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

Study Overview
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Brief Summary
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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.

Official Title
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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

Conditions
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Growth Hormone Deficiency, Pediatric, Endocrine System Diseases, Hormone Deficiency, Pituitary Diseases

Intervention / Treatment
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* Drug: TransCon hGH


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 1 Year
Maximum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: All study participants will receive TransCon hGH
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: TransCon hGH<br>Once weekly subcutaneous injection of TransCon hGH | Drug: TransCon hGH<br>* Once weekly subcutaneous injection of TransCon hGH<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Incidence of Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability] | Long-term safety and tolerability of weekly TransCon hGH treatment | Up to 4 years | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Annualized height velocity (HV) with long-term dosing of weekly TransCon hGH treatment |  | Up to 4 years | 
| Proportion of subjects with IGF-1 standard deviation score (SDS) in the normal range of 0.0 to +2.0 with long-term dosing of weekly TransCon hGH treatment |  | Up to 4 years | 
| Change in height standard deviation scores with long-term dosing of weekly TransCon hGH treatment |  | Up to 4 years | 
| Incidence of antibodies against TransCon hGH with long-term dosing of weekly TransCon hGH treatment |  | Up to 4 years | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Human Growth Hormone, hGH, GHD, rhGH, Pediatric Growth Hormone Deficiency, Long Acting Growth Hormone, Somatropin, Prodrug, Growth Failure, Growth Hormone Replacement Therapy, Sustained Release Growth Hormone, Growth Hormone Deficiency, TransCon GH