Trial Outcomes & Findings for Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children (NCT NCT02500316)
NCT ID: NCT02500316
Last Updated: 2025-04-09
Results Overview
A summary of the annualized HV at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
48 participants
Primary outcome timeframe
8 years
Results posted on
2025-04-09
Participant Flow
Participant milestones
| Measure |
MOD-4023
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Overall Study
STARTED
|
48
|
|
Overall Study
COMPLETED
|
21
|
|
Overall Study
NOT COMPLETED
|
27
|
Reasons for withdrawal
| Measure |
MOD-4023
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Protocol Violation
|
1
|
|
Overall Study
Withdrawal by Subject
|
15
|
|
Overall Study
Final height achieved
|
5
|
|
Overall Study
Due to war activity
|
1
|
Baseline Characteristics
Long Term Follow up Study of Long-acting hGH (MOD-4023) in Growth Hormone Deficient Children
Baseline characteristics by cohort
| Measure |
MOD-4023
n=48 Participants
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Age, Continuous
|
7.67 years
STANDARD_DEVIATION 2.122 • n=99 Participants
|
|
Age, Customized
< 7 years
|
25 Participants
n=99 Participants
|
|
Age, Customized
≥ 7 and < 10 years
|
13 Participants
n=99 Participants
|
|
Age, Customized
≥ 10 and ≤ 14 years
|
10 Participants
n=99 Participants
|
|
Sex: Female, Male
Female
|
16 Participants
n=99 Participants
|
|
Sex: Female, Male
Male
|
32 Participants
n=99 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Black or African American
|
1 Participants
n=99 Participants
|
|
Race (NIH/OMB)
White
|
45 Participants
n=99 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=99 Participants
|
|
Pubertal Status
Tanner I (Prepubertal)
|
47 Participants
n=99 Participants
|
|
Pubertal Status
Tanner II (Early puberty)
|
1 Participants
n=99 Participants
|
|
Pubertal Status
Tanner III (Mid puberty)
|
0 Participants
n=99 Participants
|
|
Pubertal Status
Tanner IV (Late puberty)
|
0 Participants
n=99 Participants
|
|
Pubertal Status
Tanner V (Post pubertal)
|
0 Participants
n=99 Participants
|
PRIMARY outcome
Timeframe: 8 yearsPopulation: Full analysis population
A summary of the annualized HV at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5).
Outcome measures
| Measure |
MOD-4023
n=48 Participants
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Annualized Height Velocity
OLE Year 1
|
7.99 cm/year
Standard Deviation 1.54
|
|
Annualized Height Velocity
OLE Year 4
|
4.63 cm/year
Standard Deviation NA
Standard deviation is not applicable for 1 subject
|
|
Annualized Height Velocity
OLE Year 2
|
7.46 cm/year
Standard Deviation 1.35
|
|
Annualized Height Velocity
OLE Year 3
|
7.12 cm/year
Standard Deviation 1.66
|
|
Annualized Height Velocity
PEN Year 1
|
6.98 cm/year
Standard Deviation 1.89
|
|
Annualized Height Velocity
PEN Year 2
|
6.37 cm/year
Standard Deviation 2.12
|
|
Annualized Height Velocity
PEN Year 3
|
5.31 cm/year
Standard Deviation 1.68
|
|
Annualized Height Velocity
PEN Year 4
|
6.05 cm/year
Standard Deviation 1.74
|
|
Annualized Height Velocity
PEN Year 5
|
6.31 cm/year
Standard Deviation 0.41
|
PRIMARY outcome
Timeframe: 8 yearsPopulation: Full analysis population
A summary of annual change in height SDS at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5). Height SDS (Standard Deviation Score) reflects how an individual's height compares to the average for their age and sex, calculated using Growth Analyzer and based on Switzerland 1989 (Prader) reference. A z-score of 0 indicates a height equal to the population mean, while positive scores indicate above-average heights and negative scores reflect below-average heights.
Outcome measures
| Measure |
MOD-4023
n=48 Participants
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Delta Height SDS Every 12 Months
OLE Year 1
|
0.57 Z-score
Standard Deviation 0.34
|
|
Delta Height SDS Every 12 Months
OLE Year 2
|
0.40 Z-score
Standard Deviation 0.28
|
|
Delta Height SDS Every 12 Months
OLE Year 3
|
0.34 Z-score
Standard Deviation 0.28
|
|
Delta Height SDS Every 12 Months
OLE Year 4
|
0.09 Z-score
Standard Deviation NA
Standard deviation is not applicable for 1 subject
|
|
Delta Height SDS Every 12 Months
PEN Year 1
|
0.27 Z-score
Standard Deviation 0.25
|
|
Delta Height SDS Every 12 Months
PEN Year 2
|
0.21 Z-score
Standard Deviation 0.29
|
|
Delta Height SDS Every 12 Months
PEN Year 3
|
0.10 Z-score
Standard Deviation 0.21
|
|
Delta Height SDS Every 12 Months
PEN Year 4
|
0.18 Z-score
Standard Deviation 0.34
|
|
Delta Height SDS Every 12 Months
PEN Year 5
|
0.07 Z-score
Standard Deviation 0.13
|
SECONDARY outcome
Timeframe: 8 yearsPopulation: Full analysis population
A summary of IGF-I SDS at the end of each year for Periods III/IV (OLE Years 1 - 4) and V (PEN Years 1 - 5). IGF-1 SDS (Standard Deviation Score) reflects how an individual's IGF-1 level compares to the average for their age and sex, based on reference values from Bidlingmaier et al. (2014, The Journal of Clinical Endocrinology \& Metabolism, 99(5):1712-1721). A z-score of 0 indicates a level equal to the population mean, while positive scores indicate above-average levels and negative scores reflect below-average levels. An IGF-1 SDS below -2 suggests deficiency, whereas a score above +2 is considered elevated. In the study, when IGF-1 SDS exceeded +2, a confirmatory test was performed, and if elevation was confirmed, the dose was reduced by 15%.
