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.
Study Type
OBSERVATIONAL
Enrollment
500
Participants will be treated with commercially available somapacitan according to routine clinical practice at the discretion of the treating physician.
University of Glasgow
Glasgow, Scotland, United Kingdom
Number of adverse drug reactions
Measured as count of events.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Number of medication errors (incorrect dose administration)
Measured as count of errors.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Number of participants with incident neoplasm
Measured as number of participants (yes/no).
Time frame: From baseline (week 0) to end of study (up to 10 years)
Number of participants with incident diabetes mellitus type 2
Measured as number of participants (yes/no).
Time frame: From baseline (week 0) to end of study (up to 10 years)
Height velocity
measured as centimeter per year (cm/year).
Time frame: From baseline (week 0) to end of study (up to 10 years)
Change in height velocity standard deviation score (HVSDS)
Measured as score ranging from -10 to +10.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Change in height standard deviation score (HSDS)
Measured as score ranging from -10 to +10.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Change in insulin-like growth factor I (IGF-I) standard deviation score (SDS)
Measured as score ranging from -10 to +10.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Change in bone age (measured as years)
Measured as years.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Change in bone age (measured as months)
Measured as months.
Time frame: From baseline (week 0) to end of study (up to 10 years)
Participants reaching near adult height
Near adult height defined as: height velocity \<2 cm/year over the last 9 months and chronological age \>16 years (males) or \>15 years (females) or bone age \>16 years (males) and \> 15 years (females). Measured as number of participants (yes/no). Only for those who are expected to reach near adult height during the study.
Time frame: at 10 year
Change in height SDS in participants reaching near adult height
Near adult height can be defined as: height velocity \<2 cm/year over the last 9 months and chronological age \>16 years (males) or \>15 years (females) or bone age \>16 years (males) and \> 15 years (females). Measured as score ranging from -10 to +10. Only for those who are expected to reach near adult height during the study.
Time frame: From baseline (week 0) to end of study (up to 10 years)
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