search
Back to results

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

Primary Purpose

Growth Hormone Deficiency, Endocrine System Diseases, Hormones

Status
Unknown status
Phase
Phase 3
Locations
China
Study Type
Interventional
Intervention
TransCon hGH
daily hGH
Sponsored by
Visen Pharmaceuticals (Shanghai) Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Growth Hormone Deficiency focused on measuring Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH

Eligibility Criteria

3 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,Recruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

TransCon hGH

Daily hGH

Arm Description

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.

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.

Outcomes

Primary Outcome Measures

Annualized height velocity(AHV) at Week 52
Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups

Secondary Outcome Measures

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
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
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
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
Incidence of Treatment-Emergent Adverse Events
The plasma concentration
pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group

Full Information

First Posted
March 26, 2020
Last Updated
March 27, 2020
Sponsor
Visen Pharmaceuticals (Shanghai) Co., Ltd.
Collaborators
Ascendis Pharma A/S
search

1. Study Identification

Unique Protocol Identification Number
NCT04326374
Brief Title
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

2. Study Status

Record Verification Date
March 2020
Overall Recruitment Status
Unknown status
Study Start Date
December 30, 2019 (Actual)
Primary Completion Date
April 2022 (Anticipated)
Study Completion Date
April 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Visen Pharmaceuticals (Shanghai) Co., Ltd.
Collaborators
Ascendis Pharma A/S

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Hormone Deficiency, Endocrine System Diseases, Hormones, Pituitary Diseases, Pituitary Disease, Anterior
Keywords
Growth Hormone Deficiency, TransCon hGH, human growth hormone, hGH

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
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
Masking
None (Open Label)
Allocation
Randomized
Enrollment
150 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
TransCon hGH
Arm Type
Experimental
Arm Description
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.
Arm Title
Daily hGH
Arm Type
Active Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
TransCon hGH
Other Intervention Name(s)
ACP-011
Intervention Description
Once weekly subcutaneous injection
Intervention Type
Drug
Intervention Name(s)
daily hGH
Other Intervention Name(s)
somatropin (rDNA orgin) for injection
Intervention Description
Once daily subcutaneous injection
Primary Outcome Measure Information:
Title
Annualized height velocity(AHV) at Week 52
Description
Annualized height velocity at Week 52 for weekly TransCon hGH treatment and the daily hGH treatment groups
Time Frame
52 Weeks
Secondary Outcome Measure Information:
Title
AHV and the change from baseline over 52 weeks at each visit
Description
AHV and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks at each visit
Time Frame
52 Weeks
Title
Height SDS and the change from baseline over 52 weeks
Description
Height SDS and the change from baseline for the TransCon hGH and the daily hGH treatment group over 52 weeks
Time Frame
52 weeks
Title
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
Description
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
Time Frame
52 weeks
Title
The percentage of participants achieving IGF-1 SDS 0 to +2.0
Description
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
Time Frame
52 weeks
Title
Incidence of Treatment-Emergent Adverse Events
Time Frame
52 weeks
Title
The plasma concentration
Description
pharmacokinetics measure of hGH over 52 weeks in TransCon hGH group
Time Frame
52 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
3 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
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.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Lisa Chen, MD
Phone
+86-02152999605
Email
lisa.chen@visenpharma.com
First Name & Middle Initial & Last Name or Official Title & Degree
xiangling Wang, MD
Email
xiangling.wang@visenpharma.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaoping Luo, MD
Organizational Affiliation
Tongji Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Department of Pediatrics, Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology,
City
Wuhan
State/Province
Hubei
ZIP/Postal Code
430030
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiaoping Luo, MD

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

We'll reach out to this number within 24 hrs