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Safety and Efficacy Phase 3 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children

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ClinicalTrials.gov Identifier: NCT02968004
Recruitment Status : Active, not recruiting
First Posted : November 18, 2016
Results First Posted : June 25, 2021
Last Update Posted : June 25, 2021
Sponsor:
Information provided by (Responsible Party):
OPKO Health, Inc.

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Pediatric Growth Hormone Deficiency
Interventions Drug: MOD-4023
Drug: Somatropin
Enrollment 224
Recruitment Details  
Pre-assignment Details  
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Period Title: Overall Study
Started 109 115
Completed 108 114
Not Completed 1 1
Reason Not Completed
Adverse Event             1             0
Withdrawal by Subject             0             1
Arm/Group Title MOD-4023 Genotropin Total
Hide Arm/Group Description

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Total of all reporting groups
Overall Number of Baseline Participants 109 115 224
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 109 participants 115 participants 224 participants
7.83  (2.66) 7.61  (2.37) 7.72  (2.51)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 109 participants 115 participants 224 participants
Female
27
  24.8%
36
  31.3%
63
  28.1%
Male
82
  75.2%
79
  68.7%
161
  71.9%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 109 participants 115 participants 224 participants
Hispanic or Latino
11
  10.1%
13
  11.3%
24
  10.7%
Not Hispanic or Latino
98
  89.9%
102
  88.7%
200
  89.3%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race/Ethnicity, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 109 participants 115 participants 224 participants
White
81
  74.3%
86
  74.8%
167
  74.6%
Black or African American
0
   0.0%
2
   1.7%
2
   0.9%
Asian
24
  22.0%
21
  18.3%
45
  20.1%
American Indian or Alaska Native
1
   0.9%
0
   0.0%
1
   0.4%
Native Hawaiian or Other Pacific Islander
0
   0.0%
1
   0.9%
1
   0.4%
Other
3
   2.8%
5
   4.3%
8
   3.6%
Unknown
0
   0.0%
0
   0.0%
0
   0.0%
Multiracial
0
   0.0%
0
   0.0%
0
   0.0%
Not reported
0
   0.0%
0
   0.0%
0
   0.0%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 109 participants 115 participants 224 participants
Colombia 4 4 8
Argentina 1 2 3
United States 17 25 42
Ukraine 13 11 24
United Kingdom 0 3 3
Belarus 1 1 2
India 12 14 26
Russia 8 12 20
Spain 10 9 19
Greece 3 0 3
New Zealand 3 1 4
Canada 3 2 5
South Korea 8 4 12
Turkey 1 0 1
Taiwan 0 2 2
Poland 7 12 19
Mexico 2 0 2
Georgia 5 1 6
Israel 7 8 15
Australia 2 2 4
Bulgaria 2 2 4
1.Primary Outcome
Title Annual Height Velocity
Hide Description Annual Height Velocity in cm. Annual Height Velocity at 12 months is based on the difference between the heights at 12 months and baseline.
Time Frame 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Overall Number of Participants Analyzed 109 115
Least Squares Mean (95% Confidence Interval)
Unit of Measure: cm/year
10.10
(9.58 to 10.63)
9.78
(9.29 to 10.26)
2.Secondary Outcome
Title Height Velocity at 6 Months
Hide Description Height velocity in cm measured after 6 months of treatment. Annualized Height velocity after 6 months is calculated based on the difference between the heights at 6 months and baseline.
Time Frame After 6 months of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Overall Number of Participants Analyzed 109 115
Least Squares Mean (95% Confidence Interval)
Unit of Measure: cm/year
10.59
(9.96 to 11.22)
10.04
(9.47 to 10.62)
3.Secondary Outcome
Title Change in Height Standard Deviation Score (SDS)
Hide Description Change in height Standard Deviation Score (SDS) after 6 and 12 months is calculated based on the difference between the heights at 6 and 12 months and baseline.
Time Frame After 6 and 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
Two subjects discontinued, one from each group.
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Overall Number of Participants Analyzed 109 115
Mean (95% Confidence Interval)
Unit of Measure: Standard Deviation Score
SDS at 6 months
0.54
(0.48 to 0.61)
0.48
(0.42 to 0.54)
SDS at 12 months
0.92
(0.82 to 1.02)
0.87
(0.78 to 0.96)
4.Secondary Outcome
Title Change in Bone Maturation (BM)
Hide Description Annual change in bone age measurements as per Gruelich-Pyle method
Time Frame 52 weeks
Hide Outcome Measure Data
Hide Analysis Population Description
5 participants of MOD-4023 and 13 participants of Genotropin did not have bone age measurements at 12 months.
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Overall Number of Participants Analyzed 104 102
Mean (Standard Deviation)
Unit of Measure: ratio of bone age to chronologic age
0.05  (0.09) 0.06  (0.1)
5.Secondary Outcome
Title Insulin-like Growth Factor-1 (IGF-1) Standard Deviation Score (SDS)
Hide Description Via central lab analysis
Time Frame Baseline and at 12 months
Hide Outcome Measure Data
Hide Analysis Population Description
2 subjects in MOD-4023 and 5 subjects in Genotropin didn't have the measurements at 12 months
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

