Clinical trial NCT06709040: A Study to Follow Paediatric Participants With Growth Hormone Deficiency Treated With Somapacitan for Long Term Safety Information and Clinical Parameters
Official title: A Non-interventional, Observational, Registry-based Study to Investigate Long-term Safety and Clinical Parameters of Somapacitan Treatment in Paediatric Patients With Growth Hormone Deficiency During Routine Clinical Practice
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Growth Hormone Deficiency
Neoplasms
Diabetes Mellitus Type 2
Lead sponsor: Novo Nordisk A/S
Sponsor class: INDUSTRY
First posted: 2024-11-29
Enrollment: 500
Sex: ALL
Maximum age (years): 18
Standard ages: CHILD
ADULT
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Paediatric participants with growth hormone deficiency.

Brief summary: The aim of this study is to look at the long-term safety and clinical parameters of somapacitan in paediatric participants with growth hormone deficiency under routine clinical practice conditions. The study population will include 400 paediatric growth hormone deficient participants from the Global Registry for Novel Therapies in Rare Bone and Endocrine Conditions (GloBE-Reg) treated with once-weekly somapacitan and fulfilling the eligibility criteria of the study. The total duration of the study is planned to 10 years consisting of a 5-year recruitment period in the GLoBE-Reg followed by a 5-year follow-up period.


Eligibility criteria: Inclusion Criteria:

* Treated with commercially available somapacitan according to local practice at the discretion of the physician.
* Primary confirmed diagnosis of growth hormone deficiency as per local practice.
* Male or female below 18 years of age at the time of signing informed consent in the GLoBE-Reg.

Exclusion Criteria:

* Participants with active malignancy or in treatment for active pre-existing malignancy.

Primary outcomes:
measure: Number of adverse drug reactions
timeFrame: From baseline (week 0) to end of study (up to 10 years)
Secondary outcomes:
measure: Number of medication errors (incorrect dose administration)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Number of participants with incident neoplasm
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Number of participants with incident diabetes mellitus type 2
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Height velocity
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Change in height velocity standard deviation score (HVSDS)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Change in height standard deviation score (HSDS)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Change in insulin-like growth factor I (IGF-I) standard deviation score (SDS)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Change in bone age (measured as years)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Change in bone age (measured as months)
timeFrame: From baseline (week 0) to end of study (up to 10 years)
measure: Participants reaching near adult height
timeFrame: at 10 year
measure: Change in height SDS in participants reaching near adult height
timeFrame: From baseline (week 0) to end of study (up to 10 years)
Other outcomes:
Locations:
city: Glasgow
country: United Kingdom
facility: University of Glasgow
state: Scotland
Study officials:
affiliation: Novo Nordisk A/S
name: Clinical Transparency (dept. 2834)
role: STUDY_DIRECTOR
References: