Clinical trial NCT05775523: A Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
Official title: A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Growth Hormone Deficiency
Keywords: Human Growth Hormone
hGH
rhGH
GHD
Long Acting Growth Hormone
Lonapegsomatropin
Prodrug
Growth Failure
Growth Hormone Replacement Therapy
Sustained Release Growth Hormone
Growth Hormone Deficiency
TransCon hGH
Skytrofa
SkyPASS
Lead sponsor: Ascendis Pharma A/S
Sponsor class: INDUSTRY
First posted: 2023-03-20
Enrollment: 500
Sex: ALL
Minimum age (years): 1
Maximum age (years): 18
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_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.

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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Occurrence of neoplasms (benign, malignant and unspecified)
timeFrame: 5 years
measure: Occurrence of type 2 diabetes mellitus
timeFrame: 5 years
Secondary outcomes:
measure: Occurrence of renal, hepatic, immunologic and neurologic adverse events
timeFrame: 5 years
measure: Occurrence of medication errors in patients treated with lonapegsomatropin
timeFrame: 5 years
measure: Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
timeFrame: 5 years
measure: Insulin-like Growth Factor-1 (IGF-1) response to lonapegsomatropin therapy
timeFrame: 5 years
measure: Compare the occurrence of neoplasms (benign, malignant and unspecified) in patients treated with lonapegsomatropin with historical data from literature
timeFrame: 5 years
measure: Compare the occurrence of type 2 diabetes mellitus in patients treated with lonapegsomatropin with historical data from literature
timeFrame: 5 years
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Ascendis Investigational Site
state: Arizona
city: Sacramento
country: United States
facility: Ascendis Investigational Site
state: California
city: San Francisco
country: United States
facility: Ascendis Investigational Site
state: California
city: Centennial
country: United States
facility: Ascendis Pharma Investigational Site
state: Colorado
city: Washington
country: United States
facility: Ascendis Investigational Site
state: District of Columbia
city: Orlando
country: United States
facility: Ascendis Investigational Site
state: Florida
city: Orlando
country: United States
facility: Ascendis Investigational Site
state: Florida
city: Tampa
country: United States
facility: Ascendis Investigational Site
state: Florida
city: Sandy Springs
country: United States
facility: Ascendis Investigational Site
state: Georgia
city: Boise
country: United States
facility: Ascendis Investigational Site
state: Idaho
city: Indianapolis
country: United States
facility: Ascendis Investigational Site
state: Indiana
city: Iowa City
country: United States
facility: Ascendis Investigational Site
state: Iowa
city: Louisville
country: United States
facility: Ascendis Investigational Site
state: Kentucky
city: Minneapolis
country: United States
facility: Ascendis Investigational Site
state: Minnesota
city: Las Vegas
country: United States
facility: Ascendis Investigational Site
state: Nevada
city: Lebanon
country: United States
facility: Ascendis Investigational Site
state: New Hampshire
city: Morristown
country: United States
facility: Ascendis Investigational Site
state: New Jersey
city: New Hyde Park
country: United States
facility: Ascendis Investigational Site
state: New York
city: Portland
country: United States
facility: Ascendis Investigational Site
state: Oregon
city: Columbia
country: United States
facility: Ascendis Investigational Site
state: South Carolina
city: El Paso
country: United States
facility: Ascendis Investigational Site
state: Texas
city: Norfolk
country: United States
facility: Ascendis Investigational Site
state: Virginia
city: Seattle
country: United States
facility: Ascendis Investigational Site
state: Washington
Study officials:
References: