NCT05775523

Brief 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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
500

participants targeted

Target at P75+ for all trials

Timeline
83mo left

Started Mar 2023

Longer than P75 for all trials

Geographic Reach
1 country

27 active sites

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress32%
Mar 2023Mar 2033

First Submitted

Initial submission to the registry

March 8, 2023

Completed
12 days until next milestone

First Posted

Study publicly available on registry

March 20, 2023

Completed
Same day until next milestone

Study Start

First participant enrolled

March 20, 2023

Completed
10 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2033

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2033

Last Updated

February 4, 2026

Status Verified

February 1, 2026

Enrollment Period

10 years

First QC Date

March 8, 2023

Last Update Submit

February 2, 2026

Conditions

Keywords

Human Growth HormonehGHrhGHGHDLong Acting Growth HormoneLonapegsomatropinProdrugGrowth FailureGrowth Hormone Replacement TherapySustained Release Growth HormoneGrowth Hormone DeficiencyTransCon hGHSkytrofaSkyPASS

Outcome Measures

Primary Outcomes (2)

  • Occurrence of neoplasms (benign, malignant and unspecified)

    5 years

  • Occurrence of type 2 diabetes mellitus

    5 years

Secondary Outcomes (6)

  • Occurrence of renal, hepatic, immunologic and neurologic adverse events

    5 years

  • Occurrence of medication errors in patients treated with lonapegsomatropin

    5 years

  • Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy

    5 years

  • Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy

    5 years

  • Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature

    5 years

  • +1 more secondary outcomes

Study Arms (1)

Patients on SKYTROFA (Lonapegsomatropin)Treatment

SKYTROFA (Lonapegsomatropin) administered once-weekly by subcutaneous injection

Drug: No intervention

Interventions

No intervention

Patients on SKYTROFA (Lonapegsomatropin)Treatment

Eligibility Criteria

Age1 Year - 18 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodNon-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.

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (27)

Ascendis Investigational Site

Phoenix, Arizona, 85054, United States

RECRUITING

Ascendis Investigational Site

Orange, California, 92868, United States

RECRUITING

Ascendis Investigational Site

Sacramento, California, 95821, United States

RECRUITING

Ascendis Investigational Site

San Francisco, California, 94143, United States

RECRUITING

Ascendis Pharma Investigational Site

Centennial, Colorado, 80112, United States

RECRUITING

Ascendis Investigational Site

Washington D.C., District of Columbia, 20010, United States

RECRUITING

Ascendis Investigational Site

Orlando, Florida, 32806, United States

RECRUITING

Ascendis Investigational Site

Orlando, Florida, 32827, United States

RECRUITING

Ascendis Investigational Site

Tampa, Florida, 33612, United States

RECRUITING

Ascendis Investigational Site

Sandy Springs, Georgia, 30350, United States

RECRUITING

Ascendis Investigational Site

Boise, Idaho, 83712, United States

RECRUITING

Ascendis Investigational Site

Indianapolis, Indiana, 46202, United States

RECRUITING

Ascendis Investigational Site

Iowa City, Iowa, 52242, United States

RECRUITING

Ascendis Investigational Site

Louisville, Kentucky, 40202, United States

RECRUITING

Ascendis Investigational Site

Minneapolis, Minnesota, 55455, United States

RECRUITING

Ascendis Investigational Site

Las Vegas, Nevada, 89113, United States

RECRUITING

Ascendis Investigational Site

Lebanon, New Hampshire, 03756, United States

RECRUITING

Ascendis Investigational Site

Manchester, New Hampshire, 03104, United States

RECRUITING

Ascendis Investigational Site

Morristown, New Jersey, 07960, United States

RECRUITING

Ascendis Investigational Site

New Hyde Park, New York, 11042, United States

RECRUITING

Ascendis Investigational Site

Cincinnati, Ohio, 45229, United States

RECRUITING

Ascendis Investigational Site

Portland, Oregon, 97239, United States

RECRUITING

Ascendis Investigational Site

Columbia, South Carolina, 29203, United States

RECRUITING

Ascendis Investigational Site

Dallas, Texas, 75390, United States

RECRUITING

Ascendis Investigational Site

El Paso, Texas, 79907, United States

RECRUITING

Ascendis Investigational Site

Norfolk, Virginia, 23507, United States

RECRUITING

Ascendis Investigational Site

Seattle, Washington, 98105, United States

RECRUITING

MeSH Terms

Conditions

Dwarfism, PituitaryFailure to Thrive

Condition Hierarchy (Ancestors)

DwarfismBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesBone Diseases, EndocrineHypopituitarismPituitary DiseasesHypothalamic DiseasesBrain DiseasesCentral Nervous System DiseasesNervous System DiseasesEndocrine System DiseasesSigns and SymptomsPathological Conditions, Signs and Symptoms

Study Officials

  • Medical Director, MD

    Ascendis Pharma A/S

    STUDY DIRECTOR

Central Study Contacts

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 8, 2023

First Posted

March 20, 2023

Study Start

March 20, 2023

Primary Completion (Estimated)

March 1, 2033

Study Completion (Estimated)

March 1, 2033

Last Updated

February 4, 2026

Record last verified: 2026-02

Data Sharing

IPD Sharing
Will not share

Locations