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Trial · Interventional

CompletedPhase 3Results postedRegistry data

A Long-Term Trial Investigating Safety and Efficacy of TransCon hGH in Children With Growth Hormone Deficiency Who Have Completed a Prior TransCon hGH Clinical Trial

Short name enliGHten

NCTNCT03344458 (opens ClinicalTrials.gov in a new tab)Other idTransCon hGH CT-301EXTOther id2017-003410-20Other id2017
Open at ClinicalTrials.govAsk about this trial

Overview

Status
Completed
Phase
Phase 3
Enrollment
298 participants (actual)
Sponsor
Ascendis Pharma A/S · Industry
Design
Allocation not applicable · Open label (no masking)
Countries
11 countries

United States, Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, New Zealand, Poland, Russia, Ukraine

Start
19 Dec 2017
Primary completion
21 Feb 2023
Completion
21 Feb 2023
First posted
17 Nov 2017
Last update posted
7 Jul 2026
Conditions
Growth Hormone Deficiency, Pediatric · Endocrine System Diseases · Hormone Deficiency · Pituitary Diseases

Timeline

1 trial · 1 completed · 1 with posted results

  • Completed
  • Ongoing
  • Not yet recruiting
  • Stopped
  • Estimated dates (est.)
  • Results posted
2018202020222024
  1. Ph 3

    enliGHten

    NCT03344458

    19 Dec 2017 to 21 Feb 2023

Registry summary

Text and fields below are the sponsor's entries at ClinicalTrials.gov, shown as posted.

A multicenter, phase 3, long-term extension trial of TransCon hGH administered once-weekly in children with growth hormone deficiency (GHD) who previously participated in a phase 3 TransCon hGH trial. Approximately 300 children (males and females) with GHD will be included. All study participants will receive TransCon hGH. This is a global trial that will be conducted in, but not limited to, the United States, Poland, Bulgaria, Ukraine, Armenia, Russia and Australia.

Interventions

  • TransCon hGHDrug

Arms1

  • Experimental

    TransCon hGH

    Once weekly subcutaneous injection of TransCon hGH

Primary outcomes1

Outcomes the sponsor listed as primary.

  1. Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]

    Time frame · Up to Week 208

    Registry description

    Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary outcomes(3)

  1. Annualized Height Velocity by Visit

    Time frame · Up to Week 208

    Registry description

    Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment

  2. IGF-1 SDS by Visit

    Time frame · Up to Week 208

    Registry description

    Insulin-like Growth Factor-1 (IGF-1) standard deviation score (SDS) by visit with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. IGF-1 SDS is the number of standard deviations above or below the mean IGF-1 level for a child of the same age and sex. The target range for IGF-1 SDS was 0 to +2. An IGF-1 SDS of 0 represents the population mean. If the IGF-1 SDS is \< 0, then the negative score indicates an IGF-1 below the mean IGF-1 for a child of the same age and sex. If the IGF-1 SDS is \> 0, then the positive score indicates an IGF-1 above the mean IGF-1 for a child of the same age and sex. A positive score of IGF-1 SDS indicates a better outcome.

  3. Height SDS - Change From Baseline

    Time frame · Up to Week 208

    Registry description

    Change in Height Standard Deviation Score (SDS) from baseline with long-term dosing of weekly lonapegsomatropin treatment at Week 13, Week 52, Week 104, Week 156, and Week 208. Height SDS is the number of standard deviations above or below the mean height for age and sex. A Standard Deviation Score of 0 equals the population mean. If the Height SDS is \< 0, then the negative score indicates a height below the mean height for a child of the same age and sex. If the Height SDS is \> 0, then the positive score indicates a height above the mean height for a child of the same age and sex. A positive change from baseline in Height SDS indicates improved outcomes.

Posted results

Values are shown as posted at ClinicalTrials.gov, with their units. PepBank has not converted, pooled or compared them.

Primary outcome results

Primary outcomeCount of participants· Up to Week 208

Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]

GroupValue (Participants)
Lonapegsomatropin226
Secondary and other outcome results(3)
Secondary outcomeMean· Up to Week 208

Annualized Height Velocity by Visit

GroupValue (cm/year)Spread
Lonapegsomatropin8.9042.795
Secondary outcomeMean· Up to Week 208

IGF-1 SDS by Visit

GroupValue (Standard Deviation Score)Spread
Lonapegsomatropin1.2101.400
Secondary outcomeMean· Up to Week 208

Height SDS - Change From Baseline

GroupValue (Standard Deviation Score)Spread
Lonapegsomatropin0.1410.125

Adverse events, as posted

Adverse events as posted: participants affected out of participants at risk, by group
GroupDeathsSerious eventsOther events
LonapegsomatropinOnce weekly subcutaneous injection of lonapegsomatropin (TransCon hGH)0 / 29821 / 298225 / 298

Each cell reads participants affected / participants at risk, as the registry posted them.

Registry data comes from ClinicalTrials.gov. Study results come from studies, not from the registry. How PepBank works

Studies0

Studies that cite this trial.

No study cites this trial.

Other registry references

References listed at the registry that are not in PepBank. They link to PubMed.

  • ResultManiatis AK, Casella SJ, Nadgir UM, Hofman PL, Saenger P, Chertock ED, Aghajanova EM, Korpal-Szczyrska M, Vlachopapadopoulou E, Malievskiy O, Chaychenko T, Cappa M, Song W, Mao M, Mygind PH, Smith AR, Chessler SD, Komirenko AS, Beckert M, Shu AD, Thornton PS. Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results. J Clin Endocrinol Metab. 2022 Jun 16;107(7):e2680-e2689. doi: 10.1210/clinem/dgac217. PMID 35428884

Eligibility

Who could join, as posted.

Sex
All
Age
from 1 Year up to 18 Years
Healthy volunteers
Not accepted
Inclusion and exclusion criteria, as posted
Inclusion Criteria: 1. Children who have completed a prior phase 3 TransCon hGH trial 2. Children who have not permanently discontinued study drug in the prior trial 3. Written, signed, informed consent of the parent or legal guardian of the subject and written assent of the subject as required by the IRB/HREC/IEC Exclusion Criteria: 1. Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications 2. Evidence of closed epiphyses, defined as bone age \> 14.0 years for females or \> 16.0 years for males 3. Major medical conditions unless approved by Medical Expert 4. Known hypersensitivity to the components of the trial medication 5. Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver) 6. Pregnancy 7. Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule

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