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Growth Hormone Therapy in Children with Short Stature due to Pseudohypoparathyroidism Type 1A (PHP1A): A Multicenter Retrospective Real-World Study

Growth Hormone Therapy in Children with Short Stature due to Pseudohypoparathyroidism Type 1A (PHP1A): A Multicenter Retrospective Real-World Study

Status
Active, not recruiting
Phases
Unknown
Study type
Observational
Source
ChiCTR
Registry ID
ChiCTR2500111479
Enrollment
Unknown
Registered
2025-10-31
Start date
2025-11-02
Completion date
Unknown
Last updated
2025-11-03

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

Conditions

Pseudohypoparathyroidism type 1a

Interventions

Sponsors

Children's Hospital, Zhejiang University School of Medicine
Lead Sponsor

Eligibility

Sex/Gender
All
Age
3.5 Years to 18 Years

Inclusion criteria

Inclusion criteria: 1) Age >= 3.5 years at the initiation of GH therapy; 2) Height at screening below -2 standard deviation score (SD) of the height of normal children of the same chronological age, gender, and ethnicity; or below the 3rd percentile (P3, -1.88 SD); 3) Epiphyses confirmed to be unclosed by bone age radiographs before treatment; 4) Children diagnosed with PHP1A through clinical phenotype and genetic testing; 5) Complete case records (including baseline data, GH treatment records of >= 1 year, and follow-up data).

Exclusion criteria

Exclusion criteria: 1) Retrospective cases with growth hormone (GH) treatment duration of less than 1 year; 2) Patients with diabetes mellitus, impaired liver or kidney function, epilepsy, intellectual disability, skeletal deformities, slipped capital femoral epiphysis, severe cardiopulmonary diseases, hematological system diseases, malignant tumors, systemic infections, immunodeficiency, or mental illnesses; 3) Patients allergic to GH or its protective agents; 4) Cases with missing key data (fewer than 3 bone age radiographs per year or missing data from 2 consecutive follow-ups); 5) Other conditions deemed unsuitable for inclusion in this study by the researchers.

Design outcomes

Primary

MeasureTime frame
?PAH;body composition;

Secondary

MeasureTime frame
?HtSDSCA;?HtSDSBA;?BA/?CA;GV;IGF-1;BMI;Carbohydrate Metabolism;

Countries

China

Contacts

Public ContactWei Wu

Children's Hospital, Zhejiang University School of Medicine

wuwei120@zju.edu.cn+86 136 6664 5030

Outcome results

None listed

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