Outcome measures
| Measure |
MOD-4023
n=48 Participants
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Summary of IGF-1 SDS
PEN Year 3
|
0.86 Z-score
Standard Deviation 0.89
|
|
Summary of IGF-1 SDS
OLE Year 1
|
0.64 Z-score
Standard Deviation 0.96
|
|
Summary of IGF-1 SDS
OLE Year 2
|
0.65 Z-score
Standard Deviation 1.08
|
|
Summary of IGF-1 SDS
OLE Year 3
|
1.05 Z-score
Standard Deviation 0.82
|
|
Summary of IGF-1 SDS
OLE Year 4
|
0.29 Z-score
Standard Deviation NA
Standard deviation is not applicable for 1 subject
|
|
Summary of IGF-1 SDS
PEN Year 1
|
1.29 Z-score
Standard Deviation 0.81
|
|
Summary of IGF-1 SDS
PEN Year 2
|
0.96 Z-score
Standard Deviation 1.15
|
|
Summary of IGF-1 SDS
PEN Year 4
|
1.00 Z-score
Standard Deviation 0.68
|
|
Summary of IGF-1 SDS
PEN Year 5
|
1.45 Z-score
Standard Deviation 0.33
|
Adverse Events
MOD-4023
Serious events: 4 serious events
Other events: 42 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
MOD-4023
n=48 participants at risk
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Gastrointestinal disorders
Gastric disorder
|
2.1%
1/48 • 8 years
|
|
Infections and infestations
Thyroid gland abscess
|
2.1%
1/48 • 8 years
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
2.1%
1/48 • 8 years
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Schwannoma
|
2.1%
1/48 • 8 years
|
Other adverse events
| Measure |
MOD-4023
n=48 participants at risk
Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
MOD-4023: Once weekly injection of long acting r-hGH (MOD-4023) provided as a solution for injection containing 20 or 50 mg/mL MOD-4023 in a single patient use, multi-dose, disposable pre-filled pen (PEN).
|
|---|---|
|
Infections and infestations
Upper respiratory tract infection
|
27.1%
13/48 • 8 years
|
|
Infections and infestations
Bronchitis
|
22.9%
11/48 • 8 years
|
|
Infections and infestations
Nasopharyngitis
|
14.6%
7/48 • 8 years
|
|
Infections and infestations
Rhinitis
|
14.6%
7/48 • 8 years
|
|
Infections and infestations
Tonsillitis
|
10.4%
5/48 • 8 years
|
|
Infections and infestations
Varicella
|
10.4%
5/48 • 8 years
|
|
Infections and infestations
Viral upper respiratory tract infection
|
10.4%
5/48 • 8 years
|
|
Infections and infestations
Ear infection
|
8.3%
4/48 • 8 years
|
|
Infections and infestations
Pneumonia
|
6.2%
3/48 • 8 years
|
|
Infections and infestations
Viral infection
|
6.2%
3/48 • 8 years
|
|
Infections and infestations
Conjunctivitis
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Corona virus infection
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Hand-foot-and-mouth disease
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Lower respiratory tract infection
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Lower respiratory tract infection viral
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Pharyngitis
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Respiratory tract infection
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Respiratory tract infection viral
|
4.2%
2/48 • 8 years
|
|
Infections and infestations
Rotavirus infection
|
4.2%
2/48 • 8 years
|
|
Gastrointestinal disorders
Vomiting
|
6.2%
3/48 • 8 years
|
|
Gastrointestinal disorders
Abdominal pain
|
4.2%
2/48 • 8 years
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.2%
3/48 • 8 years
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
6.2%
3/48 • 8 years
|
|
Musculoskeletal and connective tissue disorders
Osteochondrosis
|
4.2%
2/48 • 8 years
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
4.2%
2/48 • 8 years
|
|
Endocrine disorders
Delayed puberty
|
6.2%
3/48 • 8 years
|
|
Endocrine disorders
Hypothyroidism
|
4.2%
2/48 • 8 years
|
|
Endocrine disorders
Secondary hypogonadism
|
4.2%
2/48 • 8 years
|
|
General disorders
Pyrexia
|
8.3%
4/48 • 8 years
|
|
General disorders
Injection site bruising
|
4.2%
2/48 • 8 years
|
|
General disorders
Injection site erythema
|
4.2%
2/48 • 8 years
|
|
Nervous system disorders
Headache
|
8.3%
4/48 • 8 years
|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
4.2%
2/48 • 8 years
|
|
Metabolism and nutrition disorders
Obesity
|
4.2%
2/48 • 8 years
|
|
Respiratory, thoracic and mediastinal disorders
Rhinitis allergic
|
6.2%
3/48 • 8 years
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
4.2%
2/48 • 8 years
|
|
Eye disorders
Myopia
|
4.2%
2/48 • 8 years
|
|
Injury, poisoning and procedural complications
Clavicle fracture
|
4.2%
2/48 • 8 years
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place