Trial Outcomes & Findings for Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children (NCT NCT01592500)
NCT ID: NCT01592500
Last Updated: 2020-01-23
Results Overview
Annual Height Velocity in cm/year measured after 12 months of treatment
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
56 participants
Primary outcome timeframe
12 months of treatment
Results posted on
2020-01-23
Participant Flow
Participant milestones
| Measure |
MOD-4023 Low Dose
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Overall Study
STARTED
|
14
|
15
|
14
|
13
|
|
Overall Study
COMPLETED
|
13
|
15
|
14
|
11
|
|
Overall Study
NOT COMPLETED
|
1
|
0
|
0
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy Phase 2 Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Baseline characteristics by cohort
| Measure |
MOD-4023 Low Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=14 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 Participants
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
Total
n=53 Participants
Total of all reporting groups
|
|---|---|---|---|---|---|
|
Age, Categorical
<=18 years
|
13 Participants
n=5 Participants
|
15 Participants
n=7 Participants
|
14 Participants
n=5 Participants
|
11 Participants
n=4 Participants
|
53 Participants
n=21 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Age, Categorical
>=65 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
0 Participants
n=4 Participants
|
0 Participants
n=21 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=5 Participants
|
6 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
3 Participants
n=4 Participants
|
17 Participants
n=21 Participants
|
|
Sex: Female, Male
Male
|
10 Participants
n=5 Participants
|
9 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
8 Participants
n=4 Participants
|
36 Participants
n=21 Participants
|
PRIMARY outcome
Timeframe: 12 months of treatmentPopulation: Per Protocol Population
Annual Height Velocity in cm/year measured after 12 months of treatment
Outcome measures
| Measure |
MOD-4023 Low Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 Participants
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Annual Height Velocity
|
10.4 cm/year
Standard Deviation 2.6
|
11.0 cm/year
Standard Deviation 2.3
|
11.9 cm/year
Standard Deviation 3.5
|
12.5 cm/year
Standard Deviation 2.1
|
SECONDARY outcome
Timeframe: After 6 months of treatmentPopulation: Per Protocol Population
Annualized Height Velocity in cm/year measured after 6 months of treatment
Outcome measures
| Measure |
MOD-4023 Low Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 Participants
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Height Velocity at 6 Months
|
11.8 cm/year
Standard Deviation 3.6
|
12.5 cm/year
Standard Deviation 2.4
|
13.5 cm/year
Standard Deviation 5.0
|
15.0 cm/year
Standard Deviation 2.9
|
SECONDARY outcome
Timeframe: After 6 and 12 months of treatmentPopulation: Per Protocol Population
Change in height standard deviation score from baseline (compared to normal population of same age group and sex). Height SDS was calculated as height minus reference mean height divided by SD of the reference mean height
Outcome measures
| Measure |
MOD-4023 Low Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 Participants
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Change in Height Standard Deviation Score (SDS)
After 6 months of treatment
|
0.65 score on a scale
Standard Deviation 0.36
|
0.75 score on a scale
Standard Deviation 0.25
|
0.90 score on a scale
Standard Deviation 0.39
|
1.00 score on a scale
Standard Deviation 0.35
|
|
Change in Height Standard Deviation Score (SDS)
After 12 months of treatment
|
1.09 score on a scale
Standard Deviation 0.53
|
1.19 score on a scale
Standard Deviation 0.49
|
1.45 score on a scale
Standard Deviation 0.61
|
1.51 score on a scale
Standard Deviation 0.47
|
SECONDARY outcome
Timeframe: Once monthly on day 4 after the last dosePopulation: No data were scheduled to be collected for Week 23 in the Genotropin Arm/Group
Change in IGF-1 standard deviation score from reference population mean of same age group and sex (WHO source). IGF-1 SDS was calculated as IGF-1 result minus reference mean IGF-1 result divided by SD of the reference mean IGF-1 value.
