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

Recruiting

• Conditions: Growth Hormone Deficiency
• Interventions: Other: No intervention

• Registration Number: NCT05820672
• Lead Sponsor: Ascendis Pharma Endocrinology Division A/S

Brief Summary

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

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 Timeline

• Study First Submitted: 2023-03-07
• Study Start: 2023-03-20
• Study First Submitted QC: 2023-04-18
• Study First Posted: 2023-04-20
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-01
• Last Update Posted: 2025-05-02
• Primary Completion: 2033-03
• Study Completion: 2033-03

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 900

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 & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
Patients on SKYTROFA Treatment	No intervention	Patients on SKYTROFA Treatment managed in USA with appropriate written Informed Consent

Primary Outcome Measures

Name	Time	Method
Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin)	5 years	Near adult height (cm)
Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin)	5 years	Incidence of Adverse Events and Serious Adverse Events

Secondary Outcome Measures

Name	Time	Method
Assess clinical outcome assessments (COAs)	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)

Trial Locations

Locations (2)

Ascendis Investigational Site; 🇺🇸 Seattle, Washington, United States

Ascendis Pharma Investigational Site; 🇺🇸 Centennial, Colorado, United States