Trial Outcomes & Findings for Long-Term Treatment With rhIGF-1 in GHIS (NCT NCT00571727)
NCT ID: NCT00571727
Last Updated: 2023-07-06
Results Overview
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Recruitment status
COMPLETED
Study phase
PHASE2/PHASE3
Target enrollment
92 participants
Primary outcome timeframe
Baseline (Pre-dose) and up to 12 years
Results posted on
2023-07-06
Participant Flow
This Phase 3, open-label study was conducted in children with short stature due to severe primary insulin like growth factor-1 deficiency at 2 investigative sites in the US in conjunction with sites in 23 other countries (Argentina, Australia, Austria, Bahamas, Brazil, Canada, Egypt, France, Germany, Iran, Israel, Italy, Kuwait, Pakistan, Poland, Russia, Saudi Arabia, Spain, Sweden, Syria, Taiwan, Ukraine, and Yemen).
Participants who had been treated with mecasermin in previous Genentech-sponsored studies (F0206s, F0375g, F0632g, F0671g), as well as new participants naïve to mecasermin treatment, were enrolled in this study.
Participant milestones
| Measure |
Mecasermin
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 microgram per kilograms (mcg/kg) subcutaneously (SC) twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Overall Study
STARTED
|
92
|
|
Overall Study
COMPLETED
|
26
|
|
Overall Study
NOT COMPLETED
|
66
|
Reasons for withdrawal
| Measure |
Mecasermin
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 microgram per kilograms (mcg/kg) subcutaneously (SC) twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Overall Study
Did not enter in this study
|
1
|
|
Overall Study
Non-compliance
|
4
|
|
Overall Study
Lost to Follow-up
|
30
|
|
Overall Study
Poor growth
|
1
|
|
Overall Study
Parent/patient decision
|
2
|
|
Overall Study
Participant transferred to commercial drug
|
14
|
|
Overall Study
Unable to provide study drug
|
6
|
|
Overall Study
On-treatment at end of study
|
8
|
Baseline Characteristics
Long-Term Treatment With rhIGF-1 in GHIS
Baseline characteristics by cohort
| Measure |
Mecasermin
n=92 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Age, Continuous
|
7.6 years
STANDARD_DEVIATION 4.3 • n=5 Participants
|
|
Sex: Female, Male
Female
|
39 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
53 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
77 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
African American
|
3 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Hispanic
|
6 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Asian
|
4 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
2 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline (Pre-dose) and up to 12 yearsPopulation: The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous recombinant human insulin-like growth factor-1 (rhIGF-1) and whose pre-treatment height velocity were available were reported.
Height velocity is the difference between 2 height measurements, divided by years elapsed between measurements.
Outcome measures
| Measure |
Mecasermin
n=75 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Annualized Height Velocity Up to 12 Years
>= 2 years
|
5.9 centimeter per year (cm/y)
Standard Deviation 1.7
|
|
Annualized Height Velocity Up to 12 Years
Baseline (Pre-dose)
|
2.6 centimeter per year (cm/y)
Standard Deviation 1.7
|
|
Annualized Height Velocity Up to 12 Years
>= 1 year
|
8.0 centimeter per year (cm/y)
Standard Deviation 2.3
|
|
Annualized Height Velocity Up to 12 Years
>= 3 years
|
5.5 centimeter per year (cm/y)
Standard Deviation 1.8
|
|
Annualized Height Velocity Up to 12 Years
>= 4 years
|
5.2 centimeter per year (cm/y)
Standard Deviation 1.5
|
|
Annualized Height Velocity Up to 12 Years
>= 5 years
|
4.9 centimeter per year (cm/y)
Standard Deviation 1.5
|
|
Annualized Height Velocity Up to 12 Years
>= 6 years
|
4.8 centimeter per year (cm/y)
Standard Deviation 1.4
|
|
Annualized Height Velocity Up to 12 Years
>= 7 years
|
4.3 centimeter per year (cm/y)
Standard Deviation 1.5
|
|
Annualized Height Velocity Up to 12 Years
>= 8 years
|
4.4 centimeter per year (cm/y)
Standard Deviation 1.5
|
|
Annualized Height Velocity Up to 12 Years
>= 9 years
|
4.4 centimeter per year (cm/y)
Standard Deviation 1.7
|
|
Annualized Height Velocity Up to 12 Years
>= 10 years
|
4.5 centimeter per year (cm/y)
Standard Deviation 2.0
|
|
Annualized Height Velocity Up to 12 Years
>= 11 years
|
4.1 centimeter per year (cm/y)
Standard Deviation 2.0
|
|
Annualized Height Velocity Up to 12 Years
>= 12 years
|
3.9 centimeter per year (cm/y)
Standard Deviation 2.0
|
PRIMARY outcome
Timeframe: Baseline (Pre-dose) and 1 yearPopulation: The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 were reported.
