Once-weekly somapacitan in children with Noonan syndrome: randomized controlled phase 3 trial
Primary result · Annualized height velocityResult — go to the resultEstimated treatment difference 1.2 95% CI 0.32–2.03Authors' conclusion
Once-weekly somapacitan was confirmed as noninferior 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.
Study context
- Population
- Growth hormone (GH)-treatment-naïve, prepubertal boys (aged 2.5–11 years) and girls (aged 2.5–10 years) with Noonan syndrome; REAL8 Noonan syndrome substudyrandomized 2:1
- Participants
- 77 participants
- Comparison
- Once-weekly somapacitan vs daily GH
- Primary endpoint
- Estimated mean annualized height velocity
- Follow-up
- Week 52
Methodological detailsmultinational, multicenter, open-labeled, active comparator, phase 3, basket study, noninferiority
- Multinational
- Multicenter
- Open-labeled
- Active comparator
- Phase 3
- Basket study
- Noninferiority
Primary result
Annualized height velocity
Estimated treatment difference
1.2
95% CI 0.32–2.03
P < 0.01
By group
Somapacitanvs Daily GH
estimated treatment difference 1.2 (95% CI 0.32–2.03); P < 0.01
As reported in the abstract
Further findings
Estimated change
| Outcome | Somapacitan | Daily GH | Estimated treatment difference (95% CI) | |
|---|---|---|---|---|
| Change in height SD score · baseline to week 52 | 1.07 | 0.75 | 0.32 (0.16–0.48) | |
| −0.200.20.40.6Difference (linear scale) |
Safety
Safety and tolerability
- Somapacitan was well tolerated and had a similar safety profile to daily GH
Original publication
Once-weekly somapacitan in children with Noonan syndrome: randomized controlled phase 3 trial. Eur J Endocrinol. Published 3 March 2026. doi:10.
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