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Once-weekly Somapacitan in Children with Noonan Syndrome: Randomised Controlled Phase 3 Trial
Journal article   Open access   Peer reviewed

Once-weekly Somapacitan in Children with Noonan Syndrome: Randomised Controlled Phase 3 Trial

Alexander A L Jorge, Assunta Albanese, Michael Højby, Kamil Soltysik, Thomas Edouard, Anna Grandone, Michael Tansey and Jun Mori
European journal of endocrinology, Vol.194(3), pp.393-403
03/2026
DOI: 10.1093/ejendo/lvag041
PMID: 41774755
url
https://doi.org/10.1093/ejendo/lvag041View
Published (Version of record) Open Access

Abstract

Daily growth hormone (GH) injections are indicated for the treatment of short stature in children with Noonan syndrome, which presents a treatment burden for the child and their parents/caregivers. Somapacitan is a long-acting, reversible albumin-binding GH, developed for once-weekly administration. The objective of this study is to evaluate efficacy, safety and tolerability of somapacitan versus daily GH in children living with Noonan syndrome. REAL8 (NCT05330325) is a multi-national, multi-centre, randomised, open-labelled, active comparator, phase 3 basket study including four non-GH deficiency indications comprising a 52-week main phase and 104-week extension. Here, we present 52-week results from the REAL8Noonan syndrome sub-study. Seventy-seven GH-treatment-naïve, prepubertal boys (aged 2.5 to 11 years) and girls (aged 2.5 to 10 years) with Noonan syndrome were randomized 2:1 to somapacitan 0.24 mg/kg/week or daily GH 0.050 mg/kg/day, administered subcutaneously. The primary endpoint, estimated mean annualized height velocity at week 52, was 10.4 cm/year for somapacitan versus 9.2 cm/year for daily GH (estimated treatment difference [ETD]: 1.2, 95% CI [0.32; 2.03]), confirming non-inferiority and demonstrating superiority of somapacitan compared to daily GH (p<0.01). The estimated change from baseline to week 52 in height standard deviation score was 1.07 and 0.75 for somapacitan and daily GH, respectively (ETD: 0.32, 95% CI [0.16; 0.48]). Somapacitan was well tolerated and had a similar safety profile to daily GH. Once-weekly somapacitan was confirmed as non-inferior and demonstrated superiority to daily GH in HV after 52 weeks of treatment in treatment-naïve children living with Noonan syndrome. Similar safety profiles and tolerability were observed for both groups.
 long-acting growth hormone short stature somapacitan Noonan syndrome growth hormone therapy

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