Clinical trial NCT04326374: Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency
Official title: The Efficacy, Safety and Tolerability of TransCon hGH Administered Weekly Versus Daily hGH in Prepubertal Children With Growth Hormone Deficiency: a Randomized, Open-lable, Active-controlled, Parallel-group Study in China
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE3
Conditions: Growth Hormone Deficiency
Endocrine System Diseases
Hormones
Pituitary Diseases
Pituitary Disease, Anterior
Keywords: Growth Hormone Deficiency
TransCon hGH
human growth hormone
hGH
Lead sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd.
Sponsor class: INDUSTRY
Collaborators: name: Ascendis Pharma A/S
First posted: 2020-03-30
Enrollment: 150
Sex: ALL
Minimum age (years): 3
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
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
Primary purpose: TREATMENT

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

Eligibility criteria: 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.

Primary outcomes:
measure: Annualized height velocity(AHV) at Week 52
timeFrame: 52 Weeks
Secondary outcomes:
measure: AHV and the change from baseline over 52 weeks at each visit
timeFrame: 52 Weeks
measure: Height SDS and the change from baseline over 52 weeks
timeFrame: 52 weeks
measure: 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
timeFrame: 52 weeks
measure: The percentage of participants achieving IGF-1 SDS 0 to +2.0
timeFrame: 52 weeks
measure: Incidence of Treatment-Emergent Adverse Events
timeFrame: 52 weeks
measure: The plasma concentration
timeFrame: 52 weeks
Other outcomes:
Locations:
city: Wuhan
country: China
facility: Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,
state: Hubei
Study officials:
affiliation: Tongji Hospital
name: Xiaoping Luo, MD
role: PRINCIPAL_INVESTIGATOR
References: