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Completed

NCT Number: NCT05353192

A Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

To Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children with Achondroplasia

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Key information

Conditions

Achondroplasia Bone Diseases Bone Diseases, Developmental Congenital, Hereditary, and Neonatal Diseases and Abnormalities Dwarfism Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias

Age range

2 year–10 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 4

Primary location

Children's Hospital of Fudan University

Shanghai, Shanghai Municipality, 201102, China

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • 2-10 years old;
  • In Tanner I stage;
  • ACH, documented and confirmed by genetic testing;
  • Short stature;
  • Ambulatory and able to stand or walk without assistance;
  • Parent(s) or guardian(s) consent；
  • Had never been treated with growth hormone

Exclusion criteria

  • Short stature condition other than ACH;
  • Evidence of growth plate closure (proximal tibia, distal femur);
  • Had a fracture of the long bones within 6 months prior to screening;
  • Planned or expected bone-related surgery;
  • Chronic diseases condition that affect bone metabolism and weight;
  • Severe intracranial hypertension

Treatment and study plan

Recombinant human growth hormone

Drug

Recombinant human growth hormone (15IU/5mg/3ml/bottle)，0.05 mg/kg/d by subcutaneous injection for 52 weeks

Primary outcomes

  1. Change From Baseline in Annualized Height Velocity (AHV) at Week 52

    Time frame: week 52

    The change in AHV after 52 weeks treatment

Secondary outcomes

  1. Change From Baseline in AHV

    Time frame: week 4, week 13, week 26, week 39

    Change in AHV at week, week 13, week 26, week 39

  2. Change From Baseline in Height Standard Deviation Score (Ht SDS)

    Time frame: week 4, week 13, week 26, week 39, week 52

    Change From Baseline in Ht SDS at week 4, week 13, week 26, week 39, week 52

  3. Change From Baseline in BMI Standard Deviation Score (BMI SDS)

    Time frame: week 4, week 13, week 26, week 39, week 52

    Change From Baseline in BMI SDS at week 4, week 13, week 26, week 39, week 52

  4. Change From Baseline in Sitting Height/Leg Length ratio Standard Deviation Score (SH/LL SDS)

    Time frame: week 4, week 13, week 26, week 39, week 52

    Change From Baseline in SH/LL SDS at week 4, week 13, week 26, week 39, week 52

  5. Change From Baseline in IGF-1 Standard Deviation Score (IGF -1 SDS)

    Time frame: week 4, week 13, week 26, week 39, week 52

    Change From Baseline in IGF-1 SDS at week 4, week 13, week 26, week 39, week 52

  6. Change From Baseline in IGF-1/IGFBP-3 ratio

    Time frame: week 4, week 13, week 26, week 39, week 52

    Change From Baseline in IGF-1/IGFBP-3 ratio at week 4, week 13, week 26, week 39, week 52

  7. Change From Baseline in Bone Age/Chronological Age ratio (BA/CA)

    Time frame: week 52

    Change From Baseline in BA/CA at week 52

Other outcomes

  1. Change From Baseline in Metabolism-related Proteins

    Time frame: week 13, week 26, week 52

    Change From Baseline in Serum Proteomics at week 13, week 26, week 52

Sponsors and collaborators

Lead sponsor

Changchun GeneScience Pharmaceutical Co., Ltd.

Industry

Collaborators

  • Chengdu Women's and Children's Central Hospital
  • Children's Hospital of Fudan University
  • Children's Hospital of Nanjing Medical University
  • Children's Hospital of The Capital Institute of Pediatrics
  • Jiangxi Province Children's Hospital
  • Shandong Provincial Hospital
  • Shanghai Children's Hospital
  • Shanghai Children's Medical Center
  • Shengjing Hospital
  • Tongji Hospital
  • West China Second University Hospital

Registry information

Official study title

A Multicenter, Open-label, Single Arm Phase IV Clinical Study to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone in Children With Achondroplasia

Important dates

Study start
2022
Primary completion
2025
Study completion
2025
First posted
Apr 29, 2022
Registry last updated
Aug 6, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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