Phase 3
Completed N=298
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
Growth Hormone Deficiency, Pediatric · Endocrine System Diseases · Hormone Deficiency · Pituitary Diseases
Source: ClinicalTrials.gov NCT03344458 ↗
Enrolled (actual)
298
Serious AEs
7.0%
Results posted
May 2024
Primary outcomePrimary: Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] — 226 Participants
◆ Published Evidence
Established
28citations · ~7 / year
Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.
Summary
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.
Linked Publications
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Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] |
226 | — |
| SECONDARY Annualized Height Velocity by Visit |
8.904; 8.560; 7.854; 7.081; 6.462 | — |
| SECONDARY IGF-1 SDS by Visit |
1.210; 1.355; 1.613; 1.453; 1.597 | — |
| SECONDARY Height SDS - Change From Baseline |
0.141; 0.491; 0.841; 1.089; 1.242 | — |
Eligibility Criteria
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
Data sourced from ClinicalTrials.gov (NCT03344458) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.