Idiopathic Short StatureNCT06309979
A Study to Assess Growth in Children With Idiopathic Short Stature
An observational study of Idiopathic Short Stature, run by BioMarin Pharmaceutical at 38 sites in United States.
Trial summary
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest. Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Idiopathic Short Stature
- How many people
- 300 participants
- Where
- 38 sitesUnited States
- Study dates
- 8 Aug 2024to 31 Dec 2040
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 16 years
- All sexes
- No healthy volunteers
- Child
Study population: The study population will include up to 600 pediatric individuals with Idiopathic Short Stature.
Who may be able to join
- 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.
Who cannot join
- 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.
- 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. .
Trial locations and contacts
38 sites in 1 country. Contact the study team or a site near you to ask about taking part.
38 sites in 1 country
United States38 sites, 30 recruiting
- Recruiting
University of Michigan
Ann Arbor, Michigan, 48109-4000
Inas H Thomas, 734-674-5175, inash@med.umich.edu
- Recruiting
St. Luke's Children's Endocrinology
Boise, Idaho, 83712
Dr Daniel Flynn, 208-381-7340, flynnd@slhs.org
- Recruiting
UBMD Pediatrics
Buffalo, New York, 14203
Dr Teresa Quattrin, 716-323-0170, tquattrin@upa.chob.edu
- Withdrawn
Charleston Area Medical Center (CAMC) Health System
Charleston, West Virginia, 25304
- Recruiting
Cincinnati Children's Hospital Medical Center Burnet Campus
Cincinnati, Ohio, 45229
Dr Gajanthan Muthuvel, 513-803-4678, Gajanthan.muthuvel@cchmc.org
- Recruiting
Prisma Health Children's Hospital - Midlands
Columbia, South Carolina, 29203
Alison Lunsford, 803-434-7990, alison.lunsford@prismahealth.org
- Recruiting
Centricity Research
Columbus, Georgia, 31904
Dr Steven Bruce Leichter, 706-996-2921, sleichter@centricityresearch.com
- Not yet recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Sasigarn Bowden, 614-722-4118, sasigarn.bowden@nationwidechildrens.org
- Withdrawn
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
- Withdrawn
Cook Children's Endocrinology
Fort Worth, Texas, 76104
- Recruiting
ACGC Research, NYU Ambulatory Care Garden City
Garden City, New York, 11530
Dr Paul Saengar, 516-969-6120, Paul.Saenger@nyulangone.org
- Recruiting
Connecticut Children's Medical Center
Hartford, Connecticut, 06106-3322
Nordie Bilbao, 860-837-6700, nbilbao@connecticutchildrens.org
- Not yet recruiting
Baylor College of Medicine
Houston, Texas, 77030
Jennifer Bell, 713-798-4951, jennifer.bell@bcm.edu
- Recruiting
Rocky Mountain Clinical Research - Idaho Falls
Idaho Falls, Idaho, 83404
Dr Joshua Smith, 208-522-6005, jms-research@idahomed.com
- Recruiting
Nemours Children's Clinic
Jacksonville, Florida, 32207
Dr Larry Fox, 904-697-3837, larry.fox@nemours.org
- Recruiting
Dartmouth-Hitchcock Medical Center
Lebanon, New Hampshire, 03756
Robert Gensure, (603) 695-2790, robert.c.gensure@hitchcock.org
- Not yet recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Anna Ryabets-Lienhard, 323-361-6478, aryabets@chla.usc.edu
- Recruiting
Harbor-UCLA Medical Center - The Lundquist Institute (Los Angeles Biomedical Research Institute (LA BioMed)
Los Angeles, California, 90502
Dr Lynda Elizabeth Polgreen, 310 222-1961, lpolgreen@lundquist.org
- Recruiting
University of Louisville School Of Medicine - Norton Children's Hospital
Louisville, Kentucky, 40202
Dr Catalina Cabrera, 502-629-6000, catalina.salcedo@louisville.edu
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Dr Adriana Carrillo, 305-298-7553, Adriana.Carrillo@Nicklaushealth.org
- Not yet recruiting
Medical College of Wisconsin, Children's Hospital
Milwaukee, Wisconsin, 53226
Donald Basel, (414) 266-1616, dbasel@mcw.edu
- Withdrawn
University of Minnesota Medical School - Masonic Children's Hospital - Pediatric Specialty Care Explorer Clinic
Minneapolis, Minnesota, 55454
- Recruiting
Vanderbilt University Medical Center
Nashville, Tennessee, 37232-2578
Scott Ward, 615-936-5475, scott.k.ward@vumc.org
- Withdrawn
Yale School of Medicine
New Haven, Connecticut, 06511
- Withdrawn
Ochsner Health Center for Children - New Orleans
New Orleans, Louisiana, 70121
- Not yet recruiting
New York University Grossman School of Medicine
New York, New York, 10016
Emily Breidbart, (212) 263-5290, emily.breidbart@nyulangone.org
- Not yet recruiting
Icahn School of Medicine at Mount Sinai (ISMMS) - The Mount Sinai Hospital (MSH) - Pediatric Associates
New York, New York, 10029
Dr Robert Rapaport, 212.241.6936, robert.rapaport@mountsinai.org
- Not yet recruiting
Children's Hospital of Orange County Main Campus - Orange
Orange, California, 92868
Amrit Bhangoo, 714-509-8634, abhangoo@choc.org
- Recruiting
Nemours Children's Health System - Corporate Headquarters
Pensacola, Florida, 32514
Dr Jean-Claude Desmangles, 850-505-4791, jean-claude.desmangles@nemours.org
- Withdrawn
Phoenix Children's Hospital - Thomas Campus (Main)
Phoenix, Arizona, 85016
- Recruiting
University of Iowa Stead Family Children's Hospital
Princeton, New Jersey, 08540
Michael Tansey, (319) 356-4443, michael-tansey@uiowa.edu
- Withdrawn
WakeMed Children's - Pediatric Endocrinology - North Raleigh
