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Somapacitan in children born small for gestational age: a randomized controlled phase 3 trial
Agnès Linglart1, Volker Böttcher2, Michael Højby3
1Université Paris-Saclay, Inserm, AP-HP, Service d'Endocrinologie et dabète de l'enfant, Hôpital Bicêtre Paris Saclay, Le Kremlin-Bicêtre F-94270, France.
Insights
Once-weekly somapacitan demonstrated efficacy and safety comparable to daily growth hormone (GH) injections for treating short stature in children born small for gestational age (SGA). This long-acting GH option may reduce treatment burden and improve adherence.
Area of Science:
- Pediatric Endocrinology
- Growth Hormone Therapy
- Metabolic Disorders
Background:
- Children born small for gestational age (SGA) often experience short stature, necessitating growth hormone (GH) treatment.
- Current treatment involves daily recombinant GH injections, posing a significant burden on patients and families.
- Exploring long-acting GH formulations aims to improve treatment adherence and outcomes.
Purpose of the Study:
- To evaluate the efficacy and safety of once-weekly somapacitan, a long-acting GH, in prepubertal children born SGA.
- To compare the height velocity and safety profile of somapacitan with daily GH therapy.
- To assess the potential of somapacitan to reduce treatment burden in this pediatric population.
Main Methods:
- A multinational, randomized, open-label, phase 3 trial (REAL8) involving 142 treatment-naïve children born SGA.
- Participants received once-weekly somapacitan or daily GH (0.035 or 0.067 mg/kg/day) subcutaneously for 52 weeks.
- The primary endpoint was height velocity at week 52, with noninferiority and superiority analyses conducted.
Main Results:
- Somapacitan achieved a mean height velocity of 11.0 cm/year, comparable to daily GH (9.4 and 11.1 cm/year).
- Noninferiority of somapacitan to both daily GH doses was confirmed.
- Somapacitan demonstrated superiority over the lower daily GH dose (0.035 mg/kg/day) and showed similar safety profiles across groups.
Conclusions:
- Once-weekly somapacitan is effective and safe for treating short stature in children born SGA over 52 weeks.
- Somapacitan offers comparable efficacy to daily GH but with a reduced treatment burden.
- This long-acting formulation presents a promising alternative to daily injections, potentially enhancing patient adherence and treatment success.
Objective:
Short stature in children born small for gestational age (SGA) is treated with daily injections of recombinant growth hormone (GH), a significant treatment burden. The objective of this study is to demonstrate the efficacy and safety of once-weekly somapacitan, a long-acting GH, in short children born SGA.
Design:
REAL8 (NCT05330325) is a multinational, multicenter, randomized, open-labeled, 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 SGA sub-study.
Methods:
142 prepubertal, treatment-naïve children born SGA in 78 sites across 26 countries were randomized 2:1:1 to somapacitan .24 mg/kg/week or daily GH 0.035 or 0.067 mg/kg/day, all administered subcutaneously. 140 completed the main 52-week treatment period.
Results:
The primary endpoint, estimated mean height velocity at week 52, was 11.0 cm/year for somapacitan vs. 9.4 cm/year [ETD = 1.6(0.91, 2.23)95%CI] and 11.1 cm/year [ETD = -0.1(-0.75, 0.60)95%CI] for daily GH 0.035 and 0.067 mg/kg/day, respectively. Noninferiority was confirmed for somapacitan compared to both daily GH groups. Superiority was demonstrated for somapacitan versus daily GH 0.035 mg/kg/day. Safety profiles were similar between treatment groups. As expected, somapacitan reduced disease burden at week 52, as for daily GH 0.067 mg/kg/day, while somapacitan was associated with reduced treatment burden.
Conclusion:
Somapacitan provides similar efficacy, safety, and tolerability as daily GH in short children born SGA after 52 weeks of treatment. Somapacitan may be an attractive alternative to daily GH in this population, reducing treatment burden to improve adherence and treatment outcomes.
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