Evidence map›Paper›PMID 37406251›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2023

Effective GH Replacement With Somapacitan in Children With GHD: REAL4 2-year Results and After Switch From Daily GH.

Bradley S Miller, Joanne C Blair, Michael Højby Rasmussen, Aristides Maniatis, Jun Mori, Volker Böttcher, Ho-Seong Kim, Rikke Beck Bang, Michel Polak, Reiko Horikawa

Erratum issued Registry-linked trialOpen access · hybridAbstract readRandomized Controlled TrialClinical Trial, Phase III
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2023. The graph could read no effect estimate from its abstract, so it casts no vote on the map. An erratum has been issued. It is linked to trial NCT03811535 (A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency), which is not on this map. Cited by 19 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
19citing papers in PubMed, 2 pooled it
4.8field-weighted citation impact, top 4% of its field
1 · What the graph read from it

What it found

Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.

The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.

2 · The registry

The trial behind it

Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.

NCT03811535 phase3completednot on this map

A Trial Comparing the Effect and Safety of Once Weekly Dosing of Somapacitan With Daily Norditropin® in Children With Growth Hormone Deficiency

TypeinterventionalSponsorNovo Nordisk A/SRan2019 to 2025Enrolled200ConditionsGrowth Hormone Deficiency in ChildrenArmsSomapacitan, Norditropin®
3 · Its place in the literature

Who cites it

19 citing papers in PubMed, 2 syntheses or guidelines pooled it, 24 citations in OpenAlex.

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  15. New directions in growth hormone treatment in children.Pediatric endocrinology, diabetes, and metabolism · 2025
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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

10 authors at 9 institutions in 7 countries.

Bradley S MillerDivision of Pediatric Endocrinology, University of Minnesota Medical School, MHealth Fairview Masonic Children's Hospital, Minneapolis, MN 55454, USA.ORCID 0000-0003-3663-5473
Joanne C BlairDepartment of Endocrinology, Alder Hey Children's NHS Foundation Trust, Liverpool, L14 5AB, UK.
Michael Højby RasmussenClinical Drug Development, Novo Nordisk A/S, Søborg 2860, Denmark.ORCID 0000-0002-2210-0366
Aristides ManiatisRocky Mountain Pediatric Endocrinology, Centennial, 80112 CO, USA.
Jun MoriDivision of Pediatric Endocrinology and Metabolism, Children's Medical Center, Osaka City General Hospital, Osaka 534-0021, Japan.ORCID 0000-0002-1271-1577
Volker BöttcherDivision of Pediatric Endocrinology and Metabolism, MVZ Endokrinologikum Frankfurt am Main, Frankfurt 60596, Germany.
Ho-Seong KimDepartment of Pediatrics, Severance Children's Hospital, Institute of Endocrinology, Yonsei University College of Medicine, Seoul 03722, Republic of Korea.
Rikke Beck BangBiostatistics Rare Disease and Advanced Therapies, Novo Nordisk A/S, Aalborg 9220, Denmark.
Michel PolakService d'Endocrinologie, Gynécologie et Diabétologie Pédiatriques, Hôpital Universitaire Necker Enfants Malades Paris, Assistance Publique-Hôpitaux de Paris, Paris 75015, France.
Reiko HorikawaDivision of Endocrinology and Metabolism, National Center for Child Health and Development, Tokyo 157-0074, Japan.ORCID 0000-0003-3429-3246
Novo Nordisk (Denmark) · DKAlder Hey Children's NHS Foundation Trust · GBEndokrinologikum · DEHôpital Necker-Enfants Malades · FRNational Center For Child Health and Development · JPOsaka City General Hospital · JPRocky Mountain Diabetes and Osteoporosis Center · USUniversity of Minnesota · USYonsei University · KR

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

contextSomapacitan is a long-acting GH derivative for treatment of GH deficiency (GHD).

objectiveEvaluate the efficacy and tolerability of somapacitan in children with GHD after 2 years of treatment and after the switch from daily GH.

designA randomized, multinational, open-labelled, controlled parallel group phase 3 trial, comprising a 52-week main phase and 3-year safety extension (NCT03811535).

settingEighty-five sites across 20 countries. PATIENTS: A total of 200 treatment-naïve prepubertal patients were randomized and exposed; 194 completed the 2-year period.

interventionsPatients were randomized 2:1 to somapacitan (0.16 mg/kg/wk) or daily GH (0.034 mg/kg/d) during the first year, after which all patients received somapacitan 0.16 mg/kg/wk.

main outcome measuresHeight velocity (HV; cm/year) at week 104. Additional assessments included HV SD score (SDS), height SDS, IGF-I SDS, and observer-reported outcomes.

resultsHV was sustained in both groups between 52 and 104 weeks. At week 104, mean (SD) for HV between weeks 52 and 104 was 8.4 (1.5) cm/year after continuous somapacitan treatment and 8.7 (1.8) cm/year after 1 year of somapacitan treatment following switch from daily GH. Secondary height-related endpoints also supported sustained growth. Mean IGF-I SDS during year 2 was similar between groups and within normal range (-2 to +2). Somapacitan was well tolerated, with no safety or tolerability issues identified. GH patient preference questionnaire results show that most patients and their caregivers (90%) who switched treatment at year 2 preferred once-weekly somapacitan over daily GH treatment.

conclusionsSomapacitan in children with GHD showed sustained efficacy and tolerability for 2 years, and after switching from daily GH. Patients/caregivers switching from daily GH expressed a preference for somapacitan. CLINICAL

trial registrationNCT03811535.

Indexed as

Dwarfism, PituitaryHuman Growth HormoneBody HeightChildGrowth DisordersGrowth HormoneHistidineHumansInsulin-Like Growth Factor IMannitolPhenolGrowth HormoneHistidineHuman Growth HormoneInsulin-Like Growth Factor IMannitolPhenolsomapacitangrowth hormonegrowth hormone deficiencygrowth hormone replacement therapylong-acting growth hormonesomapacitan

Identifiers

PMID37406251
PMCPMC10655534
OpenAlexW4383199098

What OpenQuestion holds

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LicenceCC BY-NC-ND
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Registered trials

Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.