A Study to Assess Growth in Children With Idiopathic Short Stature

Idiopathic Short Stature Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06309979
• Other study ID #: 111-903
• Status: Recruiting
• Start date: March 25, 2024
• Est. completion date: December 31, 2040

Study information

• Verified date: March 2024
• Source: BioMarin Pharmaceutical
• Contact: Trial Specialist
• Phone: 18009834587
• Email: Medinfo[@]bmrn.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 600
• Est. completion date: December 31, 2040
• Est. primary completion date: December 31, 2040
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 16 Years

Eligibility

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. Participants whose height Z-score is = -2.5 SDs compared to age and sex matched population norms.

3. Participants are ambulatory and able to stand without assistance (not applicable for children who are < 5 years of age and less than 104 cm in length).

4. Parent(s) or guardian(s) are willing and able to provide written, signed informed consent.

5. Participants are willing and able to perform all study procedures.

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 pituitary hormone deficiencies.

3. Bone age advanced over chronological age by more than 3 years.

4. Active malignancy, active chemotherapy or radiation therapy for neoplasia.

5. Have received an investigational product (IP) or investigational medical device for any purpose within 6 months before the Screening visit.

6. Ongoing treatment for concurrent disease or condition that, in the view of the investigator, would interfere with study participation, for any reason. Participants receiving treatments for attention deficit hyperactivity disorder are not excluded.

Related Conditions & MeSH terms

• Dwarfism
• Idiopathic Short Stature

Locations

Country	Name	City	State
United States	Children's National Health System	Washington	District of Columbia

Sponsors (1)

Lead Sponsor	Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in annualized growth velocity (AGV)		Every 6 months through end of study, up to 15 years
Primary	Change in Height Z-score		Every 6 months through end of study, up to 15 years
Primary	Change in Standing Height		Every 6 months through end of study, up to 15 years
Primary	Change in BMI		Every 6 months through end of study, up to 15 years
Primary	Change in BMI Z-score		Every 6 months through end of study, up to 15 years
Secondary	Frequency of event rates of medical events of interest		Every 6 months through end of study, up to 15 years
Secondary	Frequency of Serious Medical Events		Every 6 months through end of study, up to 15 years
Secondary	Association between specific variants and growth velocity		Every 6 months through end of study, up to 15 years
Secondary	Association between other health outcomes directly related to short stature		Every 6 months through end of study, up to 15 years