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

Phase 3

Completed

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

Interventions: Drug: TransCon hGH

Registration Number: NCT03344458

Lead Sponsor: Ascendis Pharma A/S

Brief 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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: All

Target Recruitment: 298

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

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
TransCon hGH	TransCon hGH	Once weekly subcutaneous injection of TransCon hGH

Primary Outcome Measures

Name	Time	Method
Number of Subjects With Treatment-Emergent Adverse Events [Long-Term Safety and Tolerability]	Up to Week 208	Long-term safety and tolerability of weekly lonapegsomatropin (TransCon hGH) treatment

Secondary Outcome Measures

Name	Time	Method
IGF-1 SDS by Visit	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.
Height SDS - Change From Baseline	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.
Annualized Height Velocity by Visit	Up to Week 208	Annualized height velocity (AHV) by visit with long-term dosing of weekly lonapegsomatropin treatment