A US Non-interventional, Effectiveness and Safety Study of Patients Treated With SKYTROFA (SkybriGHt)

April 15, 2024 updated by: Ascendis Pharma Endocrinology Division A/S

A US Multi-center, Prospective, Non-interventional, Long Term, Effectiveness and Safety Study of Patients Treated With SKYTROFA (Lonapegsomatropin)

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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.

Study Type

Observational

Enrollment (Estimated)

900

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
    • Colorado
      • Centennial, Colorado, United States, 80112
        • Recruiting
        • Ascendis Pharma 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

  • Child
  • Adult

Accepts Healthy Volunteers

No

Sampling Method

Non-Probability Sample

Study Population

Patients being treated with SKYTROFA who are associated to selected endocrinology or pediatric clinics across USA.

Description

Inclusion Criteria:

  • Patients who are on treatment with SKYTROFA (lonapegsomatropin)
  • Patients being clinically managed in USA
  • Patients with an appropriate written informed consent/assent as applicable for the age of the patient

Exclusion Criteria:

  • Patients participating in any interventional clinical study

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 Treatment
Patients on SKYTROFA Treatment managed in USA with appropriate written Informed Consent
Other: No intervention
No intervention

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin)
Time Frame: 5 years
Incidence of Adverse Events and Serious Adverse Events
5 years
Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin)
Time Frame: 5 years
Near adult height (cm)
5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess clinical outcome assessments (COAs)
Time Frame: 5 years
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))
5 years
Assess clinical outcome assessments (COAs)
Time Frame: 5 years
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)
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

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 7, 2023

First Submitted That Met QC Criteria

April 18, 2023

First Posted (Actual)

April 20, 2023

Study Record Updates

Last Update Posted (Actual)

April 16, 2024

Last Update Submitted That Met QC Criteria

April 15, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Keywords

Other Study ID Numbers

  • ASND0037

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 device product

No

product manufactured in and exported from the U.S.

No

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.

Clinical Trials on Growth Hormone Deficiency

Clinical Trials on No intervention

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Kyrgyzstan

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

3
Subscribe