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

Sponsor

Ascendis Pharma Endocrinology Division A/S (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05775523

Collaborator

(none)

500

Enrollment

120

Anticipated Duration (Months)

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Growth Hormone Deficiency	Drug: No intervention

Study Design

Study Type:

Observational

Anticipated Enrollment :

500 participants

Observational Model:

Cohort

Time Perspective:

Prospective

Official Title:

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

Anticipated Study Start Date :

Mar 1, 2023

Anticipated Primary Completion Date :

Mar 1, 2033

Anticipated Study Completion Date :

Mar 1, 2033

Arms and Interventions

Arm	Intervention/Treatment
Patients on SKYTROFA (Lonapegsomatropin)Treatment SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection	Drug: No intervention No intervention

Arm

Intervention/Treatment

Patients on SKYTROFA (Lonapegsomatropin)Treatment

SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection

Drug: No intervention

No intervention

Outcome Measures

Primary Outcome Measures

To evaluate the occurrence of neoplasms (benign, malignant and unspecified) [5 years]
To evaluate the occurrence of type 2 diabetes mellitus [5 years]

Secondary Outcome Measures

Occurrence of renal, hepatic, immunologic and neurologic adverse events [5 years]
Occurrence of medication errors in patients treated with lonapegsomatropin [5 years]
Characterise Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy [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]

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Year to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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