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

Recruiting

• Conditions: Growth Hormone Deficiency
• Interventions: Drug: No intervention

• Registration Number: NCT05775523
• Lead Sponsor: Ascendis Pharma Endocrinology Division A/S

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.

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2023-03-08
• Study First Submitted QC: 2023-03-08
• Study Start: 2023-03-20
• Study First Posted: 2023-03-20
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-02
• Last Update Posted: 2025-05-04
• Primary Completion: 2033-03
• Study Completion: 2033-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 500

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients on SKYTROFA (Lonapegsomatropin)Treatment	No intervention	SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection

Primary Outcome Measures

Name	Time	Method
Occurrence of neoplasms (benign, malignant and unspecified)	5 years
Occurrence of type 2 diabetes mellitus	5 years

Secondary Outcome Measures

Name	Time	Method
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy	5 years	IGF-1 Standard Deviation Score (SDS)
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
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

Trial Locations

Locations (2)

Ascendis Investigational Site; 🇺🇸 Seattle, Washington, United States

Ascendis Pharma Investigational Site; 🇺🇸 Centennial, Colorado, United States