Evidence map›Paper›PMID 35428884›Full record

Trial reportThe Journal of clinical endocrinology and metabolism2022

Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.

Aristides K Maniatis, Samuel J Casella, Ulhas M Nadgir, Paul L Hofman, Paul Saenger, Elena D Chertock, Elena M Aghajanova, Maria Korpal-Szczyrska, Elpis Vlachopapadopoulou, Oleg Malievskiy and 11 more

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

Trial report in The Journal of clinical endocrinology and metabolism, 2022. The graph could read no effect estimate from its abstract, so it casts no vote on the map. It is linked to trial NCT03344458 (enliGHten), which is not on this map. Cited by 21 papers, 2 of them syntheses that pooled it.

0numbers the graph read from it
0cells of the map it votes in
21citing papers in PubMed, 2 pooled it
2.9field-weighted citation impact, top 8% 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.

NCT03344458 phase3completednot on this map

enliGHten: A Multicenter, Phase 3, Long-Term, Open-Label Trial Investigating Safety and Efficacy of TransCon hGH Administered Once-Weekly in Children With Growth Hormone Deficiency (GHD) Who Have Completed a Prior TransCon hGH Clinical Trial

TypeinterventionalSponsorAscendis Pharma A/SRan2017 to 2023Enrolled298ConditionsGrowth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary DiseasesArmsTransCon hGH
3 · Its place in the literature

Who cites it

21 citing papers in PubMed, 2 syntheses or guidelines pooled it, 23 citations in OpenAlex.

  1. Pooled it
  2. Pooled it
  3. Trial
  4. Article
  5. Review
  6. Article
  7. Review
  8. Article
  9. Article
  10. Review
  11. Article
  12. Article
  13. Rationale for Long-acting Growth Hormone Therapy and Future AspectsJournal of clinical research in pediatric endocrinology · 2025
    Review
  14. Article
  15. Article
  16. Current understanding and perspectives on growth and long-acting GH therapy in Japan.Clinical pediatric endocrinology : case reports and clinical investigations : official journal of the Japanese Society for Pediatric Endocrinology · 2025
    Review
  17. New directions in growth hormone treatment in children.Pediatric endocrinology, diabetes, and metabolism · 2025
    Review
  18. Review
  19. Article
  20. Long acting growth hormone (LAGH), an update.Frontiers in pediatrics · 2023
    Review
4 · The record

Corrections and comments

PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.

5 · Who and what money

Authors and funding

21 authors at 12 institutions in 9 countries.

Aristides K ManiatisRocky Mountain Pediatric Endocrinology, Centennial, CO, USA.
Samuel J CasellaChildren's Hospital at Dartmouth-Hitchcock, LebanonNH, USA.
Ulhas M NadgirCenter of Excellence in Diabetes and Endocrinology, Sacramento, CA, USA.
Paul L HofmanLiggins Institute, University of Auckland, Auckland, New Zealand.
Paul SaengerNYU Langone Health, New York, NY, USA.
Elena D ChertockVoronezh State Medical University, Voronezh, Russia.
Elena M AghajanovaYerevan State Medical University, Yerevan, Armenia.
Maria Korpal-SzczyrskaKlinika Pediatrii, Diabetologii i Endokrynologii Uniwersyteckie Centrum Kliniczne, Gdansk, Poland.
Elpis VlachopapadopoulouChildren's Hospital. P. A. Kyriakou, Athens, Greece.
Oleg MalievskiyBashkir State Medical University, Ufa, Russia.
Tetyana ChaychenkoMHI Regional Child Clinical Hospital, Child Endocrinology Center, Kharkiv National Medical University, Kharkiv, Ukraine.
Marco CappaUOC di Endocrinologia, Ospedale Pediatrico Bambino Gesù, IRCCS, Rome, Italy.
Wenjie SongAscendis Pharma, Palo Alto, CA, USA.
Meng MaoAscendis Pharma, Palo Alto, CA, USA.
Per Holse MygindAscendis Pharma, Palo Alto, CA, USA.
Alden R SmithAscendis Pharma, Palo Alto, CA, USA.
Steven D ChesslerAscendis Pharma, Palo Alto, CA, USA.
Allison S KomirenkoAscendis Pharma, Palo Alto, CA, USA.
Michael BeckertAscendis Pharma A/S, Hellerup, Denmark.
Aimee D ShuAscendis Pharma, Palo Alto, CA, USA.ORCID 0000-0003-1801-5289
Paul S ThorntonCook Children's Health Care System, Fort Worth, TX, USA.
Ascendis Pharma (Denmark) · DKBambino Gesù Children's Hospital · ITBashkir State Medical University · RUChildren's Hospital at Dartmouth Hitchcock · USCook Children's Health Care System · USKharkiv National Medical University · UANYU Langone Health · USPanagiotis & Aglaia Kyriakou Children's Hospital · GRUniversity Clinical Centre · PLUniversity of Auckland · NZVoronezh State Medical Academy named after N.N. Burdenko · RUYerevan State Medical University · AM

Funding

Ascendis Pharma Endocrinology Division
6 · The paper itself

Abstract

purposeThe objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess the long-term safety and efficacy of weekly administered long-acting growth hormone lonapegsomatropin in children with growth hormone deficiency.

methodsEligible subjects completing a prior Phase 3 lonapegsomatropin parent trial (heiGHt or fliGHt) were invited to participate. All subjects were treated with lonapegsomatropin. Subjects in the United States switched to the TransCon hGH Auto-Injector when available. Endpoints were long-term safety, annualized height velocity, pharmacodynamics [insulin-like growth factor-1 SD score (SDS) values], and patient- and caregiver-reported assessments of convenience and tolerability.

resultsLonapegsomatropin treatment during enliGHten was associated with continued improvements in height SDS through week 104 in treatment-naïve subjects from the heiGHt trial (-2.89 to -1.37 for the lonapegsomatropin group; -3.0 to -1.52 for the daily somatropin group). Height SDS also continued to improve among switch subjects from the fliGHt trial (-1.42 at fliGHt baseline to -0.69 at week 78). After 104 weeks, the average bone age/chronological age ratio for each treatment group was 0.8 (0.1), showing only minimal advancement of bone age relative to chronological age with continued lonapegsomatropin treatment among heiGHt subjects. Fewer local tolerability reactions were reported with the TransCon hGH Auto-Injector compared with syringe/needle.

conclusionsTreatment with lonapegsomatropin continued to be safe and well-tolerated, with no new safety signals identified. Children treated with once-weekly lonapegsomatropin showed continued improvement of height SDS through the second year of therapy without excess advancement of bone age.

Indexed as

Dwarfism, PituitaryHuman Growth HormoneBody HeightChildGrowth DisordersGrowth HormoneHumansGrowth HormoneHuman Growth Hormonegrowth hormonegrowth hormone deficiencygrowth hormone replacement therapylonapegsomatropinlong-acting growth hormoneTransCon hGH

Identifiers

PMID35428884
PMCPMC9202697
OpenAlexW4223981584

What OpenQuestion holds

Textmetadata
LicenceCC BY-NC-ND
Read underepoch 390

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.