A Study to Assess Growth in Children With Idiopathic Short Stature

ConditionIdiopathic Short Stature

Trial statusRecruiting

Trial phaseNot listed

Trial typeObservational

Biological sexAll

Age2-16

SponsorBioMarin Pharmaceutical

About this trial

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

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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.

Disqualifiers

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).

Trial population

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

Trial design

Design model

Cohort

Time perspective

Other

Treatments tested in this trial

Not listed

Trial groups

No trial groups listed