Outcome measures
| Measure |
MOD-4023 Low Dose
n=13 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=14 Participants
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 Participants
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Change in IGF-1 Standard Deviation Score
Week 1
|
-1.86 score on a scale
Standard Deviation 0.817
|
-1.94 score on a scale
Standard Deviation 0.899
|
-2.13 score on a scale
Standard Deviation 0.887
|
-2.12 score on a scale
Standard Deviation 0.813
|
|
Change in IGF-1 Standard Deviation Score
Week 10
|
-1.03 score on a scale
Standard Deviation 0.902
|
-0.279 score on a scale
Standard Deviation 0.891
|
-0.001 score on a scale
Standard Deviation 1.164
|
-0.515 score on a scale
Standard Deviation 1.238
|
|
Change in IGF-1 Standard Deviation Score
Week 14
|
-1.07 score on a scale
Standard Deviation 0.553
|
-0.275 score on a scale
Standard Deviation 0.772
|
-0.058 score on a scale
Standard Deviation 1.067
|
-0.557 score on a scale
Standard Deviation 1.216
|
|
Change in IGF-1 Standard Deviation Score
Week 18
|
-0.815 score on a scale
Standard Deviation 0.733
|
0.189 score on a scale
Standard Deviation 0.707
|
-0.083 score on a scale
Standard Deviation 1.299
|
-0.310 score on a scale
Standard Deviation 0.896
|
|
Change in IGF-1 Standard Deviation Score
Week 22
|
-0.958 score on a scale
Standard Deviation 0.673
|
-0.188 score on a scale
Standard Deviation 0.684
|
0.023 score on a scale
Standard Deviation 0.964
|
-0.495 score on a scale
Standard Deviation 1.108
|
|
Change in IGF-1 Standard Deviation Score
Week 23
|
-1.70 score on a scale
Standard Deviation 0.741
|
-1.48 score on a scale
Standard Deviation 0.653
|
-1.47 score on a scale
Standard Deviation 0.865
|
—
|
|
Change in IGF-1 Standard Deviation Score
Month 9
|
-0.119 score on a scale
Standard Deviation 0.921
|
0.184 score on a scale
Standard Deviation 0.905
|
0.261 score on a scale
Standard Deviation 1.108
|
0.124 score on a scale
Standard Deviation 1.071
|
|
Change in IGF-1 Standard Deviation Score
Month 12
|
-0.458 score on a scale
Standard Deviation 1.194
|
-0.029 score on a scale
Standard Deviation 1.296
|
0.358 score on a scale
Standard Deviation 0.709
|
-0.015 score on a scale
Standard Deviation 1.485
|
|
Change in IGF-1 Standard Deviation Score
Week 26
|
-0.725 score on a scale
Standard Deviation 0.801
|
0.088 score on a scale
Standard Deviation 0.803
|
-0.011 score on a scale
Standard Deviation 1.084
|
-0.235 score on a scale
Standard Deviation 0.919
|
Adverse Events
MOD-4023 Low Dose
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
MOD-4023 Middle Dose
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
MOD-4023 High Dose
Serious events: 0 serious events
Other events: 10 other events
Deaths: 0 deaths
Genotropin
Serious events: 0 serious events
Other events: 8 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
MOD-4023 Low Dose
n=13 participants at risk
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 Middle Dose
n=15 participants at risk
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
MOD-4023 High Dose
n=14 participants at risk
Once weekly subcutaneous injection of long acting r-hGH (MOD-4023)
|
Genotropin
n=11 participants at risk
Once daily subcutaneous injection of Somatropin (r-hGH; Genotropin)
|
|---|---|---|---|---|
|
Blood and lymphatic system disorders
Anemia
|
7.7%
1/13
|
13.3%
2/15
|
21.4%
3/14
|
9.1%
1/11
|
|
Blood and lymphatic system disorders
Iron Deficiency Anemia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Ear and labyrinth disorders
Vertigo
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Endocrine disorders
Adrenal Insufficiency
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Endocrine disorders
Hypothyroidism
|
7.7%
1/13
|
6.7%
1/15
|
7.1%
1/14
|
9.1%
1/11
|
|
Endocrine disorders
Secondary Adrenocortical Insufficiency
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Amblyopia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Astigmatism
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Conjunctivitis
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Eye Inflammation
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Eyelid Oedema
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Eye disorders
Hypermetropia
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Gastrointestinal disorders
Abdominal Pain
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Gastrointestinal disorders
Acetonaemic Vomiting
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Gastrointestinal disorders
Dental Caries
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Gastrointestinal disorders
Enteritis
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Gastrointestinal disorders
Nausea
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Gastrointestinal disorders
Toothache
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
9.1%
1/11
|
|
Gastrointestinal disorders
Vomiting
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
General disorders
