An observational study in Growth Hormone Deficiency, sponsored by Ascendis Pharma Endocrinology Division A/S. Recruiting at 27 sites in United States. Open to participants aged 1 Year to 18 Years. Per ClinicalTrials.gov, last updated 2026-02-04.
Sponsored by Ascendis Pharma Endocrinology Division A/S · Observational
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
252 studies on the registry are indexed under Dwarfism, Pituitary; 30 are open to participants now.
This study's planned enrollment of 500 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 from centres in Europe and the USA will be eligible for enrollment into the study.
Patients may be enrolled in the study if they are on treatment with lonapegsomatropin. The decision to treat the patient with lonapegsomatropin will be made prior to and independently of the decision to invite the patient to enroll into the study. Both patients naïve to GH treatment and patients previously treated with GH therapy will be eligible for enrollment.
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.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
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.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Excipients
The other ingredients in a medicine besides the active drug, such as fillers, colours or preservatives.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection
Drug: No intervention
No intervention
Occurrence of neoplasms (benign, malignant and unspecified)
Time frame: 5 years
Occurrence of type 2 diabetes mellitus
Time frame: 5 years
Occurrence of renal, hepatic, immunologic and neurologic adverse events
Time frame: 5 years
Occurrence of medication errors in patients treated with lonapegsomatropin
Frequency and type of medication errors as reported by treating physician
Time frame: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 level (ng/mL)
Time frame: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 Standard Deviation Score (SDS)
Time frame: 5 years
Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature
Time frame: 5 years
Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature
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.
Contact study teamGet an email when the registry record changes — status, dates, results — or when someone posts here.
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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