A Study to Assess Growth in Children With Idiopathic Short Stature

NCT ID: NCT06309979

Last Updated: 2025-11-14

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

300 participants

Study Classification

OBSERVATIONAL

Study Start Date

2024-08-08

Study Completion Date

2040-12-31

Brief Summary

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

Detailed Description

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Conditions

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Idiopathic Short Stature

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Eligibility Criteria

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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. .
Minimum Eligible Age

2 Years

Maximum Eligible Age

16 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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Phoenix Children's Hospital - Thomas Campus (Main)

Phoenix, Arizona, United States

Site Status RECRUITING

Children's Hospital Los Angeles

Los Angeles, California, United States

Site Status RECRUITING

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

Los Angeles, California, United States

Site Status RECRUITING

Children's Hospital of Orange County Main Campus - Orange

Orange, California, United States

Site Status RECRUITING

Center Of Excellence in Diabetes and Endocrinology

Sacramento, California, United States

Site Status RECRUITING

Rady Children's Hospital - San Diego

San Diego, California, United States

Site Status RECRUITING

University of California San Francisco (UCSF)

San Francisco, California, United States

Site Status RECRUITING

Connecticut Children's Medical Center

Hartford, Connecticut, United States

Site Status RECRUITING

Yale School of Medicine

New Haven, Connecticut, United States

Site Status RECRUITING

Children's National Health System

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

Site Status RECRUITING

Nemours Children's Clinic

Jacksonville, Florida, United States

Site Status RECRUITING

Nicklaus Children's Hospital

Miami, Florida, United States

Site Status RECRUITING

Nemours Children's Health System - Corporate Headquarters

Pensacola, Florida, United States

Site Status RECRUITING

Centricity Research

Columbus, Georgia, United States

Site Status RECRUITING

St. Luke's Children's Endocrinology

Boise, Idaho, United States

Site Status RECRUITING

Rocky Mountain Diabetes Center - Idaho Falls

Idaho Falls, Idaho, United States

Site Status RECRUITING

Rocky Mountain Clinical Research - Idaho Falls

Idaho Falls, Idaho, United States

Site Status RECRUITING

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

Louisville, Kentucky, United States

Site Status RECRUITING

Ochsner Health Center for Children - New Orleans

New Orleans, Louisiana, United States

Site Status RECRUITING

University of Michigan

Ann Arbor, Michigan, United States

Site Status RECRUITING

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

Minneapolis, Minnesota, United States

Site Status RECRUITING

Dartmouth-Hitchcock Medical Center

Lebanon, New Hampshire, United States

Site Status RECRUITING

University of Iowa Stead Family Children's Hospital

Princeton, New Jersey, United States

Site Status RECRUITING

UBMD Pediatrics

Buffalo, New York, United States

Site Status RECRUITING

ACGC Research, NYU Ambulatory Care Garden City

Garden City, New York, United States

Site Status RECRUITING

New York University Grossman School of Medicine

New York, New York, United States

Site Status RECRUITING

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

New York, New York, United States

Site Status RECRUITING

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

The Bronx, New York, United States

Site Status RECRUITING

WakeMed Children's - Pediatric Endocrinology - North Raleigh

Raleigh, North Carolina, United States

Site Status RECRUITING

Cincinnati Children's Hospital Medical Center Burnet Campus

Cincinnati, Ohio, United States

Site Status RECRUITING

Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status RECRUITING

Prisma Health Children's Hospital - Midlands

Columbia, South Carolina, United States

Site Status RECRUITING

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Site Status RECRUITING

University of Texas Southwestern Medical Center

Dallas, Texas, United States

Site Status RECRUITING

Cook Children's Endocrinology

Fort Worth, Texas, United States

Site Status RECRUITING

Baylor College of Medicine

Houston, Texas, United States

Site Status RECRUITING

Seattle Children's Hospital

Seattle, Washington, United States

Site Status RECRUITING

Charleston Area Medical Center (CAMC) Health System

Charleston, West Virginia, United States

Site Status RECRUITING

Charleston Area Medical Center (CAMC) Health System

Charleston, West Virginia, United States

Site Status RECRUITING

Medical College of Wisconsin, Children's Hospital

Milwaukee, Wisconsin, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Trial Specialist

Role: CONTACT

Phone: 18009834587

Email: [email protected]

Facility Contacts

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Pamela Smith

Role: primary

Anna Ryabets-Lienhard

Role: primary

Dr Lynda Elizabeth Polgreen

Role: primary

Amrit Bhangoo

Role: primary

Dr Gnanagurudasan Prakasam

Role: primary

Mary (Marybeth) Patterson

Role: primary

Dr Hannah Chesser

Role: primary

Nordie Bilbao

Role: primary

Anisha Patel

Role: primary

Dr Andrew Dauber

Role: primary

Dr Larry Fox

Role: primary

Dr Adriana Carrillo

Role: primary

Dr Jean-Claude Desmangles

Role: primary

Dr Steven Bruce Leichter

Role: primary

Dr Daniel Flynn

Role: primary

Joshua M. Smith

Role: primary

Dr Joshua Smith

Role: primary

Dr Catalina Cabrera

Role: primary

Carmen Mironovici

Role: primary

Inas H Thomas

Role: primary

Dr Nishitha Pillai

Role: primary

Robert Gensure

Role: primary

Michael Tansey

Role: primary

Dr Teresa Quattrin

Role: primary

Dr Paul Saengar

Role: primary

Emily Breidbart

Role: primary

Dr Robert Rapaport

Role: primary

Dr Laurie Cohen

Role: primary

William Lagarde

Role: primary

Dr Gajanthan Muthuvel

Role: primary

Sasigarn Bowden

Role: primary

Alison Lunsford

Role: primary

Scott Ward

Role: primary

Nadia Merchant

Role: primary

Dr Joel Steelman

Role: primary

Jennifer Bell

Role: primary

Patricia Fechner

Role: primary

Dr Sachin Bendre

Role: primary

Sachin Bendre

Role: primary

Donald Basel

Role: primary

Other Identifiers

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111-903

Identifier Type: -

Identifier Source: org_study_id