Diabetes Mellitus Type 2, Growth Hormone Deficiency, Neoplasms
Conditions
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.
Interventions
DRUGSomapacitan
Participants will be treated with commercially available somapacitan according to routine clinical practice at the discretion of the treating physician.
Sponsors
Novo Nordisk A/S
Study design
Observational model
CASE_ONLY
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Age
No minimum to 18 Years
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of adverse drug reactions | From baseline (week 0) to end of study (up to 10 years) | Measured as count of events. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of medication errors (incorrect dose administration) | From baseline (week 0) to end of study (up to 10 years) | Measured as count of errors. |
| Number of participants with incident neoplasm | From baseline (week 0) to end of study (up to 10 years) | Measured as number of participants (yes/no). |
| Number of participants with incident diabetes mellitus type 2 | From baseline (week 0) to end of study (up to 10 years) | Measured as number of participants (yes/no). |
| Height velocity | From baseline (week 0) to end of study (up to 10 years) | measured as centimeter per year (cm/year). |
| Change in height velocity standard deviation score (HVSDS) | From baseline (week 0) to end of study (up to 10 years) | Measured as score ranging from -10 to +10. |
| Change in height standard deviation score (HSDS) | From baseline (week 0) to end of study (up to 10 years) | Measured as score ranging from -10 to +10. |
| Change in insulin-like growth factor I (IGF-I) standard deviation score (SDS) | From baseline (week 0) to end of study (up to 10 years) | Measured as score ranging from -10 to +10. |
| Change in bone age (measured as years) | From baseline (week 0) to end of study (up to 10 years) | Measured as years. |
| Change in bone age (measured as months) | From baseline (week 0) to end of study (up to 10 years) | Measured as months. |
| Participants reaching near adult height | at 10 year | 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. |
| Change in height SDS in participants reaching near adult height | From baseline (week 0) to end of study (up to 10 years) | 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. |
Countries
United Kingdom
Contacts
STUDY_DIRECTORClinical Transparency (dept. 2834)
Novo Nordisk A/S
Outcome results
None listed