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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05820672
Recruitment Status : Recruiting
First Posted : April 20, 2023
Last Update Posted : April 16, 2024
Sponsor:
Information provided by (Responsible Party):
Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division A/S )

Study Description
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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

Condition or disease Intervention/treatment
Growth Hormone Deficiency Other: No intervention

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 Design
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Study Type : Observational [Patient Registry]
Estimated Enrollment : 900 participants
Observational Model: Cohort
Time Perspective: Prospective
Target Follow-Up Duration: 5 Years
Official Title: A US Multi-center, Prospective, Non-interventional, Long Term, Effectiveness and Safety Study of Patients Treated With SKYTROFA (Lonapegsomatropin)
Actual Study Start Date : March 20, 2023
Estimated Primary Completion Date : March 2033
Estimated Study Completion Date : March 2033

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hormones

Groups and Cohorts
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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


Outcome Measures
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Primary Outcome Measures :
  1. Assess the safety of patients treated with SKYTROFA (Lonapegsomatropin) [ Time Frame: 5 years ]
    Incidence of Adverse Events and Serious Adverse Events

  2. Assess the effectiveness of patients treated with SKYTROFA (Lonapegsomatropin) [ Time Frame: 5 years ]
    Near adult height (cm)


Secondary Outcome Measures :
  1. 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))

  2. 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)


Eligibility Criteria
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Ages Eligible for Study:   1 Year to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
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.
Criteria

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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05820672


Contacts
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Contact: Isabel Couto +1 844-442-7236 Skybright_contact@ascendispharma.com

Locations
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United States, Colorado
Ascendis Pharma Investigational Site Recruiting
Centennial, Colorado, United States, 80112
Sponsors and Collaborators
Ascendis Pharma Endocrinology Division A/S
More Information
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Top of Page Study Description Study Design Groups and Cohorts Outcome Measures Eligibility Criteria Contacts and Locations More Information
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Responsible Party: Ascendis Pharma Endocrinology Division A/S
ClinicalTrials.gov Identifier: NCT05820672    
Other Study ID Numbers: ASND0037
First Posted: April 20, 2023    Key Record Dates
Last Update Posted: April 16, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: No
Keywords provided by Ascendis Pharma A/S ( Ascendis Pharma Endocrinology Division A/S ):
Human Growth Hormone
SKYTROFA