A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin (SkyPASS)

February 2, 2026 updated by: Ascendis Pharma Endocrinology Division A/S

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Estimated)

500

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
        • Recruiting
        • Ascendis Investigational Site
    • California
      • Orange, California, United States, 92868
        • Recruiting
        • Ascendis Investigational Site
      • Sacramento, California, United States, 95821
        • Recruiting
        • Ascendis Investigational Site
      • San Francisco, California, United States, 94143
        • Recruiting
        • Ascendis Investigational Site
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Recruiting
        • Ascendis Pharma Investigational Site
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20010
        • Recruiting
        • Ascendis Investigational Site
    • Florida
      • Orlando, Florida, United States, 32806
        • Recruiting
        • Ascendis Investigational Site
      • Orlando, Florida, United States, 32827
        • Recruiting
        • Ascendis Investigational Site
      • Tampa, Florida, United States, 33612
        • Recruiting
        • Ascendis Investigational Site
    • Georgia
      • Sandy Springs, Georgia, United States, 30350
        • Recruiting
        • Ascendis Investigational Site
    • Idaho
      • Boise, Idaho, United States, 83712
        • Recruiting
        • Ascendis Investigational Site
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Ascendis Investigational Site
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • Ascendis Investigational Site
    • Kentucky
      • Louisville, Kentucky, United States, 40202
        • Recruiting
        • Ascendis Investigational Site
    • Minnesota
      • Minneapolis, Minnesota, United States, 55455
        • Recruiting
        • Ascendis Investigational Site
    • Nevada
      • Las Vegas, Nevada, United States, 89113
        • Recruiting
        • Ascendis Investigational Site
    • New Hampshire
      • Lebanon, New Hampshire, United States, 03756
        • Recruiting
        • Ascendis Investigational Site
      • Manchester, New Hampshire, United States, 03104
        • Recruiting
        • Ascendis Investigational Site
    • New Jersey
      • Morristown, New Jersey, United States, 07960
        • Recruiting
        • Ascendis Investigational Site
    • New York
      • New Hyde Park, New York, United States, 11042
        • Recruiting
        • Ascendis Investigational Site
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Ascendis Investigational Site
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Ascendis Investigational Site
    • South Carolina
      • Columbia, South Carolina, United States, 29203
        • Recruiting
        • Ascendis Investigational Site
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • Ascendis Investigational Site
      • El Paso, Texas, United States, 79907
        • Recruiting
        • Ascendis Investigational Site
    • Virginia
      • Norfolk, Virginia, United States, 23507
        • Recruiting
        • Ascendis Investigational Site
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Ascendis Investigational Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (Child, Adult)

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

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.

Description

Inclusion Criteria:

  • 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

Exclusion Criteria:

  • 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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

Cohorts and Interventions

Group / Cohort
Intervention / Treatment
Patients on SKYTROFA (Lonapegsomatropin)Treatment
SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection
Drug: No intervention
No intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Occurrence of neoplasms (benign, malignant and unspecified)
Time Frame: 5 years
5 years
Occurrence of type 2 diabetes mellitus
Time Frame: 5 years
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Occurrence of renal, hepatic, immunologic and neurologic adverse events
Time Frame: 5 years
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
5 years
Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature
Time Frame: 5 years
5 years
Occurrence of medication errors in patients treated with lonapegsomatropin
Time Frame: 5 years
Frequency and type of medication errors as reported by treating physician
5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
Time Frame: 5 years
IGF-1 level (ng/mL)
5 years
Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
Time Frame: 5 years
IGF-1 Standard Deviation Score (SDS)
5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Ascendis Pharma Endocrinology Division A/S

Investigators

  • Study Director: Medical Director, MD, Ascendis Pharma A/S

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 20, 2023

Primary Completion (Estimated)

March 1, 2033

Study Completion (Estimated)

March 1, 2033

Study Registration Dates

First Submitted

March 8, 2023

First Submitted That Met QC Criteria

March 8, 2023

First Posted (Actual)

March 20, 2023

Study Record Updates

Last Update Posted (Actual)

February 4, 2026

Last Update Submitted That Met QC Criteria

February 2, 2026

Last Verified

February 1, 2026

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ASND0033

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

Yes

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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