Efficacy and Safety of Recombinant Human Growth Hormone on Height Velocity in Subjects With Idiopathic Short Stature
Primary Purpose
Growth Disorder, Idiopathic Short Stature
Status
Completed
Phase
Phase 3
Locations
Korea, Republic of
Study Type
Interventional
Intervention
somatropin
Sponsored by
About this trial
This is an interventional treatment trial for Growth Disorder
Eligibility Criteria
Inclusion Criteria:
- Informed consent obtained from subject's parents or legally acceptable representative before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.)
- Pre-pubertal status (males aged from 4 to 11 [both inclusive], females aged from 4 to 9 [both inclusive]): an absence of breast development in females (Tanner 1 only) and testicular volume below 4 mL in males
- Growth hormone level above 10 ng/mL following a stimulation test (test result within 6 months from screening can be used)
- Height below 3 percentile
- Bone age below or equal to 12 year
- Epiphyses confirmed as open in patients at least 10 years or more of age
Exclusion Criteria:
- Known presence of one or more pituitary hormone deficiencies (ACTH (adrenocorticotropic hormone), ADH (antidiuretic hormone), FSH (follicle-stimulating hormone), LH (luteinising hormone), TSH (thyroid-stimulating hormone))
- Known primary hypothyroidism, adrenal insufficiency or hypogonadism (treated or untreated)
- Specific types of growth failure including, but not limited to, known chromosomal abnormalities associated with growth failure and altered sensitivity to growth hormone
- Bone age is advanced over chronological age more than 3 years
- Active malignancy, CNS (central nervous system) trauma, active chemotherapy or radiation therapy for neoplasia
- Prior history of intracranial hypertension
- Hypertrophic cardiomyopathy
Sites / Locations
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
- Novo Nordisk Investigational Site
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Active Comparator
Arm Label
hGH:12months treatment
hGH: 6 month un-treatment + 6 month treatment
Arm Description
Outcomes
Primary Outcome Measures
Height Velocity (Ht-V)
Height velocity (Ht-V) (cm/year) is the change in height per year (after 6 months of treatment). Ht-V was calculated by Novo Nordisk.
Secondary Outcome Measures
Change in Ht-SDS (Height Standard Deviation Score)
Height standard deviation scores (HSDS) were calculated using Korean growth data (reported by the Korea Centre for Disease Control and Prevention). The mean normal range for HSDS is from -2 to +2. Negative scores below -2 indicate a height below normal range, whereas positive scores above +2 indicate a height above normal.
Change in IGF Related Factors: IGF-I (Insulin-like Growth Factor-I)
IGF-I (insulin-like growth factor-1) was measured at Visit 1 (screening),Visit 3 (3 months ± 7 days ),Visit 4 (6 months ± 7 days),Visit 5 (9 months ± 7 days ) and Visit 6 (12 months ± 7 days ). Change of IGF-I from baseline to 6 months treatment was calculated.
Change in IGF Related Factors: IGFBP-3 (Insulin-like Growth Factor Binding Protein-3)
IGFBP-3 was measured at Visit 1(screening), Visit 3 (3 months ± 7 days ), Visit 4 (6 months ± 7 days), 5 (9 months ± 7 days ) and 6 ( 12 months ± 7 days). Change of IGFBP-3 from baseline to 6 months treatment were calculated.
Change in Bone Age
Change in bone age from the baseline to 6 months.
Occurrence of Adverse Events
AEs were collected throughout the trial in both groups.
Ht-V (Height Velocity)
Height velocity (Ht-V) (cm/year) is the change in height per year (after 6 months of treatment). Three sort of Ht-V was calculated from height data at Visit 2 (day 0), 4 (6 months ± 7 days) and 6 (12 months ± 7 days), as follows: Between Visits 2 and 4, between Visit 4 and 6 and between Visit 2 and 6. Ht-V was calculated by Novo Nordisk. It is the difference between Ht-V for the last 6 months and Ht-V for the first 6 months of treatment. This endpoint was only evaluated for Group A as per the trial protocol.
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT01778023
Brief Title
Efficacy and Safety of Recombinant Human Growth Hormone on Height Velocity in Subjects With Idiopathic Short Stature
Official Title
A 12-month, Open-labelled, Randomised, Parallel-group, Multi-centre, Interventional Trial to Evaluate the Efficacy and Safety of Recombinant Human Growth Hormone (hGH) (Norditropin® Nordilet®) Therapy on Height Velocity (Ht-V) in Patients With Idiopathic Short Stature in Korea
Study Type
Interventional
2. Study Status
Record Verification Date
May 2017
Overall Recruitment Status
Completed
Study Start Date
January 17, 2013 (Actual)
Primary Completion Date
December 17, 2014 (Actual)
Study Completion Date
December 17, 2014 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novo Nordisk A/S
4. Oversight
Data Monitoring Committee
No
5. Study Description
Brief Summary
This trial is conducted in Asia. The aim of this trial is to evaluate the efficacy and safety of recombinant human growth hormone (hGH) in subjects with idiopathic short stature in Korea.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Growth Disorder, Idiopathic Short Stature
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Randomized
Enrollment
54 (Actual)
8. Arms, Groups, and Interventions
Arm Title
hGH:12months treatment
Arm Type
Experimental
Arm Title
hGH: 6 month un-treatment + 6 month treatment
Arm Type
Active Comparator
Intervention Type
Drug
Intervention Name(s)
somatropin
Intervention Description
A weekly dosage of 0.469 mg of somatropin per kg of body weight per week will be injected subcutaneously (under the skin) in the evening in 7 days per week.
