Trial Outcomes & Findings for An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia (NCT NCT01078675)
NCT ID: NCT01078675
Last Updated: 2015-04-07
Results Overview
Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
315 participants
Primary outcome timeframe
At Month 3, Month 12 and Month 24
Results posted on
2015-04-07
Participant Flow
250 patients with FH were screened. Additionally, 65 healthy siblings (HS) were enrolled. HS refers to healthy subjects that were siblings of either the study participants or other paediatric patients with HeFH that were not participating in the study. HS were enrolled to have assessments of cIMT, but did not participate further.
Participant milestones
| Measure |
Rosuvastatin
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Overall Study
STARTED
|
250
|
65
|
|
Overall Study
Patients Treated
|
198
|
0
|
|
Overall Study
COMPLETED
|
182
|
59
|
|
Overall Study
NOT COMPLETED
|
68
|
6
|
Reasons for withdrawal
| Measure |
Rosuvastatin
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Overall Study
Withdrawal by Subject
|
7
|
3
|
|
Overall Study
Protocol Violation
|
56
|
0
|
|
Overall Study
Provided in CRF for specific reasons
|
2
|
3
|
|
Overall Study
Adverse Event
|
3
|
0
|
Baseline Characteristics
An Study to Evaluate Rosuvastatin in Children and Adolescents With Familial Hypercholesterolaemia
Baseline characteristics by cohort
| Measure |
Rosuvastatin
n=198 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
n=65 Participants
Controls to HeFH patients in the cIMT evaluations
|
Total
n=263 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
Years
|
11.6 Years
STANDARD_DEVIATION 3.33 • n=93 Participants
|
11.5 Years
STANDARD_DEVIATION 3.53 • n=4 Participants
|
11.55 Years
STANDARD_DEVIATION 3.43 • n=27 Participants
|
|
Sex: Female, Male
Female
|
110 Participants
n=93 Participants
|
32 Participants
n=4 Participants
|
142 Participants
n=27 Participants
|
|
Sex: Female, Male
Male
|
88 Participants
n=93 Participants
|
33 Participants
n=4 Participants
|
121 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
Caucasian
|
178 Participants
n=93 Participants
|
53 Participants
n=4 Participants
|
231 Participants
n=27 Participants
|
|
Race/Ethnicity, Customized
non-Caucasian
|
20 Participants
n=93 Participants
|
12 Participants
n=4 Participants
|
32 Participants
n=27 Participants
|
PRIMARY outcome
Timeframe: At Month 3, Month 12 and Month 24Population: Intent-to-treat analysis set and Per-protocol analysis set
Negative values represent a decrease and positive values represent an increase. In total, 198 patients were treated. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Percent Change From Baseline in LDL-C
LDL-C %Change from Baseline at month 3
|
-37.86 Percentage change
Standard Deviation 14.392
|
—
|
|
Percent Change From Baseline in LDL-C
LDL-C %Change from Baseline at month 12
|
-43.67 Percentage change
Standard Deviation 14.896
|
—
|
|
Percent Change From Baseline in LDL-C
LDL-C %Change from Baseline at month 24
|
-42.88 Percentage change
Standard Deviation 18.222
|
—
|
PRIMARY outcome
Timeframe: At BaselinePopulation: Safety analysis set
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Sexual Maturation by Tanner Staging at Baseline
Tanner Stage I at Baseline
|
81 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Baseline
Tanner Stage II at Baseline
|
32 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Baseline
Tanner Stage III at Baseline
|
18 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Baseline
Tanner Stage IV at Baseline
|
44 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Baseline
Tanner Stage V at Baseline
|
21 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Baseline
Not Recorded at Baseline
|
1 Participants
|
—
|
PRIMARY outcome
Timeframe: Serial blood samples over 24 hours.Population: Single dose PK analysis set
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Outcome measures
| Measure |
Rosuvastatin
n=12 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Single Dose PK - Cmax
|
3.5717 ng/mL
Standard Deviation 3.22350
|
—
|
PRIMARY outcome
Timeframe: At Month 12 and Month 24Population: Safety Population
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Percent Change From Baseline in Height
%Change from Baseline at Month 12
|
3.20 Percent change
Standard Deviation 2.023
|
—
|
|
Percent Change From Baseline in Height
%Change from Baseline at Month 24
|
5.91 Percent change
Standard Deviation 3.968
|
—
|
PRIMARY outcome
Timeframe: At BaselinePopulation: Safety analysis set
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Sexual Maturation by Tanner Staging at Month 12
Tanner Stage I at Month 12
|
61 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 12
Tanner Stage II at Month 12
|
31 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 12
Tanner Stage III at Month 12
|
21 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 12
Tanner Stage IV at Month 12
|
32 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 12
Tanner Stage V at Month 12
|
42 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 12
Not Recorded at Month 12
|
10 Participants
|
—
|
PRIMARY outcome
Timeframe: At BaselinePopulation: Safety analysis set
Tanner stages (I-V) was used to characterize physical development in children and adolescent. The stages was based on external primary and secondary sex characteristics, such as the size of the breasts, genitalia, and development of pubic hair. Tanner stage is considered going up when the organs grow bigger.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Sexual Maturation by Tanner Staging at Month 24
Tanner Stage I at Month 24
|
43 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 24
Tanner Stage II at Month 24
|
33 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 24
Tanner Stage III at Month 24
|
23 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 24
