Growth Hormone Deficiency
Conditions
Keywords
Human Growth Hormone, SKYTROFA
Brief summary
The goal of this study is to genrate evidence on long-term effectiveness and safety of SKYTROFA (lonapegsomatropin) in patients with growth hormone deficiency under routine clinical care
Detailed description
Patients will be treated according to routine clinical practice, and no additional visits, examinations or tests will be required beyond those performed as part of routine clinical practice except for COAs.
Interventions
OTHERNo intervention
No intervention
Sponsors
Ascendis Pharma Endocrinology Division A/S
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin) | 5 years | Incidence of Adverse Events and Serious Adverse Events |
| Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin) | 5 years | Near adult height (cm) |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Assess clinical outcome assessments (COAs) | 5 years | Change in Quality of Life in Short Stature Youth (QoLISSY) scores from baseline through End of Study (QOLISSY scores range on a scale of 0 to 100 with higher scores representing better quality of life. (For both child and parent version)) |
Countries
United States
Contacts
CONTACTDeborah Bowlby
STUDY_DIRECTORMedical Director, MD
Ascendis Pharma A/S
Outcome results
None listed