A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)

Phase 2Not Yet RecruitingNCT07264595

Changchun GeneScience Pharmaceutical Co., Ltd.88 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Pediatric Growth Hormone Deficiency

Interventions

Eligibility

Sex: ALLMin age: 3 Years

Medical Language ↔ Plain English

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

Outcomes

Primary Outcomes

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 Outcomes

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)