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Efficacy and safety of rhGH in PTH/PTHrP signaling disorders

Efficacy and safety of rhGH in PTH/PTHrP signaling disorders: a multicenter, open-label, observational cohort study

Status
Active, not recruiting
Phases
Phase 4
Study type
Observational
Source
ChiCTR
Registry ID
ChiCTR2500098947
Enrollment
Unknown
Registered
2025-03-17
Start date
2025-03-18
Completion date
Unknown
Last updated
2025-03-24

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

PTH/PTHrP signaling pathway disorders

Interventions

rhGH treatment group:None
Observation group:None

Sponsors

Children's Hospital of Soochow University
Lead Sponsor

Eligibility

Sex/Gender
All
Age
3 Years to 16 Years

Inclusion criteria

Inclusion criteria: 1. Age >= 3.5 years; 2. The bone age of girls = 13 years and that of boys = 15 years; 3. PTH/PTHrP signaling pathway disorders diagnosed by clinical phenotypic and genetic tests (including PHP1A, PHP1C, ACRDYS1, ACRDYS2, HTNB, PTHLH and TRPS1 gene mutations); 4. Subjects and guardians are willing and able to cooperate in completing the scheduled visit, treatment plan, laboratory examinations, and other testing procedures, and they or their guardians sign the informed consent voluntarily.

Exclusion criteria

Exclusion criteria: 1. Retrospective cases treated with growth hormone for less than two years; 2. Patients with diabetes, epilepsy, mental retardation, skeletal deformity, femoral head slippage, severe cardiopulmonary, hematological, and malignant tumors or systemic infections, immunodeficiency, and mental illness; 3. Those allergic to growth hormone or its protective agents; 4. Failure to provide complete medical records, failure to expect complete follow-up, or failure to collect complete medical records; 5. Other conditions deemed unsuitable by the investigator for inclusion in this clinical trial.

Design outcomes

Secondary

MeasureTime frame
?HtSDS ( CA, BA), GV,IGF-1,Bone metabolism,BMI,Body compositon before and after treatment;Glycolipid metabolic profile;Adverse events and complications;

Primary

MeasureTime frame
The change in predicted adult height before and after treatment(?PAH);

Countries

China

Contacts

Public ContactTing Chen

Children's Hospital of Soochow University

chenting888@suda.edu.cn+86 512 80698211

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026