Recruiting NCT05775523
A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: No intervention.
- Who it may be relevant to
- Registry conditions: Growth Hormone Deficiency. Basic parameters: 1 year — 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
Overview
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
- Drug No intervention
No intervention
Primary outcome measures
- Occurrence of neoplasms (benign, malignant and unspecified) [Time frame: 5 years]
- Occurrence of type 2 diabetes mellitus [Time frame: 5 years]
Secondary outcome measures (6)
- Occurrence of renal, hepatic, immunologic and neurologic adverse events [Time frame: 5 years]
- Occurrence of medication errors in patients treated with lonapegsomatropin [Time frame: 5 years]
- Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy [Time frame: 5 years]
- Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy [Time frame: 5 years]
- Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature [Time frame: 5 years]
- Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature [Time frame: 5 years]
Eligibility criteria
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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 27 centers
- Ascendis Investigational Site — Phoenix
- Ascendis Investigational Site — Orange
- Ascendis Investigational Site — Sacramento
- Ascendis Investigational Site — San Francisco
- Ascendis Pharma Investigational Site — Centennial
- Ascendis Investigational Site — Washington D.C.
- Ascendis Investigational Site — Orlando
- Ascendis Investigational Site — Orlando
- … and 19 more centers
Identifiers
NCT: NCT05775523 · ASND0033