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Outcome measures
| Measure |
Mecasermin
n=81 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Number of Naive Participants With Height Velocity <5 cm/y at the End of 1 Year of Study Treatment
|
7 Participants
|
SECONDARY outcome
Timeframe: Baseline (Pre-dose) and up to 12 yearsPopulation: The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 and whose pre-treatment height velocity were available were reported.
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height velocity-standard deviation score was calculated as height velocity minus reference mean height velocity divided by standard deviation of the reference mean height velocity. Greater height velocity standard deviation score indicates better outcome.
Outcome measures
| Measure |
Mecasermin
n=75 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Height Velocity Standard Deviation Score Up to 12 Years
Baseline (Pre-dose)
|
-3.4 Standard deviation score
Standard Deviation 1.6
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 1 year
|
1.7 Standard deviation score
Standard Deviation 2.8
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 2 years
|
-0.0 Standard deviation score
Standard Deviation 1.7
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 3 years
|
-0.1 Standard deviation score
Standard Deviation 1.9
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 4 years
|
-0.2 Standard deviation score
Standard Deviation 1.9
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 5 years
|
-0.3 Standard deviation score
Standard Deviation 1.7
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 6 years
|
-0.2 Standard deviation score
Standard Deviation 1.6
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 7 years
|
-0.5 Standard deviation score
Standard Deviation 1.7
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 8 years
|
-0.2 Standard deviation score
Standard Deviation 1.6
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 9 years
|
-0.4 Standard deviation score
Standard Deviation 0.8
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 10 years
|
0.1 Standard deviation score
Standard Deviation 1.6
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 11 years
|
0.5 Standard deviation score
Standard Deviation 2.6
|
|
Height Velocity Standard Deviation Score Up to 12 Years
>= 12 years
|
-0.1 Standard deviation score
Standard Deviation 1.5
|
SECONDARY outcome
Timeframe: Baseline (Pre-dose) and up to 12 yearsPopulation: The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 were reported.
Center for disease control growth charts from the US were used as reference for age and gender-dependent mean and standard deviation. Height standard deviation score was calculated as height minus reference mean height divided by standard deviation of the reference mean height. A higher height standard deviation score indicates a better outcome.
Outcome measures
| Measure |
Mecasermin
n=81 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Height Standard Deviation Score Up to 12 Years
Baseline (Pre-dose)
|
-6.9 Standard deviation score
Standard Deviation 1.8
|
|
Height Standard Deviation Score Up to 12 Years
>= 1 year
|
-6.1 Standard deviation score
Standard Deviation 1.8
|
|
Height Standard Deviation Score Up to 12 Years
>= 2 years
|
-5.6 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 3 years
|
-5.3 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 4 years
|
-5.1 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 5 years
|
-5.0 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 6 years
|
-4.9 Standard deviation score
Standard Deviation 1.6
|
|
Height Standard Deviation Score Up to 12 Years
>= 7 years
|
-4.9 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 8 years
|
-5.1 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 9 years
|
-5.0 Standard deviation score
Standard Deviation 1.6
|
|
Height Standard Deviation Score Up to 12 Years
>= 10 years
|
-5.0 Standard deviation score
Standard Deviation 1.7
|
|
Height Standard Deviation Score Up to 12 Years
>= 11 years
|
-4.7 Standard deviation score
Standard Deviation 1.2
|
|
Height Standard Deviation Score Up to 12 Years
>= 12 years
|
-4.4 Standard deviation score
Standard Deviation 1.3
|
SECONDARY outcome
Timeframe: Baseline (Pre-dose) and up to 19 yearsPopulation: The intention-to-treat population consisted of all 92 participants. Only data from the participants naïve to exogenous rhIGF-1 and who attained near adult height were reported.
Height measurements were performed using wall-mounted stadiometers for analysis of growth data.
Outcome measures
| Measure |
Mecasermin
n=21 Participants
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Approximate Increase in Height Over Expected for Naïve Participants With Near-Adult Height
|
13.3 cm
Standard Deviation 8.4
|
Adverse Events
Mecasermin
Serious events: 18 serious events
Other events: 73 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Mecasermin
n=92 participants at risk
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
2.2%
2/92 • Number of events 3 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Infectious pleural effusion
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Injury, poisoning and procedural complications
Radius fracture
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Appendicitis
|
2.2%
2/92 • Number of events 2 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Haematemesis
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
3.3%
3/92 • Number of events 3 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Nervous system disorders
Benign intracranial hypertension
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Musculoskeletal and connective tissue disorders
Epiphysiolysis
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Abdominal pain
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Cardiac disorders
Dilatation ventricular
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Nervous system disorders
Grand mal convulsion
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Metabolism and nutrition disorders
Diabetes mellitus
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Nervous system disorders
Febrile convulsion
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Injury, poisoning and procedural complications
Skull fracture
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Renal and urinary disorders
Nephrolithiasis
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Metabolism and nutrition disorders
Hypoglycaemic seizure
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Bronchitis
|
1.1%
1/92 • Number of events 1 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
Other adverse events
| Measure |
Mecasermin
n=92 participants at risk
Participants who were entered from previous studies continued to receive mecasermin 80 to 120 mcg/kg SC twice daily and naïve-to-treatment participants were administered mecasermin 60 to 80 mcg/kg SC twice daily for 1 to 2 weeks, and then increased to 120 mcg/kg as tolerated.
