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

ConditionPediatric Growth Hormone Deficiency
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age3+
SponsorChangchun GeneScience Pharmaceutical Co., Ltd.

About this trial

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 criteria

This trial does not accept healthy volunteers

Qualifiers

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)

Disqualifiers

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

Trial design

Design model

Sequential

Treatments tested in this trial

  • GS3-007a dry suspension

    Drug

    GS3-007a

  • Placebo for GS3-007a dry suspension

    Drug

    Placebo for GS3-007a dry suspension

  • rhGH injection

    Drug

    rhGH injection

Treatment groups

88 Participants
are divided into 3 treatment groups
Group A: Dose Level 1~ Dose Level 4Placebo comparator 2 interventions
Group B: Cohort 1~ Cohort 3Active comparator 2 interventions
Group C: GS3-007a cohortExperimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

Number of Participants With Treatment Emergent Adverse Events (TEAEs)

Time frame
up to 21 days (Ib phase)
2

Annualized height velocity (AHV) at Week 26 of treatment

Time frame
26 weeks (II phase)
3

Number of Participants With TEAEs

Time frame
up to 160 weeks (II phase extension)

Secondary outcomes

1

Plasma Concentrations of GS3-007a and metabolite GS3-017. Due to Spare sampling time, there are no PK parameter

Time frame
up to 7 days (Ib phase)
2

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)
3

AHVs at each evaluation point

Time frame
13 weeks, 39 weeks, 52 weeks (II phase)
4

The standard deviation score (SDS) for an individual IGF-I measurement is calculated using the age-specific L, M, and S parameters, no minimum and maximum values, with higher scores indicating treatment improvement in PGHD

Time frame
4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Changchun GeneScience Pharmaceutical Co., Ltd.

Lead sponsor

Source ClinicalTrials.gov
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