Endocrine System Diseases, Growth Hormone Deficiency, Hormones, Pituitary Disease, Anterior, Pituitary Diseases
Conditions
Keywords
Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH
Brief summary
This study is conducted in China only. The purpose is to demonstrate the efficacy and safety of once weekly dosing of TransCon hGH, a long-acting growth hormone product, compare to once-daily dosing of human growth hormone (hGH) after 52 weeks of treatment in prepubertal children with growth hormone deficiency (GHD).
Detailed description
Not Provided
Interventions
DRUGTransCon hGH
Once weekly subcutaneous injection
DRUGdaily hGH
Once daily subcutaneous injection
Sponsors
Ascendis Pharma A/S
Visen Pharmaceuticals (Shanghai) Co., Ltd.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Eligible subjects will be randomized to 1 of the 2 treatment groups: either weekly TransCon hGH or daily hGH, in a 2:1 ratio
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
* Prepubertal children with GHD in Tanner stage 1, aged of 3 years and below 17 years; * Impaired 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 Chinese 2005 standard； * 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 the chronological age； * Baseline IGF-1 level of at least 1.0 SD below the mean IGF-1 level standardized for age and sex (IGF-1 SDS ≤-1.0)； * 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 8 years old or above).
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 dye recommended) at Screening * Children born SGA (birth weight ≤10th percentile for gestational age according to the Chinese reference)； * Children with psychosocial dwarfism； * Children with idiopathic short stature； * Other causes of short stature such as coeliac disease, hypothyroidism, or rickets； * History or presence of malignant disease; any evidence of present tumor growth; * Subjects with diabetes mellitus； * 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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Annualized height velocity(AHV) at Week 52 | 52 Weeks | Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Height SDS and the change from baseline over 52 weeks | 52 weeks | Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks |
| Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline over 52 weeks at each visit | 52 weeks | Serum IGF-1 and IGFBP-3 levels, and IGF-1 SDS, IGFBP-3 SDS, and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit |
| AHV and the change from baseline over 52 weeks at each visit | 52 Weeks | AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit |
| Incidence of Treatment-Emergent Adverse Events | 52 weeks | — |
| The plasma concentration | 52 weeks | pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group |
| The percentage of participants achieving IGF-1 SDS 0 to +2.0 | 52 weeks | The normalization of IGF-1 SDS (percentage of participants achieving IGF-1 SDS 0 to +2.0 over 52 weeks for the TransCon hGH and the daily hGH treatment group |
Countries
China
Contacts
Primary ContactLisa Chen, MD
Backup Contactxiangling Wang, MD
Outcome results
None listed