A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin

Study Overview
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Brief Summary
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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.

Official Title
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A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin

Conditions
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Growth Hormone Deficiency

Intervention / Treatment
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* Drug: No intervention


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Patients on SKYTROFA (Lonapegsomatropin)Treatment<br>SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection | Drug: No intervention<br>* No intervention<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence of neoplasms (benign, malignant and unspecified) |  | 5 years | 
| Occurrence of type 2 diabetes mellitus |  | 5 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Occurrence of renal, hepatic, immunologic and neurologic adverse events |  | 5 years | 
| Occurrence of medication errors in patients treated with lonapegsomatropin | Frequency and type of medication errors as reported by treating physician | 5 years | 
| Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy | IGF-1 level (ng/mL) | 5 years | 
| Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy | IGF-1 Standard Deviation Score (SDS) | 5 years | 
| 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 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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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