Recruiting
Phase 1/2
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
Condition(s) studied
Pediatric Growth Hormone Deficiency
Investigational drug(s) / intervention(s)
GS3-007a dry suspension: GS3-007a
Placebo for GS3-007a dry suspension: Placebo for GS3-007a dry suspension
rhGH injection: rhGH injection
Study summary
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
Primary outcome measure(s)
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) — up to 21 days (Ib phase)
- Annualized height velocity (AHV) at Week 26 of treatment — 26 weeks (II phase)
- Number of Participants With TEAEs — up to 160 weeks (II phase extension)
Trial sites (1)
| Facility | City | Region | Status |
|---|
| Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology |
Wuhan |
Hubei |
Recruiting |
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