AGV)	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
Change in Standing Height	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

Secondary Outcome Measures

Name	Time	Method
Association between specific variants and growth velocity	Every 6 months through end of study, up to 15 years
Association between other health outcomes directly related to short stature	Every 6 months through end of study, up to 15 years
Frequency of event rates of medical events of interest	Every 6 months through end of study, up to 15 years
Frequency of Serious Medical Events	Every 6 months through end of study, up to 15 years

Trial Locations

Locations (19)

Center Of Excellence in Diabetes and Endocrinology; 🇺🇸 Sacramento, California, United States

University of California San Francisco (UCSF); 🇺🇸 San Francisco, California, United States

Nicklaus Children's Hospital; 🇺🇸 Miami, Florida, United States

Nemours Children's Health System - Corporate Headquarters; 🇺🇸 Pensacola, Florida, United States

Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM); 🇺🇸 Bronx, New York, United States

ACGC Research, NYU Ambulatory Care Garden City; 🇺🇸 Garden City, New York, United States

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates; 🇺🇸 New York, New York, United States

Cook Children's Endocrinology; 🇺🇸 Fort Worth, Texas, United States

Charleston Area Medical Center (CAMC) Health System; 🇺🇸 Charleston, West Virginia, United States

Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed); 🇺🇸 Los Angeles, California, United States

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