Raleigh, North Carolina, 27615
- Recruiting
Center Of Excellence in Diabetes and Endocrinology
Sacramento, California, 95821
Dr Gnanagurudasan Prakasam, 916-570-2756, Gnanagurudasan.Prakasam@sutterhealth.org
- Not yet recruiting
Rady Children's Hospital - San Diego
San Diego, California, 92123
Mary (Marybeth) Patterson, 858-966-4032, mpatterson2@rchsd.org
- Recruiting
University of California San Francisco (UCSF)
San Francisco, California, 94143
Dr Hannah Chesser, hannah.chesser@ucsf.edu
- Not yet recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Patricia Fechner, 206-884-1331, patricia.fechner@seattlechildrens.org
- Recruiting
Montefiore Medical Center (MMC) - The Children's Hospital at Montefiore (CHAM)
The Bronx, New York, 10467
Dr Laurie Cohen, 718-920-4664, lacohen@montefiore.org
- Recruiting
Children's National Health System
Washington D.C., District of Columbia, 20010
Dr Andrew Dauber, 202-476-1241, adauber@childrensnational.org
About this study
Official title: 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.
- Conditions
- Idiopathic Short Stature
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in annualized growth velocity (AGV)
Measured: Every 6 months through end of study, up to 15 years
Change in Height Z-score
Measured: Every 6 months through end of study, up to 15 years
Change in Standing Height
Measured: Every 6 months through end of study, up to 15 years
Change in BMI
Measured: Every 6 months through end of study, up to 15 years
Change in BMI Z-score
Measured: Every 6 months through end of study, up to 15 years
Secondary outcomes (4)
Frequency of event rates of medical events of interest
Measured: Every 6 months through end of study, up to 15 years
Frequency of Serious Medical Events
Measured: Every 6 months through end of study, up to 15 years
Association between specific variants and growth velocity
Measured: Every 6 months through end of study, up to 15 years
Association between other health outcomes directly related to short stature
Measured: Every 6 months through end of study, up to 15 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Mar 2024
- Study start8 Aug 2024
- Primary completion31 Dec 2040 (estimated)
- Study completion31 Dec 2040 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Idiopathic Short StatureStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 300 participants (estimated)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06309979
- Sponsor's protocol ID
- 111-903
- Status last verified
- Jun 2026
- First submitted
- 7 Mar 2024
- Last updated
- 23 Jun 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Jun 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Observational Study to Characterize Growth in Children With Idiopathic Short Stature
- ClinicalTrials.gov ID
- NCT06309979
- Acronym / sponsor ID
- 111-903
- Conditions
- Idiopathic Short Stature
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 300 participants (estimated)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 8 Aug 2024 (actual)
- Primary completion
- 31 Dec 2040 (estimated)
- Study completion
- 31 Dec 2040 (estimated)
- First posted
- 13 Mar 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Study 111-903 will generate baseline growth data in children with ISS by collecting growth measurements and other variables of interest.
Study design
- Observational model
- Cohort
- Time perspective
- Other
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- The study population will include up to 600 pediatric individuals with Idiopathic Short Stature.
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (6 listed)
- 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.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Change in annualized growth velocity (AGV)
Time frame: 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
Change in Standing Height
Time frame: 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
Change in BMI Z-score
Time frame: Every 6 months through end of study, up to 15 years
4 secondary outcomes
Frequency of event rates of medical events of interest
Time frame: 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
Association between specific variants and growth velocity
Time frame: 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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Jun 2026.
Related trials for Idiopathic Short Stature
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Turner Syndrome, Short Stature Homeobox Gene Mutation
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Phase 3 Study to Evaluate the Efficacy and Safety of Human Growth Hormone Injection (Sinotropin AQ) for the Treatment of Children With Idiopathic Short Stature
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A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
38 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Idiopathic Short Stature, run by BioMarin Pharmaceutical at 38 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 23 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Trial Specialist (18009834587, Medinfo@bmrn.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in annualized growth velocity (AGV) (time frame: Every 6 months through end of study, up to 15 years). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Jun 2026.