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

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

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).

Study Overview

• Status: Unknown
• Conditions: Endocrine System Diseases; Pituitary Diseases; Growth Hormone Deficiency; Hormones; Pituitary Disease, Anterior
• Intervention / Treatment: Drug: TransCon hGH; Drug: daily hGH

Detailed Description

Not Provided

• Study Type: Interventional
• Enrollment (Anticipated): 150
• Phase: Phase 3

Contacts and Locations

• Study Contact: Name: Lisa Chen, MD; Phone Number: +86-02152999605; Email: lisa.chen@visenpharma.com
• Study Contact Backup: Name: xiangling Wang, MD; Email: xiangling.wang@visenpharma.com

Study Locations

• China
  • Hubei
    • Wuhan, Hubei, China, 430030
      • Recruiting
      • Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,
      • Contact: Xiaoping Luo, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 1 year to 15 years (Child)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: TransCon hGH; TransCon hGH will be self-administered or injected by parents once weekly. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.	Drug: TransCon hGH; Once weekly subcutaneous injection; Other Names: ACP-011
Active Comparator: Daily hGH; Daily hGH will be self-administered or injected by parents once daily. The dose will be adjusted based on subject's weight. The treatment will continue 52 weeks.	Drug: daily hGH; Once daily subcutaneous injection; Other Names: somatropin (rDNA orgin) for injection

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
Incidence of Treatment-Emergent Adverse Events		52 weeks
The plasma concentration	pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group	52 weeks

Collaborators and Investigators

• Sponsor: Visen Pharmaceuticals (Shanghai) Co., Ltd.
• Collaborators: Ascendis Pharma A/S
• Investigators: Principal Investigator: Xiaoping Luo, MD, Tongji Hospital

Study record dates

Study Major Dates

• Study Start (Actual): December 30, 2019
• Primary Completion (Anticipated): April 1, 2022
• Study Completion (Anticipated): April 1, 2022

Study Registration Dates

• First Submitted: March 26, 2020
• First Submitted That Met QC Criteria: March 27, 2020
• First Posted (Actual): March 30, 2020

Study Record Updates

• Last Update Posted (Actual): March 30, 2020
• Last Update Submitted That Met QC Criteria: March 27, 2020
• Last Verified: March 1, 2020

More Information

Terms related to this study

• Keywords: Growth Hormone Deficiency; hGH; TransCon hGH; human growth hormone
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Musculoskeletal Diseases; Hypothalamic Diseases; Bone Diseases; Bone Diseases, Endocrine; Dwarfism; Bone Diseases, Developmental; Hypopituitarism; Pituitary Diseases; Dwarfism, Pituitary; Endocrine System Diseases
• Other Study ID Numbers: CT301-CN; CTR20200399 (Other Identifier: Center for drug evaluation,NMPA)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No