What is this study trying to find out about growth hormone deficiency?

This study is observing the long-term safety of lonapegsomatropin treatment in children with growth hormone deficiency. It aims to understand potential risks like tumors and diabetes over five years. The research is sponsored by Ascendis Pharma Endocrinology Division A/S and focuses on real-world patient experiences.

Who may be eligible for this growth hormone deficiency clinical trial?

Children aged 1 to 18 years with growth hormone deficiency who are treated with lonapegsomatropin may be eligible. Participants must not be receiving other growth hormone or IGF-1 therapies and should not have active tumors or closed growth plates. Please review eligibility criteria for more details.

Is there any cost to participate in this growth hormone deficiency clinical trial?

Study-related care, including the study drug or procedure and all trial visits, is provided at no cost. However, personal travel expenses may vary and are not guaranteed to be covered. Participants should plan accordingly for any travel needs.

Actively Recruiting

Age: 1Year - 18Years

All Genders

ID05775523

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

Led by Ascendis Pharma Endocrinology Division A/S · Updated on 2026-02-04

500

Participants Needed

Research Sites

N/A

Total Duration

AI-Summary

What this Trial Is About

Researchers are evaluating the long-term safety of lonapegsomatropin in children with growth hormone deficiency who are already being treated with this medication. This study is observational and aims to gather real-world safety data over time after the drug has been authorized for use. The focus is on monitoring potential risks such as the development of tumors and type 2 diabetes over a period of five years. Participants receive lonapegsomatropin, administered once weekly by subcutaneous injection as part of their usual care. The study does not involve additional interventions but observes patients who are already treated with this therapy. The observational period extends for at least five years to collect safety data and compare it with historical information from previous studies. During the study, researchers will monitor for the occurrence of benign and malignant tumors, type 2 diabetes, and other adverse events affecting the kidneys, liver, immune system, and nervous system. They will also track medication errors and measure the response of Insulin-like Growth Factor-1 IGF-1 to treatment. Participants are followed up regularly to collect this information, with the study lasting until March 2033.

CONDITIONS

Brief Title

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

Who Can Participate

Age: 1Year - 18Years

All Genders

Eligibility Criteria

You may qualify if you...

Paediatric patients with growth hormone deficiency who are on treatment with lonapegsomatropin
Patients being clinically managed in Europe or the USA
Appropriate written informed consent or assent as applicable for the age of the patient
Patients willing to comply with follow-up requirements of the study

You will not qualify if you...

Patients participating in any interventional clinical trial for short stature
Patients being treated with a growth hormone or IGF-1 therapy other than lonapegsomatropin at enrollment
Patients for whom treatment with lonapegsomatropin is contraindicated
Patients with closed growth plates (epiphyses)
Patients with active malignant tumors
Patients under antitumor therapy within the past 12 months prior to starting growth hormone therapy
Hypersensitivity to somatropin or any of the ingredients in lonapegsomatropin

Research Team

Deborah Bowlby

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