Overall Number of Participants Analyzed 109 115
Mean (Standard Deviation)
Unit of Measure: Standard Deviation Score (SDS)
Baseline Number Analyzed 109 participants 115 participants
-1.95  (0.89) -1.72  (0.90)
At 12 months Number Analyzed 107 participants 110 participants
0.65  (1.32) -0.69  (1.09)
6.Other Pre-specified Outcome
Title Device
Hide Description Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA
Time Frame 6 weeks
Outcome Measure Data Not Reported
7.Other Pre-specified Outcome
Title Device
Hide Description Proportion of successful single injections out of total number of single injections using the MOD-4023 Pen in the USA at Week 1 based on the Observer Assessment Tool (OAT)
Time Frame 1 week
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title MOD-4023
Hide Arm/Group Description:

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Overall Number of Participants Analyzed 17
Measure Type: Count of Participants
Unit of Measure: Participants
17
 100.0%
Time Frame 1 year
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title MOD-4023 Genotropin
Hide Arm/Group Description

Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)

MOD-4023: Once weekly subcutaneous injection using pre-filled pen device.

Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)

Somatropin: Once daily subcutaneous injection of Genotropin

All-Cause Mortality
MOD-4023 Genotropin
Affected / at Risk (%) Affected / at Risk (%)
Total   0/109 (0.00%)   0/115 (0.00%) 
Hide Serious Adverse Events
MOD-4023 Genotropin
Affected / at Risk (%) Affected / at Risk (%)
Total   3/109 (2.75%)   2/115 (1.74%) 
Infections and infestations     
Chronic tonsillitis   1/109 (0.92%)  0/115 (0.00%) 
Gastroenteritis   1/109 (0.92%)  0/115 (0.00%) 
Pneumonia   1/109 (0.92%)  0/115 (0.00%) 
Tonsillitis   0/109 (0.00%)  1/115 (0.87%) 
Renal and urinary disorders     
Ureterolithiasis   0/109 (0.00%)  1/115 (0.87%) 
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 2%
MOD-4023 Genotropin
Affected / at Risk (%) Affected / at Risk (%)
Total   92/109 (84.40%)   90/115 (78.26%) 
Blood and lymphatic system disorders     
Anaemia   7/109 (6.42%)  7/115 (6.09%) 
Iron deficiency anaemia   3/109 (2.75%)  2/115 (1.74%) 
Thrombocytopenia   0/109 (0.00%)  3/115 (2.61%) 
Ear and labyrinth disorders     
Ear pain   2/109 (1.83%)  7/115 (6.09%) 
Endocrine disorders     
Hypothyroidism   7/109 (6.42%)  3/115 (2.61%) 
Eye disorders     
Conjunctivitis allergic   3/109 (2.75%)  0/115 (0.00%) 
Gastrointestinal disorders     
Abdominal pain   4/109 (3.67%)  2/115 (1.74%) 
Abdominal pain upper   2/109 (1.83%)  6/115 (5.22%) 
Constipation   2/109 (1.83%)  4/115 (3.48%) 
Diarrhoea   3/109 (2.75%)  4/115 (3.48%) 
Nausea   3/109 (2.75%)  1/115 (0.87%) 
Vomiting   8/109 (7.34%)  9/115 (7.83%) 
