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

Growth Hormone Deficiency Clinical Trial

— SkybriGHt  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05820672
• Other study ID #: ASND0037
• Status: Recruiting
• Start date: March 20, 2023
• Est. completion date: March 2033

Study information

• Verified date: April 2024
• Source: Ascendis Pharma A/S
• Contact: Isabel Couto
• Phone: +1 844-442-7236
• Email: Skybright_contact[@]ascendispharma.com
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical Trial 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

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 900
• Est. completion date: March 2033
• Est. primary completion date: March 2033
• Accepts healthy volunteers: No
• Gender: All
• Age group: 1 Year to 18 Years

Eligibility

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

Related Conditions & MeSH terms

• Growth Hormone Deficiency

Intervention

Other:
• No intervention
No intervention

Locations

Country	Name	City	State
United States	Ascendis Pharma Investigational Site	Centennial	Colorado

Sponsors (1)

Lead Sponsor	Collaborator
Ascendis Pharma Endocrinology Division A/S

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin)	Incidence of Adverse Events and Serious Adverse Events	5 years
Primary	Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin)	Near adult height (cm)	5 years
Secondary	Assess clinical outcome assessments (COAs)	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
Secondary	Assess clinical outcome assessments (COAs)	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