Phase 3
N=162
A Phase 3 Trial of the Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Children With Growth Hormone Deficiency (GHD)
Growth Hormone Deficiency, Pediatric · hGH (Human Growth Hormone) · Endocrine System Diseases · Hormones · Pituitary Diseases
Bottom Line
View on ClinicalTrials.gov: NCT02781727 ↗Enrolled (actual)
162
Serious AEs
1.2%
Results posted
Jan 2022
Primary outcome: Primary: Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups — 11.17; 10.31 cm/year — p=0.0088
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Once weekly subcutaneous injection of TransCon hGH (Drug); Once daily subcutaneous injection of Genotropin (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- Ascendis Pharma Endocrinology Division A/S
- Primary completion
- Jan 2019
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Annualized Height Velocity at 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups |
11.17; 10.31 | 0.0088 sig |
| SECONDARY Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] |
81; 39; 12; 10; 1; 1 | — |
| SECONDARY Annualized Height Velocity Over 52 Weeks for Weekly Lonapegsomatropin and Daily hGH Treatment Groups |
13.54; 12.83; 13.28; 12.22; 12.65; 11.21 | — |
| SECONDARY Change in Height Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups |
0.13; 0.12; 0.38; 0.33; 0.68; 0.58 | — |
| SECONDARY Average IGF-1 Standard Deviation Score Over 52 Weeks for the Weekly Lonapegsomatropin and Daily hGH Treatment Groups |
0.31; -0.60; 0.46; -0.51; 0.59; -0.30 | — |
| SECONDARY Number of Participants With Treatment Emergent Anti-hGH Binding Antibody Formation |
6; 2 | — |
Summary
A 52 week trial of TransCon hGH, a long-acting growth hormone product, versus human growth hormone therapy. TransCon hGH will be given once-a-week, human growth hormone (hGH) will be given daily. Approximately 150 prepubertal, hGH-treatment naïve children (males and females) with GHD will be included. Randomization will occur in a 2:1 ratio (TransCon hGH : Genotropin). This is a global trial that will be conducted in Armenia, Australia, Belarus, Bulgaria, Georgia, Greece, Italy, New Zealand, Poland, Romania, Russia, Turkey, Ukraine, and the United States.
Eligibility Criteria
Inclusion Criteria
- Prepubertal children with GHD (either isolated or as part of a multiple pituitary hormone deficiency) in Tanner stage 1 (Tanner 1982) aged:
- Boys: 3-12 years, inclusive
- Girls: 3-11 years, inclusive
- Impaired height (HT) defined as at least 2.0 standard deviations (SD) below the mean height for chronological age and sex (HT SDS ≤ -2.0) according to the 2000 CDC Growth Charts for the United States Methods and Development, available at http://www.cdc.gov/growthcharts/
- Diagnosis of GHD confirmed by 2 different GH stimulation tests, defined as a peak GH level of ≤10 ng/mL, determined with a validated assay
- Bone age (BA) at least 6 months less than chronological age
- Baseline IGF-1 level of at least 1 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1)
- Written, signed informed consent of the parent(s) or legal guardian(s) of the subject and written assent of the subject (if the subject is able to read, understand, and sign)
Exclusion Criteria
- Children with a body weight below 12 kg
- Prior exposure to recombinant hGH or IGF-1 therapy
- Children with past or present intracranial tumor growth as confirmed by a sellar MRI scan (with contrast) at Screening (MRI results from up to 6 months prior to Screening may be accepted)
- Children with psychosocial dwarfism
- Children with idiopathic short stature
- History or presence of malignant disease; any evidence of present tumor growth
- Closed epiphyses
- Major medical conditions and/or presence of contraindication to hGH treatment
- Participation in any other trial of an investigational agent within 3 months prior to Screening
Data sourced from ClinicalTrials.gov (NCT02781727). 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.