General disorders     
Injection site erythema   9/109 (8.26%)  0/115 (0.00%) 
Injection site induration   4/109 (3.67%)  1/115 (0.87%) 
Injection site pain   43/109 (39.45%)  29/115 (25.22%) 
Injection site pruritus   6/109 (5.50%)  0/115 (0.00%) 
Injection site swelling   5/109 (4.59%)  0/115 (0.00%) 
Pyrexia   18/109 (16.51%)  16/115 (13.91%) 
Infections and infestations     
Bronchitis   3/109 (2.75%)  9/115 (7.83%) 
Conjunctivitis   3/109 (2.75%)  3/115 (2.61%) 
Enterobiasis   4/109 (3.67%)  2/115 (1.74%) 
Gastroenteritis   4/109 (3.67%)  3/115 (2.61%) 
Gastroenteritis viral   0/109 (0.00%)  3/115 (2.61%) 
Influenza   5/109 (4.59%)  3/115 (2.61%) 
Molluscum contagiosum   3/109 (2.75%)  0/115 (0.00%) 
Nasopharyngitis   25/109 (22.94%)  29/115 (25.22%) 
Otitis externa   4/109 (3.67%)  4/115 (3.48%) 
Otitis media   4/109 (3.67%)  7/115 (6.09%) 
Otitis media acute   1/109 (0.92%)  3/115 (2.61%) 
Pharyngitis   7/109 (6.42%)  5/115 (4.35%) 
Rhinitis   6/109 (5.50%)  1/115 (0.87%) 
Tonsillitis   5/109 (4.59%)  6/115 (5.22%) 
Injury, poisoning and procedural complications     
Arthropod bite   6/109 (5.50%)  1/115 (0.87%) 
Investigations     
Blood creatine phosphokinase increased   2/109 (1.83%)  8/115 (6.96%) 
Blood thyroid stimulating hormone decreased   0/109 (0.00%)  3/115 (2.61%) 
Free fatty acids increased   5/109 (4.59%)  1/115 (0.87%) 
Metabolism and nutrition disorders     
Hypertriglyceridaemia   3/109 (2.75%)  0/115 (0.00%) 
Hypoinsulinaemia   4/109 (3.67%)  3/115 (2.61%) 
Iron deficiency   1/109 (0.92%)  3/115 (2.61%) 
Musculoskeletal and connective tissue disorders     
Arthralgia   5/109 (4.59%)  8/115 (6.96%) 
Pain in extremity   5/109 (4.59%)  5/115 (4.35%) 
Nervous system disorders     
Headache   18/109 (16.51%)  25/115 (21.74%) 
Respiratory, thoracic and mediastinal disorders     
Cough   9/109 (8.26%)  9/115 (7.83%) 
Nasal congestion   1/109 (0.92%)  3/115 (2.61%) 
Oropharyngeal pain   6/109 (5.50%)  4/115 (3.48%) 
Rhinitis allergic   3/109 (2.75%)  1/115 (0.87%) 
Rhinorrhoea   3/109 (2.75%)  4/115 (3.48%) 
Skin and subcutaneous tissue disorders     
Erythema   1/109 (0.92%)  3/115 (2.61%) 
Rash   2/109 (1.83%)  3/115 (2.61%) 
Rash generalised   3/109 (2.75%)  2/115 (1.74%) 
Indicates events were collected by systematic assessment
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: OPKO Health Inc
Organization: OPKO Health Inc
Phone: 3055754100
EMail: contact@opko.com
Layout table for additonal information
Responsible Party: OPKO Health, Inc.
ClinicalTrials.gov Identifier: NCT02968004    
Other Study ID Numbers: CP-4-006
First Submitted: November 13, 2016
First Posted: November 18, 2016
Results First Submitted: May 10, 2021
Results First Posted: June 25, 2021
Last Update Posted: June 25, 2021