An observational study in Growth Hormone Deficiency, sponsored by Ascendis Pharma Endocrinology Division A/S. Recruiting at 27 sites in United States. Per ClinicalTrials.gov, last updated 2026-04-21.
Sponsored by Ascendis Pharma Endocrinology Division A/S · Observational
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
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.
252 studies on the registry are indexed under Dwarfism, Pituitary; 30 are open to participants now.
This study's planned enrollment of 900 is above the median of 70 across 73 observational studies indexed under Dwarfism, Pituitary.
Browse Dwarfism, Pituitary studies →Ascendis Pharma Endocrinology Division A/S is the lead sponsor of 7 studies on the registry; 2 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Patients being treated with SKYTROFA who are associated to selected endocrinology or pediatric clinics across USA.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Patients on SKYTROFA Treatment managed in USA with appropriate written Informed Consent
Other: No intervention
No intervention
Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin)
Incidence of Adverse Events and Serious Adverse Events
Time frame: 5 years
Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin)
Near adult height (cm)
Time frame: 5 years
Assess clinical outcome assessments (COAs)
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))
Time frame: 5 years
Assess clinical outcome assessments (COAs)
Change in Treatment Satisfaction Questionnaire for Medicine (TSQM-9) scores from baseline through End of Study (The TSQM-9 domain scores range from 0 to 100 with higher scores representing higher satisfaction on that domain)
Time frame: 5 years
Plan to share: No
No publications or documents are linked to this record.
Started Mar 2023 (actual). Primary completion is estimated for Mar 2033, 120 months after the start.
Of 30 observational studies under Dwarfism, Pituitary, half reached primary completion within 85 months of starting (the median), counting those still running; a quarter within 54 and three quarters within 102. This study's planned 120 months is above that.
How these figures are worked out, and which studies are left out of them.
Eligibility is decided by the study team. Share this record with your doctor or contact the team directly.
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Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Ascendis Pharma Endocrinology Division A/S