A Study to Assess Growth in Children With Idiopathic Short Stature

November 13, 2025 updated by: BioMarin Pharmaceutical

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

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

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
300

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85016
        • Recruiting
        • Phoenix Children's Hospital - Thomas Campus (Main)
        • Contact:
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
      • Los Angeles, California, United States, 90502
        • Recruiting
        • Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
        • Contact:
      • Orange, California, United States, 92868
        • Recruiting
        • Children's Hospital of Orange County Main Campus - Orange
        • Contact:
      • Sacramento, California, United States, 95821
      • San Diego, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital - San Diego
        • Contact:
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California San Francisco (UCSF)
        • Contact:
    • Connecticut
      • Hartford, Connecticut, United States, 06106-3322
      • New Haven, Connecticut, United States, 06511
        • Recruiting
        • Yale School of Medicine
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
    • Florida
      • Jacksonville, Florida, United States, 32207
        • Recruiting
        • Nemours Children's Clinic
        • Contact:
      • Miami, Florida, United States, 33155
      • Pensacola, Florida, United States, 32514
        • Recruiting
        • Nemours Children's Health System - Corporate Headquarters
        • Contact:
    • Georgia
      • Columbus, Georgia, United States, 31904
    • Idaho
      • Boise, Idaho, United States, 83712
        • Recruiting
        • St. Luke's Children's Endocrinology
        • Contact:
      • Idaho Falls, Idaho, United States, 83404
        • Recruiting
        • Rocky Mountain Clinical Research - Idaho Falls
        • Contact:
      • Idaho Falls, Idaho, United States, 83404-7596
        • Recruiting
        • Rocky Mountain Diabetes Center - Idaho Falls
        • Contact:
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • University of Louisville School Of Medicine - Norton Children's Hospital
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70121
        • Recruiting
        • Ochsner Health Center for Children - New Orleans
        • Contact:
    • Michigan
      • Ann Arbor, Michigan, United States, 48109-4000
        • Recruiting
        • University of Michigan
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55454
        • Recruiting
        • University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic
        • Contact:
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
    • New Jersey
      • Princeton, New Jersey, United States, 08540
        • Recruiting
        • University of Iowa Stead Family Children's Hospital
        • Contact:
    • New York
      • Buffalo, New York, United States, 14203
        • Recruiting
        • UBMD Pediatrics
        • Contact:
      • Garden City, New York, United States, 11530
        • Recruiting
        • ACGC Research, NYU Ambulatory Care Garden City
        • Contact:
      • New York, New York, United States, 10016
        • Recruiting
        • New York University Grossman School of Medicine
        • Contact:
      • New York, New York, United States, 10029
        • Recruiting
        • Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates
        • Contact:
      • The Bronx, New York, United States, 10467
        • Recruiting
        • Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
        • Contact:
    • North Carolina
      • Raleigh, North Carolina, United States, 27615
        • Recruiting
        • WakeMed Children's - Pediatric Endocrinology - North Raleigh
        • Contact:
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center Burnet Campus
        • Contact:
      • Columbus, Ohio, United States, 43205
    • South Carolina
      • Columbia, South Carolina, United States, 29203
    • Tennessee
      • Nashville, Tennessee, United States, 37232-2578
        • Recruiting
        • Vanderbilt University Medical Center
        • Contact:
    • Texas
      • Dallas, Texas, United States, 75390
      • Fort Worth, Texas, United States, 76104
      • Houston, Texas, United States, 77030
        • Recruiting
        • Baylor College of Medicine
        • Contact:
    • Washington
    • West Virginia
      • Charleston, West Virginia, United States, 25304
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Medical College of Wisconsin, Children's Hospital
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

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

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Change in annualized growth velocity (AGV)
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Change in Height Z-score
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Change in BMI
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Change in BMI Z-score
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Change in Standing Height
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Frequency of event rates of medical events of interest
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Frequency of Serious Medical Events
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Association between specific variants and growth velocity
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years
Association between other health outcomes directly related to short stature
Time Frame: Every 6 months through end of study, up to 15 years
Every 6 months through end of study, up to 15 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
BioMarin Pharmaceutical

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 8, 2024

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2040

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2040

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 7, 2024

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 7, 2024

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 13, 2024

Study Record Updates

Last Update Posted (Estimated)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
November 14, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 13, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
November 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 111-903

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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