Asthenia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
General disorders
Chest Pain
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
General disorders
Injection Site Erythema
|
7.7%
1/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
General disorders
Injection Site Haematoma
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
General disorders
Injection Site Pain
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
General disorders
Injection Site Pruritus
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
General disorders
Injection Site Swelling
|
7.7%
1/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
General disorders
Edema Peripheral
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
General disorders
Pyrexia
|
15.4%
2/13
|
6.7%
1/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Hepatobiliary disorders
Biliary dyskinesia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Hepatobiliary disorders
Sphincter of Oddi Dysfunction
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Acute Tonsillitis
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Infections and infestations
Bronchitis
|
23.1%
3/13
|
0.00%
0/15
|
0.00%
0/14
|
18.2%
2/11
|
|
Infections and infestations
Cystitis
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Ear Infection
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Fungal Skin Infection
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Gastroenteritis
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Infections and infestations
Helminthic Infection
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Infections and infestations
Nasopharyngitis
|
15.4%
2/13
|
6.7%
1/15
|
0.00%
0/14
|
18.2%
2/11
|
|
Infections and infestations
Pulpitis Dental
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Infections and infestations
Respiratory Tract Infection
|
15.4%
2/13
|
6.7%
1/15
|
14.3%
2/14
|
27.3%
3/11
|
|
Infections and infestations
Respiratory Tract Infection Viral
|
0.00%
0/13
|
13.3%
2/15
|
7.1%
1/14
|
9.1%
1/11
|
|
Infections and infestations
Rhinitis
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Infections and infestations
Sinusitis
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Infections and infestations
Tracheitis
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Tracheobronchitis
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Infections and infestations
Varicella
|
7.7%
1/13
|
0.00%
0/15
|
14.3%
2/14
|
0.00%
0/11
|
|
Infections and infestations
Viral Infection
|
0.00%
0/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Injury, poisoning and procedural complications
Accidental Overdose
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Injury, poisoning and procedural complications
Meniscus Cyst
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Investigations
Body Temperature Increased
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Investigations
Hemoglobin Decreased
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Investigations
Insulin-Like Growth Factor Increased
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Investigations
Red Blood Cell Count Decreased
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Investigations
Thyroxine Decreased
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
9.1%
1/11
|
|
Metabolism and nutrition disorders
Hypercalcaemia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Metabolism and nutrition disorders
Impaired Fasting Glucose
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Musculoskeletal and connective tissue disorders
Back Pain
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Musculoskeletal and connective tissue disorders
Connective Tissue Disorder
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Musculoskeletal and connective tissue disorders
Pain in Extremity
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin Papilloma
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Nervous system disorders
Headache
|
30.8%
4/13
|
6.7%
1/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Psychiatric disorders
Attention Deficit/Hyperactivity Disorder
|
0.00%
0/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Renal and urinary disorders
Hematuria
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
7.7%
1/13
|
6.7%
1/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Nasal Congestion
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
9.1%
1/11
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis Allergic
|
0.00%
0/13
|
6.7%
1/15
|
7.1%
1/14
|
0.00%
0/11
|
|
Respiratory, thoracic and mediastinal disorders
Snoring
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Skin and subcutaneous tissue disorders
Dermatitis Atopic
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Skin and subcutaneous tissue disorders
Petechia
|
7.7%
1/13
|
0.00%
0/15
|
0.00%
0/14
|
0.00%
0/11
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
0.00%
0/13
|
0.00%
0/15
|
7.1%
1/14
|
0.00%
0/11
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place