A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: GS3-007a dry suspension, Placebo for GS3-007a dry suspension, rhGH injection.
- Who it may be relevant to
- Registry conditions: Pediatric Growth Hormone Deficiency. Basic parameters: from 3 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Study to Evaluate the Safety and Tolerability of Multiple Ascending Doses of GS3-007a Dry Suspension in Patients With PGHD and to Evaluate the Efficacy and Safety of 52-week of Treatment With GS3-007a Dry Suspension: a Two-part, Multicenter, Randomized, Phase Ib/II Clinical Trial
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.
Interventions
- Drug GS3-007a dry suspension
GS3-007a - Drug Placebo for GS3-007a dry suspension
Placebo for GS3-007a dry suspension - Drug rhGH injection
rhGH injection
Primary outcome measures
- Number of Participants With Treatment Emergent Adverse Events (TEAEs) [Time frame: up to 21 days (Ib phase)]
- Annualized height velocity (AHV) at Week 26 of treatment [Time frame: 26 weeks (II phase)]
- Number of Participants With TEAEs [Time frame: up to 160 weeks (II phase extension)]
Secondary outcome measures (6)
- PK concentrations of GS3-007a and metabolite GS3-017 [Time frame: up to 7 days (Ib phase)]
- Concentrations of serum growth hormone (GH), insulin-like growth factor-1 (IGF-1), and insulin-like growth factor-binding protein 3 (IGFBP-3) [Time frame: up to 14 days (Ib phase)]
- AHVs at each evaluation point [Time frame: 13 weeks, 39 weeks, 52 weeks (II phase)]
- Changes from baseline (ΔIGF-1 SDS) in the standard deviation score of the PD indicator insulin-like growth factor-1 (IGF-1 SDS) at each evaluation point [Time frame: 4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)]
- AHVs at each evaluation point [Time frame: up to 156 weeks (II phase extension)]
- Change from extension baseline in the height standard deviation score at each evaluation point (ΔHT SDS) [Time frame: up to 156 weeks (II phase extension)]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Sequential
- Masking
- Triple blind
- Primary purpose
- Treatment
Study locations
China · 1 center
- Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology — Wuhan
Identifiers
NCT: NCT07264595 · GenSci073-201