Growth Hormone Deficiency
Conditions
Keywords
Human Growth Hormone, hGH, rhGH, GHD, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon hGH, Skytrofa, SkyPASS
Brief summary
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
Interventions
DRUGNo intervention
No intervention
Sponsors
Ascendis Pharma Endocrinology Division A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
1 Years to 18 Years
Healthy volunteers
No
Inclusion criteria
* Paediatric patients with GHD who are on treatment with lonapegsomatropin * Patients being clinically managed in Europe or the USA * Appropriate written informed consent/assent as applicable for the age of the patient * Patients willing to comply with follow-up requirements of the study
Exclusion criteria
* Patients participating in any interventional clinical trial for short stature * Patients being treated with a GH or IGF-1 therapy, other than lonapegsomatropin, at enrollment * Patients for whom treatment with lonapegsomatropin is contraindicated * Patients with closed epiphyses * Patients with active malignant tumours * Patients under antitumour therapy within the past 12 months prior to instituting GH therapy * Hypersensitivity to somatropin or any of the excipients in lonapegsomatropin
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Occurrence of neoplasms (benign, malignant and unspecified) | 5 years |
| Occurrence of type 2 diabetes mellitus | 5 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Occurrence of renal, hepatic, immunologic and neurologic adverse events | 5 years | — |
| Occurrence of medication errors in patients treated with lonapegsomatropin | 5 years | Frequency and type of medication errors as reported by treating physician |
| Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy | 5 years | IGF-1 level (ng/mL) |
| Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature | 5 years | — |
| Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature | 5 years | — |
Countries
United States
Contacts
CONTACTDeborah Bowlby
STUDY_DIRECTORMedical Director, MD
Ascendis Pharma A/S
Outcome results
None listed