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

27

Research Sites

N/A

Total Duration

On this page

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

Eligible

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
Not Eligible

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

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Your Study Journey

Screening

Duration - 2 to 4 weeks

Participants are screened for eligibility to participate in the trial.

1 visit (in-person)

Long-term Monitoring

Duration - Up to 5 years

Participants who undergo routine care with lonapegsomatropin are observed for safety outcomes over several years.

Periodic visits as part of routine clinical management

Trial Site Locations

Total: 27 locations

1

Ascendis Investigational Site

Phoenix, Arizona, United States, 85054

Actively Recruiting

2

Ascendis Investigational Site

Orange, California, United States, 92868

Actively Recruiting

3

Ascendis Investigational Site

Sacramento, California, United States, 95821

Actively Recruiting

4

Ascendis Investigational Site

San Francisco, California, United States, 94143

Actively Recruiting

5

Ascendis Pharma Investigational Site

Centennial, Colorado, United States, 80112

Actively Recruiting

6

Ascendis Investigational Site

Washington D.C., District of Columbia, United States, 20010

Actively Recruiting

7

Ascendis Investigational Site

Orlando, Florida, United States, 32806

Actively Recruiting

8

Ascendis Investigational Site

Orlando, Florida, United States, 32827

Actively Recruiting

9

Ascendis Investigational Site

Tampa, Florida, United States, 33612

Actively Recruiting

10

Ascendis Investigational Site

Sandy Springs, Georgia, United States, 30350

Actively Recruiting

11

Ascendis Investigational Site

Boise, Idaho, United States, 83712

Actively Recruiting

12

Ascendis Investigational Site

Indianapolis, Indiana, United States, 46202

Actively Recruiting

13

Ascendis Investigational Site

Iowa City, Iowa, United States, 52242

Actively Recruiting

14

Ascendis Investigational Site

Louisville, Kentucky, United States, 40202

Actively Recruiting

15

Ascendis Investigational Site

Minneapolis, Minnesota, United States, 55455

Actively Recruiting

16

Ascendis Investigational Site

Las Vegas, Nevada, United States, 89113

Actively Recruiting

17

Ascendis Investigational Site

Lebanon, New Hampshire, United States, 03756

Actively Recruiting

18

Ascendis Investigational Site

Manchester, New Hampshire, United States, 03104

Actively Recruiting

19

Ascendis Investigational Site

Morristown, New Jersey, United States, 07960

Actively Recruiting

20

Ascendis Investigational Site

New Hyde Park, New York, United States, 11042

Actively Recruiting

21

Ascendis Investigational Site

Cincinnati, Ohio, United States, 45229

Actively Recruiting

22

Ascendis Investigational Site

Portland, Oregon, United States, 97239

Actively Recruiting

23

Ascendis Investigational Site

Columbia, South Carolina, United States, 29203

Actively Recruiting

24

Ascendis Investigational Site

Dallas, Texas, United States, 75390

Actively Recruiting

25

Ascendis Investigational Site

El Paso, Texas, United States, 79907

Actively Recruiting

26

Ascendis Investigational Site

Norfolk, Virginia, United States, 23507

Actively Recruiting

27

Ascendis Investigational Site

Seattle, Washington, United States, 98105

Actively Recruiting

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Research Team

D

Deborah Bowlby

How is the study designed?

Study Type

OBSERVATIONAL

Masking

N/A

Allocation

N/A

Model

N/A

Primary Purpose

N/A

Number of Arms

1

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