Tanner Stage IV at Month 24
|
32 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 24
Tanner Stage V at Month 24
|
64 Participants
NA
|
—
|
|
Sexual Maturation by Tanner Staging at Month 24
Not Recorded at Month 24
|
2 Participants
|
—
|
PRIMARY outcome
Timeframe: Serial blood samples over 24 hoursPopulation: Single dose PK analysis set
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Outcome measures
| Measure |
Rosuvastatin
n=12 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Single Dose PK - Tmax
|
2.664 hr
Standard Deviation 1.8851
|
—
|
PRIMARY outcome
Timeframe: Serial blood samples over 24 hoursPopulation: Single dose PK analysis set
Serial plasma samples were taken at baseline (Week 0) at: 0.5 hours pre-dose and at 0.5, 1, 2, 3, 4, 5, 6, 9, 12 hours and on Day 1 at 24 hours after the single 10 mg dosing
Outcome measures
| Measure |
Rosuvastatin
n=12 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Single Dose PK - AUC(0-24)
|
27.675 ng*hr/mL
Standard Deviation 26.6417
|
—
|
SECONDARY outcome
Timeframe: At Month 3, Month 12 and Month 24Population: Intent-to-treat analysis set (LOCF)
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC/HDL-C %Change from Baseline at Month 3
|
-31.77 Percent change
Standard Deviation 14.874
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC/HDL-C %Change from Baseline at Month 12
|
-36.54 Percent change
Standard Deviation 14.474
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
HDL-C %Change from Baseline at Month 3
|
5.67 Percent change
Standard Deviation 17.445
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
HDL-C %Change from Baseline at Month 12
|
6.35 Percent change
Standard Deviation 16.725
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
HDL-C %Change from Baseline at Month 24
|
11.73 Percent change
Standard Deviation 19.996
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC %Change from Baseline at Month 3
|
-29.60 Percent change
Standard Deviation 11.433
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC %Change from Baseline at Month 12
|
-33.91 Percent change
Standard Deviation 12.050
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC %Change from Baseline at Month 24
|
-32.03 Percent change
Standard Deviation 14.530
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Triglycerides %Change from Baseline at Month 3
|
-7.95 Percent change
Standard Deviation 34.482
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Triglycerides %Change from Baseline at Month 12
|
-7.85 Percent change
Standard Deviation 37.530
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Triglycerides %Change from Baseline at Month 24
|
-0.12 Percent change
Standard Deviation 37.682
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL C %Change from Baseline at Month 3
|
-36.35 Percent change
Standard Deviation 13.368
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL C %Change from Baseline at Month 12
|
-41.66 Percent change
Standard Deviation 14.235
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL C %Change from Baseline at Month 24
|
-40.40 Percent change
Standard Deviation 17.555
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
LDL-C/HDL-C %Change from Baseline at Month 3
|
-39.66 Percent change
Standard Deviation 17.381
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
LDL-C/HDL-C %Change from Baseline at Month 12
|
-45.63 Percent change
Standard Deviation 17.082
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
LDL-C/HDL-C %Change from Baseline at Month 24
|
-46.95 Percent change
Standard Deviation 20.126
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
TC/HDL-C %Change from Baseline at Month 24
|
-37.39 Percent change
Standard Deviation 17.079
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Trig/HDL-C %Change from Baseline at Month 3
|
-9.05 Percent change
Standard Deviation 41.765
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Trig/HDL-C %Change from Baseline at Month 12
|
-10.50 Percent change
Standard Deviation 40.633
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
Trig/HDL-C %Change from Baseline at Month 24
|
-7.12 Percent change
Standard Deviation 40.585
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL-C/HDL-C %Change from Baseline at Month 3
|
-37.98 Percent change
Standard Deviation 17.369
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL-C/HDL-C %Change from Baseline at Month 12
|
-43.71 Percent change
Standard Deviation 16.731
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
non-HDL-C/HDL-C %Change from Baseline at Month 24
|
-44.74 Percent change
Standard Deviation 19.896
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoA-I %Change from Baseline at Month 3
|
4.77 Percent change
Standard Deviation 14.680
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoA-I %Change from Baseline at Month 12
|
1.41 Percent change
Standard Deviation 13.747
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoA-I %Change from Baseline at Month 24
|
2.34 Percent change
Standard Deviation 15.027
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB %Change from Baseline at Month 3
|
-29.29 Percent change
Standard Deviation 12.456
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB %Change from Baseline at Month 12
|
-35.65 Percent change
Standard Deviation 12.424
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB %Change from Baseline at Month 24
|
-35.72 Percent change
Standard Deviation 15.710
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB/ApoA-I %Change from Baseline at Month 3
|
-31.30 Percent change
Standard Deviation 15.524
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB/ApoA-I %Change from Baseline at Month 12
|
-35.66 Percent change
Standard Deviation 13.920
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
ApoB/ApoA-I %Change from Baseline at Month 24
|
-35.94 Percent change
