Safety, Tolerability and Efficacy of TransCon hGH Weekly Versus Daily hGH in Chinese Pediatric Growth Hormone Deficiency

Growth Hormone Deficiency Clinical Trial

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04326374
• Other study ID #: CT301-CN
• Secondary ID: CTR20200399
• Status: Recruiting
• Phase: Phase 3
• Start date: December 30, 2019
• Est. completion date: April 2022

Study information

• Verified date: March 2020
• Source: Visen Pharmaceuticals (Shanghai) Co., Ltd.
• Contact: Lisa Chen, MD
• Phone: +86-02152999605
• Email: lisa.chen[@]visenpharma.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

Description:

Not Provided

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 150
• Est. completion date: April 2022
• Est. primary completion date: April 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 3 Years to 17 Years

Eligibility

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.

Related Conditions & MeSH terms

• Dwarfism, Pituitary
• Endocrine System Diseases
• Growth Hormone Deficiency
• Hormones
• Pituitary Disease, Anterior
• Pituitary Diseases

Intervention

Drug:
• TransCon hGH
Once weekly subcutaneous injection
• daily hGH
Once daily subcutaneous injection

Locations

Country	Name	City	State
China	Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,	Wuhan	Hubei

Sponsors (2)

Lead Sponsor	Collaborator
Visen Pharmaceuticals (Shanghai) Co., Ltd.	Ascendis Pharma A/S

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Annualized height velocity(AHV) at Week 52	Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups	52 Weeks
Secondary	AHV and the change from baseline over 52 weeks at each visit	AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit	52 Weeks
Secondary	Height SDS and the change from baseline over 52 weeks	Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks	52 weeks
Secondary	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	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	52 weeks
Secondary	The percentage of participants achieving IGF-1 SDS 0 to +2.0	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	52 weeks
Secondary	Incidence of Treatment-Emergent Adverse Events		52 weeks
Secondary	The plasma concentration	pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group	52 weeks