Evaluating the effects of PEGylated growth hormone on short stature in children
A Real-world Study of the Efficacy and Safety of PEGylated Recombinant Human Growth Hormone Injection in the Treatment of Short Stature in Chinese Children From the Global Registry for Novel Therapies For Rare Bone or Endocrine Conditions
This study is testing if a new growth hormone treatment can help children who are shorter than average grow taller and stay healthy over time.
Quick facts
| Study type | Observational |
|---|---|
| Enrollment | 2600 (estimated) |
| Ages | N/A to 18 Years |
| Sex | All |
| Sponsor | Beijing Children's Hospital Academic / other |
| Locations | 1 site (Beijing, Beijing Municipality) |
| Trial ID | NCT06037473 on ClinicalTrials.gov |
What this trial studies
This observational study aims to assess the long-term safety and effectiveness of polyethylene glycol-recombinant human growth hormone (PEG-rhGH) in treating growth disorders in children, including growth hormone deficiency and idiopathic short stature. It involves a retrospective analysis of treatment outcomes and factors influencing efficacy, followed by prospective monitoring. Data collected will be uploaded to the Global Registry of New Therapies for Rare Endocrine Diseases and Bone Disorders (GloBE-Reg) to enhance understanding of real-world PEG-GH treatment.
Who should consider this trial
Good fit: Ideal candidates include children diagnosed with endogenous growth hormone deficiency, Turner syndrome, or idiopathic short stature who are receiving PEG-rhGH treatment.
Not a fit: Patients with serious comorbid conditions such as severe heart or lung disease, malignancies, or significant immune dysfunction may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide valuable insights into effective treatments for children with growth disorders, potentially improving their growth outcomes.
How similar studies have performed: Other studies have shown promising results with similar long-acting growth hormone treatments, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: Patients with endogenous growth hormone deficiency, Turner Syndrome, SGA, idiopathic short stature, etc. treated with polyethylene glycol recombinant human growth hormone injection Exclusion Criteria: Patients with serious heart and lung, blood system, malignant tumors and other diseases or systemic infections, immune function is low and Persons with mental illness;
Where this trial is running
Beijing, Beijing Municipality
- Department of Endocrinology, Genetics, Metabolism — Beijing, Beijing Municipality, China (Recruiting)
Study contacts
- Principal investigator: Bingyan Cao, Dr. — Beijing Children's Hospital
- Study coordinator: Chunxiu Gong, Dr.
- Email: chunxiugong@163.com
- Phone: 13370115001
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.