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

J
John Fuqua, MD

Primary Investigator

Recruiting
17 years or below
All
Phase N/A
1 participants needed
1 Location

Brief description of study

What is the purpose of this study?
This is a non-interventional, observational research study of a marketed drug, Skytrofa. The purpose of this study is to evaluate the long-term effectiveness and safety of Skytrofa treatment in a large group of subjects. Skytrofa is approved in the U.S. for sale and use in children with growth hormone deficiency (GHD). This study is being done to find out how safe and useful Skytrofa is for long-term treatment.
 

Detailed description of study

What will happen during the study?
  • Parent and child will be asked to answer a number of questionnaires every 6 months on functioning and well-being and treatment satisfaction.  
  • The questionnaires will be collected using a Web-based or electronic tablet.
  • All subjects will be followed for a minimum of 5 years and up to 10 years depending on when the last subject is enrolled.

Eligibility of study

You may be eligible for this study if you meet the following criteria:

  • Conditions: Growth Hormone Deficiency, GHD, Riley
  • Age: 17 years or below
  • Gender: All

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

Updated on 22 Apr 2025. Study ID: PENDO-ASCENDIS-SKYPASS, 21841

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