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

Growth Hormone Deficiency Clinical Trial

— SkyPASS  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05775523
• Other study ID #: ASND0033
• Status: Recruiting
• Start date: March 20, 2023
• Est. completion date: March 2033

Study information

• Verified date: January 2024
• Source: Ascendis Pharma A/S
• Contact: Mandeep Singh
• Phone: +1 650-505-4946
• Email: msh[@]ascendispharma.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: March 2033
• Est. primary completion date: March 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Growth Hormone Deficiency

Intervention

Drug:
• No intervention
No intervention

Locations

Country	Name	City	State
United States	Ascendis Pharma Investigational Site	Centennial	Colorado

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma Endocrinology Division A/S

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Occurrence of neoplasms (benign, malignant and unspecified)		5 years
Primary	Occurrence of type 2 diabetes mellitus		5 years
Secondary	Occurrence of renal, hepatic, immunologic and neurologic adverse events		5 years
Secondary	Occurrence of medication errors in patients treated with lonapegsomatropin	Frequency and type of medication errors as reported by treating physician	5 years
Secondary	Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy	IGF-1 level (ng/mL)	5 years
Secondary	Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy	IGF-1 Standard Deviation Score (SDS)	5 years
Secondary	Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature		5 years
Secondary	Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature		5 years