Primary Outcome Measure Information:
Title
Height Velocity (Ht-V)
Description
Height velocity (Ht-V) (cm/year) is the change in height per year (after 6 months of treatment). Ht-V was calculated by Novo Nordisk.
Time Frame
After 6 months of treatment
Secondary Outcome Measure Information:
Title
Change in Ht-SDS (Height Standard Deviation Score)
Description
Height standard deviation scores (HSDS) were calculated using Korean growth data (reported by the Korea Centre for Disease Control and Prevention). The mean normal range for HSDS is from -2 to +2. Negative scores below -2 indicate a height below normal range, whereas positive scores above +2 indicate a height above normal.
Time Frame
After 6 months of treatment.
Title
Change in IGF Related Factors: IGF-I (Insulin-like Growth Factor-I)
Description
IGF-I (insulin-like growth factor-1) was measured at Visit 1 (screening),Visit 3 (3 months ± 7 days ),Visit 4 (6 months ± 7 days),Visit 5 (9 months ± 7 days ) and Visit 6 (12 months ± 7 days ). Change of IGF-I from baseline to 6 months treatment was calculated.
Time Frame
After 6 months of treatment.
Title
Change in IGF Related Factors: IGFBP-3 (Insulin-like Growth Factor Binding Protein-3)
Description
IGFBP-3 was measured at Visit 1(screening), Visit 3 (3 months ± 7 days ), Visit 4 (6 months ± 7 days), 5 (9 months ± 7 days ) and 6 ( 12 months ± 7 days). Change of IGFBP-3 from baseline to 6 months treatment were calculated.
Time Frame
After 6 months of treatment.
Title
Change in Bone Age
Description
Change in bone age from the baseline to 6 months.
Time Frame
After 6 months of treatment.
Title
Occurrence of Adverse Events
Description
AEs were collected throughout the trial in both groups.
Time Frame
Throughout the trial (12 months)
Title
Ht-V (Height Velocity)
Description
Height velocity (Ht-V) (cm/year) is the change in height per year (after 6 months of treatment). Three sort of Ht-V was calculated from height data at Visit 2 (day 0), 4 (6 months ± 7 days) and 6 (12 months ± 7 days), as follows: Between Visits 2 and 4, between Visit 4 and 6 and between Visit 2 and 6. Ht-V was calculated by Novo Nordisk. It is the difference between Ht-V for the last 6 months and Ht-V for the first 6 months of treatment. This endpoint was only evaluated for Group A as per the trial protocol.
Time Frame
At the first 6 months and the last 6 months in group A
10. Eligibility
Sex
All
Minimum Age & Unit of Time
4 Years
Maximum Age & Unit of Time
11 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Informed consent obtained from subject's parents or legally acceptable representative before any trial-related activities. (Trial-related activities are any procedure that would not have been performed during normal management of the subject.)
Pre-pubertal status (males aged from 4 to 11 [both inclusive], females aged from 4 to 9 [both inclusive]): an absence of breast development in females (Tanner 1 only) and testicular volume below 4 mL in males
Growth hormone level above 10 ng/mL following a stimulation test (test result within 6 months from screening can be used)
Height below 3 percentile
Bone age below or equal to 12 year
Epiphyses confirmed as open in patients at least 10 years or more of age
Exclusion Criteria:
Known presence of one or more pituitary hormone deficiencies (ACTH (adrenocorticotropic hormone), ADH (antidiuretic hormone), FSH (follicle-stimulating hormone), LH (luteinising hormone), TSH (thyroid-stimulating hormone))
Known primary hypothyroidism, adrenal insufficiency or hypogonadism (treated or untreated)
Specific types of growth failure including, but not limited to, known chromosomal abnormalities associated with growth failure and altered sensitivity to growth hormone
Bone age is advanced over chronological age more than 3 years
Active malignancy, CNS (central nervous system) trauma, active chemotherapy or radiation therapy for neoplasia
Prior history of intracranial hypertension
Hypertrophic cardiomyopathy
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Global Clinical Registry (GCR, 1452)
Organizational Affiliation
Novo Nordisk A/S
Official's Role
Study Director
Facility Information:
Facility Name
Novo Nordisk Investigational Site
City
Busan
ZIP/Postal Code
614-735
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Daegu
ZIP/Postal Code
700-721
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
02841
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
03722
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
137-701
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
138-736
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Seoul
ZIP/Postal Code
150-713
Country
Korea, Republic of
Facility Name
Novo Nordisk Investigational Site
City
Suwon
ZIP/Postal Code
443-721
Country
Korea, Republic of
12. IPD Sharing Statement
Citations:
Citation
Safety and Efficacy Evaluation of Human Growth Hormone (GH) Therapy in Patients with Idiopathic Short Stature (ISS) in Korea - a Randomized Controlled Trial; Min Ho Jung, Byung-Kyu Suh, Cheol Woo Ko et al.; 028-042-GH-Pediatrics (posters) ENDO meeting 2016, Boston Massachusetts
Results Reference
result
PubMed Identifier
32595770
Citation
Jung MH, Suh BK, Ko CW, Lee KH, Jin DK, Yoo HW, Hwang JS, Chung WY, Han HS, Prusty V, Kim HS. Efficacy and Safety Evaluation of Human Growth Hormone Therapy in Patients with Idiopathic Short Stature in Korea - A Randomised Controlled Trial. Eur Endocrinol. 2020 Apr;16(1):54-59. doi: 10.17925/EE.2020.16.1.54. Epub 2019 Oct 15.
Results Reference
derived
Links:
URL
http://novonordisk-trials.com
Description
Clinical Trials at Novo Nordisk
Learn more about this trial
Efficacy and Safety of Recombinant Human Growth Hormone on Height Velocity in Subjects With Idiopathic Short Stature
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