Overview
This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for 14 days. This part is double-blinded, meaning neither the participants nor the doctors will know who is receiving the treatment or placebo. The goal is to find a safe and well-tolerated dose.
In the second part, participants will be randomly assigned to receive either the selected dose of GS3-007a or another approved treatment for 52 weeks. This part is open-label, so everyone will know which treatment is being given. After that, all participants may continue taking GS3-007a for another 156 weeks in an extension phase to study long-term effects.
Eligibility
Inclusion Criteria:
- chronological age (CA) ≥3 years at Screening
- Prepubertal girls or boys
- Height at Screening lower than the reference height for normal children of the same chronological age and sex minus 2 standard deviations (-2 SD)
- A confirmed diagnosis of growth hormone deficiency (GHD)
- Having not been treated with any growth-promoting drugs
- BA delayed by ≥6 months compared with the CA at Screening Subjects who meet all of the following criteria are eligible to enroll in the extension study (applicable for Part II extension period)
- Subjects who have completed the 52-week treatment period of phase II
- Subjects who do not permanently discontinue the investigational Medicinal Product (IMP) during the 52-week treatment period of phase II
Exclusion Criteria:
- A highly allergic constitution
- Suspected or confirmed total pituitary deficiency, including patients previously confirmed with deficiency of ≥2 pituitary hormones other than GH
- Being confirmed with other chromosomal abnormalities or growth abnormalities affecting growth
- Congenital skeletal dysplasia or serious spinal anomalies
- Cognitive hypofunction, neurodevelopmental disorders, or psychiatric/psychological disorders that, in the investigator's opinion, may interfere with evaluation of study endpoints
- Any clinically significant abnormality that may affect growth or evaluation of the IMP
- Screening magnetic resonance imaging (MRI) scan of the sellar region confirming prior or current intracranial tumor growth
- Concurrent use of any medications that may affect growth or response to growth hormone therapy
- Epiphyseal closure
- Electrocardiogram (ECG) QTcF interval abnormal, with a history of QT/QTc interval prolonged
- Hepatic function indicators abnormal at Screening Patients meeting any of the following criteria may not be enrolled in this extension study (applicable for Part II extension period)
- Subjects with closed epiphyses
- Any clinically significant abnormality that may affect growth or evaluation of the IMP
- Known or suspected allergy to the IMP
- Women with positive blood human chorionic gonadotropin (hCG) at the pre-treatment visit
