Growth Hormone DeficiencySkyPASSNCT05775523
A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
An observational study of Growth Hormone Deficiency, run by Ascendis Pharma Endocrinology Division A/S at 27 sites in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 1 year to 18 years
- Conditions the study is for
- Growth Hormone Deficiency
- Type of treatment
- Injection or infusion
- How many people
- 500 participants
- Where
- 27 sitesUnited States
- Study dates
- 20 Mar 2023to Mar 2033
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Study population: 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.
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
27 sites in 1 country. Contact the study team or a site near you to ask about taking part.
27 sites in 1 country
United States27 sites, 27 recruiting
- Recruiting
Ascendis Investigational Site
Boise, Idaho, 83712
- Recruiting
Ascendis Pharma Investigational Site
Centennial, Colorado, 80112
- Recruiting
Ascendis Investigational Site
Cincinnati, Ohio, 45229
- Recruiting
Ascendis Investigational Site
Columbia, South Carolina, 29203
- Recruiting
Ascendis Investigational Site
Dallas, Texas, 75390
- Recruiting
Ascendis Investigational Site
El Paso, Texas, 79907
- Recruiting
Ascendis Investigational Site
Indianapolis, Indiana, 46202
- Recruiting
Ascendis Investigational Site
Iowa City, Iowa, 52242
- Recruiting
Ascendis Investigational Site
Las Vegas, Nevada, 89113
- Recruiting
Ascendis Investigational Site
Lebanon, New Hampshire, 03756
- Recruiting
Ascendis Investigational Site
Louisville, Kentucky, 40202
- Recruiting
Ascendis Investigational Site
Manchester, New Hampshire, 03104
- Recruiting
Ascendis Investigational Site
Minneapolis, Minnesota, 55455
- Recruiting
Ascendis Investigational Site
Morristown, New Jersey, 07960
- Recruiting
Ascendis Investigational Site
New Hyde Park, New York, 11042
- Recruiting
Ascendis Investigational Site
Norfolk, Virginia, 23507
- Recruiting
Ascendis Investigational Site
Orange, California, 92868
- Recruiting
Ascendis Investigational Site
Orlando, Florida, 32806
- Recruiting
Ascendis Investigational Site
Orlando, Florida, 32827
- Recruiting
Ascendis Investigational Site
Phoenix, Arizona, 85054
- Recruiting
Ascendis Investigational Site
Portland, Oregon, 97239
- Recruiting
Ascendis Investigational Site
Sacramento, California, 95821
- Recruiting
Ascendis Investigational Site
San Francisco, California, 94143
- Recruiting
Ascendis Investigational Site
Sandy Springs, Georgia, 30350
- Recruiting
Ascendis Investigational Site
Seattle, Washington, 98105
- Recruiting
Ascendis Investigational Site
Tampa, Florida, 33612
- Recruiting
Ascendis Investigational Site
Washington D.C., District of Columbia, 20010
About this study
Official title: A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
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.
- Conditions
- Growth Hormone Deficiency
- Being tested
- No intervention
- Keywords
- Human Growth Hormone, hGH, rhGH, GHD, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon hGH, Skytrofa, SkyPASS
- Medical subject headings
- Dwarfism, Pituitary, Failure to Thrive
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No interventionDrug
No intervention
| Study group | Role | What they receive |
|---|---|---|
| Patients on SKYTROFA (Lonapegsomatropin)Treatment | SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection Receives: Drug: No intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Occurrence of neoplasms (benign, malignant and unspecified)
Measured: 5 years
Occurrence of type 2 diabetes mellitus
Measured: 5 years
Secondary outcomes (6)
Occurrence of renal, hepatic, immunologic and neurologic adverse events
Measured: 5 years
Occurrence of medication errors in patients treated with lonapegsomatropin
Frequency and type of medication errors as reported by treating physician
Measured: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 level (ng/mL)
Measured: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
IGF-1 Standard Deviation Score (SDS)
Measured: 5 years
Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature
Measured: 5 years
Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature
Measured: 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2023
- Study start20 Mar 2023
- Primary completionMar 2033 (estimated)
- Study completionMar 2033 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Growth Hormone DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 500 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/S
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medical Director, MD, Study director, Ascendis Pharma A/S
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05775523
- Sponsor's protocol ID
- ASND0033
- Acronym
- SkyPASS
- Status last verified
- Feb 2026
- First submitted
- 8 Mar 2023
- Last updated
- 4 Feb 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
- ClinicalTrials.gov ID
- NCT05775523
- Acronym / sponsor ID
- SkyPASS / ASND0033
- Conditions
- Growth Hormone Deficiency
- Keywords
- Human Growth Hormone, hGH, rhGH, GHD, Long Acting Growth Hormone, Lonapegsomatropin, Prodrug, Growth Failure
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 500 participants (estimated)
- Lead sponsor
- Ascendis Pharma Endocrinology Division A/SIndustry (pharma, biotech, devices)
- Study start
- 20 Mar 2023 (actual)
- Primary completion
- Mar 2033 (estimated)
- Study completion
- Mar 2033 (estimated)
- First posted
- 20 Mar 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients on SKYTROFA (Lonapegsomatropin)Treatment | – | Drug: No interventionSKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection |
Who may qualify
- Ages
- 1 year to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- 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.
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No interventionNo intervention | Drug | Patients on SKYTROFA (Lonapegsomatropin)Treatment |
Primary outcome
Occurrence of neoplasms (benign, malignant and unspecified)
Time frame: 5 years
Occurrence of type 2 diabetes mellitus
Time frame: 5 years
6 secondary outcomes
Occurrence of renal, hepatic, immunologic and neurologic adverse events
Time frame: 5 years
Occurrence of medication errors in patients treated with lonapegsomatropin
Time frame: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
Time frame: 5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
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
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2026.
- Individual participant dataSharing planned: No
Related trials for Growth Hormone Deficiency
RecruitingPhase 3
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Turner Syndrome, Short Stature Homeobox Gene Mutation
RecruitingPhase 3
Randomized Clinical Trial of Two Different Initial Growth Hormone Doses in Children
Growth Disorders, Growth Failure
RecruitingPhase 3
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Growth Hormone Deficiency (GHD)
RecruitingPhase 3
Growth Hormone Replacement Therapy in Veterans With Mild Traumatic Brain Injury (mTBI) and Adult Growth Hormone Deficiency (AGHD)
Adult Growth Hormone Deficiency, Mild Traumatic Brain Injury
Enrolling by invitationObservational study
Influence of Adherence to Growth Hormone Therapy (GHT) With Norditropin® on Near Final Height in Patients With Growth Hormone Deficiency (GHD) and Born Small for Gestational Age (SGA)
Growth Hormone Deficiency in Children, Born Small for Gestational Age
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Medical Director, MD study director
Ascendis Pharma A/S
27 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Growth Hormone Deficiency, run by Ascendis Pharma Endocrinology Division A/S at 27 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 4 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Deborah Bowlby (+1 844-442-7236, SkyPASS_Contact@ascendispharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Occurrence of neoplasms (benign, malignant and unspecified) (time frame: 5 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2026.