A US Multi-center, Prospective, Non-interventional, Long Term, Effectiveness and Safety Study of Patients Treated With SKYTROFA (Lonapegsomatropin)

J
John Fuqua, MD

Primary Investigator

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

Brief description of study

Why is this study being done?
This is a non-interventional, observational research study of a marketed drug, Skytrofa. These studies are carried out to identify and/or measure effects of medicines or treatment under real-life conditions.  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. The purpose of this study is to evaluate the long-term effectiveness and safety of Skytrofa treatment in  children.
 

Detailed description of study

What will happen during the study?
  • Participants' care will follow the normal treatment practices at the clinic.  No additional visits will be performed beyond the usual clinical practice.
  • Participants will be asked to answer 2 questionnaires every 6 months on functioning and well-being and treatment satisfaction.
  • The questionnaires will be collected using a Web-based or electronic tablet.
  • Enrollment into this study will occur over a 5-year period. 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: 1 years - 17 years
  • Gender: All

Inclusion Criteria:
 

  •     Patients who are on treatment with SKYTROFA (lonapegsomatropin)
  •   Patients being clinically managed in USA
  •   Patients with an appropriate written informed consent/assent as applicable for the age of the patient

Exclusion Criteria:
 

  •     Patients participating in any interventional clinical study

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

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