A Study to Assess Growth in Children With Idiopathic Short Stature

NCT06309979 | Recruiting

• 1 other identifier: 111-903
• Study Type: observational
• Target: 300
• Locations: 1 country
• Sites: 40

Brief Summary

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

Conditions

Idiopathic Short Stature

Outcome Measures

Primary Outcomes (5)

• 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

Secondary Outcomes (4)

• 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

• 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

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)
• Sampling Method: Non-Probability Sample

Study Population

The study population will include up to 600 pediatric individuals with Idiopathic Short Stature.

You may qualify if:

• Participants must be \> 2 years old, and ≤ 14 years old (female) or ≤ 16 years old (males) at the time of signing the informed consent.
• 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).
• Participants who have either never received hGH, or who are currently receiving hGH treatment.
• Historic stimulation test result with serum or plasma GH level greater than 10 μg/L.
• Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.

You may not qualify if:

• Known presence of one or more pituitary hormone deficiencies
• Bone age advanced over chronological age by more than 3 years.
• 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).
• 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.
• Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (40)

Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona, 85016, United States

RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, 90027, United States

RECRUITING

Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)

Los Angeles, California, 90502, United States

RECRUITING

Children's Hospital of Orange County Main Campus - Orange

Orange, California, 92868, United States

RECRUITING

Center Of Excellence in Diabetes and Endocrinology

Sacramento, California, 95821, United States

RECRUITING

Rady Children's Hospital - San Diego

San Diego, California, 92123, United States

RECRUITING

University of California San Francisco (UCSF)

San Francisco, California, 94143, United States

RECRUITING

Connecticut Children's Medical Center

Hartford, Connecticut, 06106-3322, United States

RECRUITING

Yale School of Medicine

New Haven, Connecticut, 06511, United States

RECRUITING

Children's National Health System

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Nemours Children's Clinic

Jacksonville, Florida, 32207, United States

RECRUITING

Nicklaus Children's Hospital

Miami, Florida, 33155, United States

RECRUITING

Nemours Children's Health System - Corporate Headquarters

Pensacola, Florida, 32514, United States

RECRUITING

Centricity Research

Columbus, Georgia, 31904, United States

RECRUITING

St. Luke's Children's Endocrinology

Boise, Idaho, 83712, United States

RECRUITING

Rocky Mountain Diabetes Center - Idaho Falls

Idaho Falls, Idaho, 83404-7596, United States

RECRUITING

Rocky Mountain Clinical Research - Idaho Falls

Idaho Falls, Idaho, 83404, United States

RECRUITING

University of Louisville School Of Medicine - Norton Children's Hospital

Louisville, Kentucky, 40202, United States

RECRUITING

Ochsner Health Center for Children - New Orleans

New Orleans, Louisiana, 70121, United States

RECRUITING

University of Michigan

Ann Arbor, Michigan, 48109-4000, United States

RECRUITING

University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic

Minneapolis, Minnesota, 55454, United States

RECRUITING

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

RECRUITING

University of Iowa Stead Family Children's Hospital

Princeton, New Jersey, 08540, United States

RECRUITING

UBMD Pediatrics

Buffalo, New York, 14203, United States

RECRUITING

ACGC Research, NYU Ambulatory Care Garden City

Garden City, New York, 11530, United States

RECRUITING

New York University Grossman School of Medicine

New York, New York, 10016, United States

RECRUITING

Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates

New York, New York, 10029, United States

RECRUITING

Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)

The Bronx, New York, 10467, United States

RECRUITING

WakeMed Children's - Pediatric Endocrinology - North Raleigh

Raleigh, North Carolina, 27615, United States

RECRUITING

Cincinnati Children's Hospital Medical Center Burnet Campus

Cincinnati, Ohio, 45229, United States

RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, 43205, United States

RECRUITING

Prisma Health Children's Hospital - Midlands

Columbia, South Carolina, 29203, United States

RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, 37232-2578, United States

RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, 75390, United States

RECRUITING

Cook Children's Endocrinology

Fort Worth, Texas, 76104, United States

RECRUITING

Baylor College of Medicine

Houston, Texas, 77030, United States

RECRUITING

Seattle Children's Hospital

Seattle, Washington, 98105, United States

RECRUITING

Charleston Area Medical Center (CAMC) Health System

Charleston, West Virginia, 25304, United States

RECRUITING

Charleston Area Medical Center (CAMC) Health System

Charleston, West Virginia, 25304, United States

RECRUITING

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, 53226, United States

RECRUITING

Central Study Contacts

Trial Specialist

CONTACT

18009834587; Medinfo@bmrn.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 7, 2024
• First Posted: March 13, 2024
• Study Start: August 8, 2024
• Primary Completion (Estimated): December 31, 2040
• Study Completion (Estimated): December 31, 2040
• Last Updated: November 14, 2025

Record last verified: 2025-11

Locations

US (40)