Pediatric endocrinology

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3

J Clin Endocrinol MetabPublished Long-term safety extension of a controlled phase 2 trial

Authors' conclusion

Somapacitan showed similar efficacy and safety in patients who continued somapacitan treatment and those who switched from daily GH to somapacitan. Once-weekly injections may lead to a reduced treatment burden relative to once-daily injections. A plain-language summary of this work is available.

Study context

Population
Prepubertal, GH-naive children with growth hormone deficiency (GHD)50 completed 4 years
Comparison
Somapacitan continued vs switch from daily GH to somapacitan
Outcome
Main outcomes: HV, change from baseline in HV SDS and height SDS, disease burden, and treatment burden for patients and parents/guardians
Follow-up
4 years
Methodological detailsmulticenter, 29 sites in 11 countries
  • Multicenter
  • 29 sites in 11 countries

Main finding

As reported in the abstract

Change from baseline in HV and HV SDS
  • Changes from baseline in height velocity (HV) and HV SD score (SDS) were similar and as expected in both groups

Further findings

Treatment burden (observer-reported)

Reduced treatment burden for patients and parents/guardians when switching from daily GH to somapacitan

Parent/guardian preference

Strongly/very strongly preferred somapacitan over daily GH
81.8%

Original publication

Weekly Somapacitan in GH Deficiency: 4-Year Efficacy, Safety, and Treatment/Disease Burden Results From REAL 3. J Clin Endocrinol Metab. Published 30 March 2023. doi:10.1210/clinem/dgad183

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