Evidence map›Paper›PMID 34272849›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2021

Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial.

Paul S Thornton, Aristides K Maniatis, Elena Aghajanova, Elena Chertok, Elpis Vlachopapadopoulou, Zhengning Lin, Wenjie Song, Eva Dam Christoffersen, Vibeke Miller Breinholt, Tatiana Kovalenko and 6 more

Registry-linked trialOpen access · bronzeAbstract readClinical Trial, Phase IIIRandomized Controlled Trial
PubMed Full text at PMC Publisher
In one paragraph

Trial report in The Journal of clinical endocrinology and metabolism, 2021. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT02781727 (A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency), which is not on this map. Cited by 55 papers, 5 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
55citing papers in PubMed, 5 pooled it
7.8field-weighted citation impact, top 2% 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.

NCT02781727 phase3completednot on this map

A Multicenter, Phase 3, Randomized, Open-label, Active-controlled, Parallel-group Trial Investigating the Safety, Tolerability, and Efficacy of TransCon hGH Administered Once a Week Versus Standard Daily hGH Replacement Therapy Over 52 Weeks in Prepubertal Children With Growth Hormone Deficiency (GHD)

TypeinterventionalSponsorAscendis Pharma Endocrinology Division A/SRan2016 to 2019Enrolled162ConditionsGrowth Hormone Deficiency, Pediatric, hGH (Human Growth Hormone), Endocrine System Diseases, HormonesArmsOnce weekly subcutaneous injection of TransCon hGH, Once daily subcutaneous injection of Genotropin
3 · Its place in the literature

Who cites it

55 citing papers in PubMed, 5 syntheses or guidelines pooled it, 73 citations in OpenAlex.

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4 · The record

Corrections and comments

5 · Who and what money

Authors and funding

16 authors at 8 institutions in 7 countries.

Paul S ThorntonCook Children's Medical Center, Fort Worth, TX, USA.
Aristides K ManiatisRocky Mountain Pediatric Endocrinology, Centennial, CO, USA.
Elena AghajanovaYerevan State Medical University, Yerevan, Armenia.
Elena ChertokVoronezh State Medical University, Voronezh, Russia.
Elpis VlachopapadopoulouChildren's Hospital P.A. Kyriakou, Athens, Greece.
Zhengning LinAscendis Pharma, Inc, Palo Alto, CA, USA.
Wenjie SongAscendis Pharma, Inc, Palo Alto, CA, USA.
Eva Dam ChristoffersenAscendis Pharma, A/S, Hellerup, Denmark.
Vibeke Miller BreinholtAscendis Pharma, A/S, Hellerup, Denmark.
Tatiana KovalenkoRepublican Children's Clinical Hospital of the Ministry of Health of the Udmurt Republic, Izhevsk, Russia.
Elene GiorgadzeLTD National Institute of Endocrinology, Tbilisi, Georgia.
Maria Korpal-SzczyrskaMedical University of Gdansk, Gdansk, Poland.
Paul L HofmanLiggins Institute, University of Auckland, Auckland, New Zealand.
David B KarpfAscendis Pharma, Inc, Palo Alto, CA, USA.
Aimee D ShuAscendis Pharma, Inc, Palo Alto, CA, USA.ORCID 0000-0003-1801-5289
Michael BeckertAscendis Pharma, A/S, Hellerup, Denmark.
Ascendis Pharma (Denmark) · DKCook Children's Medical Center · USGdańsk Medical University · PLPanagiotis & Aglaia Kyriakou Children's Hospital · GRThe First Republican Clinical Hospital of the Ministry of Health of the Udmurt Republic · RUUniversity of Auckland · NZVoronezh State Medical Academy named after N.N. Burdenko · RUYerevan State Medical University · AM

Funding

No grant is acknowledged in the PubMed record.

6 · The paper itself

Abstract

contextFor children with growth hormone deficiency (GHD), treatment burden with daily somatropin injections [human growth hormone (hGH)] is high, which may lead to poor adherence and suboptimal overall treatment outcomes. Lonapegsomatropin (TransCon hGH) is an investigational long-acting, once-weekly prodrug for the treatment of GHD.

objectiveThe objective of this study was to evaluate the efficacy and safety of once-weekly lonapegsomatropin vs daily somatropin.

designThe heiGHt trial was a randomized, open-label, active-controlled, 52-week Phase 3 trial (NCT02781727).

settingThis trial took place at 73 sites across 15 countries. PATIENTS: This trial enrolled and dosed 161 treatment-naïve, prepubertal patients with GHD.

interventionsPatients were randomized 2:1 to receive lonapegsomatropin 0.24 mg hGH/kg/week or an equivalent weekly dose of somatropin delivered daily.

main outcome measureThe primary end point was annualized height velocity (AHV) at week 52. Secondary efficacy end points included change from baseline in height SD scores (SDS).

resultsLeast squares (LS) mean (SE) AHV at 52 weeks was 11.2 (0.2) cm/year for lonapegsomatropin vs 10.3 (0.3) cm/year for daily somatropin (P = 0.009), with lonapegsomatropin demonstrating both noninferiority and superiority over daily somatropin. LS mean (SE) height SDS increased from baseline to week 52 by 1.10 (0.04) vs 0.96 (0.05) in the weekly lonapegsomatropin vs daily somatropin groups (P = 0.01). Bone age/chronological age ratio, adverse events, tolerability, and immunogenicity were similar between groups.

conclusionsThe trial met its primary objective of noninferiority in AHV and further showed superiority of lonapegsomatropin compared to daily somatropin, with similar safety, in treatment-naïve children with GHD.

Indexed as

ChildDwarfism, PituitaryFemaleFollow-Up StudiesHormone Replacement TherapyHuman Growth HormoneHumansInsulin-Like Growth Factor Binding Protein 3Insulin-Like Growth Factor IMalePrognosisHuman Growth HormoneInsulin-Like Growth Factor Binding Protein 3Insulin-Like Growth Factor I

Identifiers

PMID34272849
PMCPMC8530727
OpenAlexW3180350077

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.