|
|---|---|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
44.6%
41/92 • Number of events 175 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Metabolism and nutrition disorders
Hypoglycaemic seizure
|
5.4%
5/92 • Number of events 13 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Injection site hypertrophy
|
34.8%
32/92 • Number of events 211 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Pyrexia
|
20.7%
19/92 • Number of events 77 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Fatigue
|
9.8%
9/92 • Number of events 16 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Injection site haematoma
|
9.8%
9/92 • Number of events 22 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Hypertrophy
|
8.7%
8/92 • Number of events 52 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Asthenia
|
5.4%
5/92 • Number of events 7 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
General disorders
Chest pain
|
5.4%
5/92 • Number of events 10 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Upper respiratory tract infection
|
25.0%
23/92 • Number of events 73 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Otitis media
|
20.7%
19/92 • Number of events 76 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Nasopharyngitis
|
15.2%
14/92 • Number of events 36 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Influenza
|
13.0%
12/92 • Number of events 17 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Bronchitis
|
7.6%
7/92 • Number of events 8 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Ear infection
|
7.6%
7/92 • Number of events 19 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Gastroenteritis
|
7.6%
7/92 • Number of events 9 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Pharyngitis
|
7.6%
7/92 • Number of events 16 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Respiratory tract infection
|
6.5%
6/92 • Number of events 7 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Tonsillitis
|
6.5%
6/92 • Number of events 8 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Infections and infestations
Varicella
|
5.4%
5/92 • Number of events 5 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Snoring
|
21.7%
20/92 • Number of events 111 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Tonsillar hypertrophy
|
20.7%
19/92 • Number of events 102 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
17.4%
16/92 • Number of events 48 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Adenoidal hypertrophy
|
7.6%
7/92 • Number of events 12 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
9.8%
9/92 • Number of events 37 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Vomiting
|
17.4%
16/92 • Number of events 28 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Dental caries
|
12.0%
11/92 • Number of events 45 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Abdominal pain
|
7.6%
7/92 • Number of events 13 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Constipation
|
6.5%
6/92 • Number of events 10 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Diarrhoea
|
6.5%
6/92 • Number of events 14 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
5.4%
5/92 • Number of events 6 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Tooth crowding
|
5.4%
5/92 • Number of events 24 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Gastrointestinal disorders
Toothache
|
5.4%
5/92 • Number of events 11 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Nervous system disorders
Headache
|
27.2%
25/92 • Number of events 109 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Nervous system disorders
Dizziness
|
6.5%
6/92 • Number of events 9 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.7%
8/92 • Number of events 24 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
8.7%
8/92 • Number of events 25 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
7.6%
7/92 • Number of events 18 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Skin and subcutaneous tissue disorders
Acne
|
10.9%
10/92 • Number of events 26 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
10.9%
10/92 • Number of events 31 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Skin and subcutaneous tissue disorders
Skin hypertrophy
|
10.9%
10/92 • Number of events 36 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Skin and subcutaneous tissue disorders
Rash
|
6.5%
6/92 • Number of events 9 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Ear and labyrinth disorders
Conductive deafness
|
12.0%
11/92 • Number of events 70 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
10.9%
10/92 • Number of events 21 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Ear and labyrinth disorders
Deafness
|
6.5%
6/92 • Number of events 50 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Ear and labyrinth disorders
Ear pain
|
6.5%
6/92 • Number of events 9 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Ear and labyrinth disorders
Ear discomfort
|
5.4%
5/92 • Number of events 27 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Investigations
Cardiac murmur
|
7.6%
7/92 • Number of events 10 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Blood and lymphatic system disorders
Thymus enlargement
|
9.8%
9/92 • Number of events 24 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
5.4%
5/92 • Number of events 9 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Eye disorders
Papilloedema
|
5.4%
5/92 • Number of events 7 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Cardiac disorders
Tachycardia
|
6.5%
6/92 • Number of events 6 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Reproductive system and breast disorders
Gynaecomastia
|
6.5%
6/92 • Number of events 18 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Surgical and medical procedures
Ear tube insertion
|
7.6%
7/92 • Number of events 11 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
|
Surgical and medical procedures
Dental operation
|
6.5%
6/92 • Number of events 6 • Treatment-emergent adverse events were collected from first date of mecasermin intake until last dose, approximately 19 years
The safety population consisted of all participants who had received at least 1 dose of mecasermin treatment. Adverse events were not collected per dose level as pre-specified.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: OTHER