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NCT Number: NCT03344458

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

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.

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Key information

Conditions

Growth Hormone Deficiency, Pediatric Bone Diseases Bone Diseases, Developmental Bone Diseases, Endocrine Brain Diseases Central Nervous System Diseases Dwarfism Dwarfism, Pituitary Endocrine System Diseases Failure to Thrive Hormone Deficiency Hypopituitarism Hypothalamic Diseases Musculoskeletal Diseases Nervous System Diseases Pathological Conditions, Signs and Symptoms Pituitary Diseases Signs and Symptoms

Age range

1 year–18 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Ascendis Pharma Investigational Site, Yerevan, Armenia

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Children who have completed a prior phase 3 TransCon hGH trial
  • Children who have not permanently discontinued study drug in the prior trial
  • 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

  • Poorly-controlled diabetes mellitus (HbA1c ≥ 8.0%) or diabetic complications
  • Evidence of closed epiphyses, defined as bone age > 14.0 years for females or > 16.0 years for males
  • Major medical conditions unless approved by Medical Expert
  • Known hypersensitivity to the components of the trial medication
  • Likely to be non-compliant with respect to trial conduct (in regards to the subject and/or the parent/legal guardian/caregiver)
  • Pregnancy
  • Any other reason that in the opinion of the investigator would prevent the subject from completing participation or following the trial schedule

Treatment and study plan

TransCon hGH

Drug

Once weekly subcutaneous injection of TransCon hGH

Primary outcomes

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

    Time frame: Up to Week 208

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

Secondary outcomes

  1. Annualized Height Velocity by Visit

    Time frame: Up to Week 208

    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

    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

    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.

Sponsors and collaborators

Lead sponsor

Ascendis Pharma A/S

Industry

Registry information

Official study title

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

Acronym: enliGHten

Important dates

Study start
2017
Primary completion
2023
Study completion
2023
First posted
Nov 17, 2017
Registry last updated
Jul 7, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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