RECRUITING

A Study to Assess Growth in Children with Idiopathic Short Stature

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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

Official Title

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

Quick Facts

Study Start:2024-08-08

Study Completion:2040-12-31

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06309979

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.

Ages Eligible for Study:2 Years to 16 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:No

Standard Ages:CHILD

Inclusion Criteria	Exclusion 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.	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. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome). 5. Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .

Inclusion Criteria

Exclusion 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.

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. Known chromosomal imbalance or genetic variant causing short stature syndrome, including but not limited to Laron syndrome, Prader-Willi syndrome, Russell-Silver Syndrome, Turner syndrome, disproportionate skeletal dysplasias, abnormal SHOX gene analysis, or Rasopathy (including Noonan syndrome).
5. Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit. .

Contacts and Locations

Study Contact

Trial Specialist

CONTACT

18009834587

Medinfo@bmrn.com

Study Locations (Sites)

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

Los Angeles, California, 90502

United States

Children's National Health System

Washington, District of Columbia, 20010

United States

Nemours Children's Clinic

Jacksonville, Florida, 32207

United States

Centricity Research

Columbus, Georgia, 31904

United States

St. Luke's Children's Endocrinology

Boise, Idaho, 83712

United States

Rocky Mountain Clinical Research - Idaho Falls

Idaho Falls, Idaho, 83404

United States

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

Louisville, Kentucky, 40202

United States

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

Minneapolis, Minnesota, 55454

United States

UBMD Pediatrics

Buffalo, New York, 14203

United States

NYU Langone Diabetes & Endocrinology Associates

New York, New York, 10016

United States

Cincinnati Children's Hospital Medical Center Burnet Campus

Cincinatti, Ohio, 45229

United States

Collaborators and Investigators

Sponsor: 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 Registration Dates

Study Start Date2024-08-08

Study Completion Date2040-12-31

Study Record Updates

Study Start Date2024-08-08

Study Completion Date2040-12-31

Terms related to this study

Additional Relevant MeSH Terms

Idiopathic Short Stature