Standard Deviation 18.743
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
hsCRP %Change from Baseline at Month 3
|
512.57 Percent change
Standard Deviation 4724.249
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
hsCRP %Change from Baseline at Month 12
|
42.96 Percent change
Standard Deviation 226.954
|
—
|
|
Percent Change From Baseline in HDL-C, TC, TG, Non-HDL-C, LDL-C/HDL-C, TC/HDL-C, Non HDL C/HDL-C, ApoB, ApoA-1, and ApoB/ApoA-1
hsCRP %Change from Baseline at Month 24
|
98.36 Percent change
Standard Deviation 565.444
|
—
|
SECONDARY outcome
Timeframe: At Month 12 and Month 24Population: Intent-to-treat analysis set (LOCF) and healthy siblings
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
n=65 Participants
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
Max cIMT Change from Baseline at Month 12
|
0.00626 mm
Standard Deviation 0.073446
|
0.01707 mm
Standard Deviation 0.056223
|
|
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
Max cIMT Change from Baseline at Month 24
|
0.00189 mm
Standard Deviation 0.060864
|
0.01202 mm
Standard Deviation 0.049102
|
|
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
Mean cIMT Change from Baseline at Month 12
|
0.00282 mm
Standard Deviation 0.041186
|
0.01564 mm
Standard Deviation 0.032052
|
|
Change From Baseline in Max and Mean Carotid Intima and Media Wall Thickness (cIMT)
Mean cIMT Change from Baseline at Month 24
|
0.01056 mm
Standard Deviation 0.040762
|
0.02779 mm
Standard Deviation 0.031004
|
SECONDARY outcome
Timeframe: 2-year study periodPopulation: Safety analysis set
Number of participants with Various Categories of AE's. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Adverse Events
Any AE
|
172 Participant
|
—
|
|
Adverse Events
AE Leading to Death
|
0 Participant
|
—
|
|
Adverse Events
AE Leading to Discontinuation
|
3 Participant
|
—
|
|
Adverse Events
Serious AE
|
9 Participant
|
—
|
|
Adverse Events
Treatment Related AE
|
29 Participant
|
—
|
|
Adverse Events
Treatment Related AE Leading to Death
|
0 Participant
|
—
|
|
Adverse Events
Treatment Related AE Leading to Discontinuation
|
3 Participant
|
—
|
|
Adverse Events
Treatment Related Serious AE
|
0 Participant
|
—
|
SECONDARY outcome
Timeframe: 2-year study periodPopulation: Safety analysis set
Total duration of exposure was calculated as \[last dose date of rosuva - first dose date of rosuva + 1 day\]. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Total Duration of Exposure
|
703.5 Days
Standard Deviation 97.25
|
—
|
SECONDARY outcome
Timeframe: 2-year study periodPopulation: Safety analysis set
Overall adherence rate was calculated as the weighted mean of adherence rates of all consecutive visits after baseline, in which the adherence rate between 2 consecutive visits was a percentage of the number of rosuvastatin taken divided by duration of exposure. One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
Outcome measures
| Measure |
Rosuvastatin
n=197 Participants
Rosuvastatin 5 mg, 10 mg or 20 mg
|
Healthy Siblings
Controls to HeFH patients in the cIMT evaluations
|
|---|---|---|
|
Overal Treatment Adherence
|
89.6 Percent of doses
Standard Deviation 12.25
|
—
|
Adverse Events
Rosuvastatin
Serious events: 9 serious events
Other events: 172 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Rosuvastatin
n=197 participants at risk
Rosuvastatin 5 mg, 10 mg or 20 mg
|
|---|---|
|
Infections and infestations
Appendicitis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Urinary retention postoperative
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Forearm fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Streptococcal toxic shock syndrome
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Viral pericarditis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Multiple fractures
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Leg fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Epilepsy
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Congenital, familial and genetic disorders
Pectus carinatum
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
Other adverse events
| Measure |
Rosuvastatin
n=197 participants at risk
Rosuvastatin 5 mg, 10 mg or 20 mg
|
|---|---|
|
Eye disorders
Conjunctivitis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Eye disorders
Myopia
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Eye disorders
Optic nerve disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Abdominal discomfort
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Abdominal distension
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Abdominal pain
|
6.6%
13/197 • Number of events 18 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
7.6%
15/197 • Number of events 21 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Constipation
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Dental discomfort
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Blood and lymphatic system disorders
Lymphadenopathy
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Congenital, familial and genetic disorders
Pectus carinatum
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Auricular swelling
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Deafness unilateral
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Ear discomfort
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Ear pain
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Middle ear effusion
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Ear and labyrinth disorders
Motion sickness
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Endocrine disorders
Early menarche
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Endocrine disorders
Hypothyroidism
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Diarrhoea
|
3.0%
6/197 • Number of events 9 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Dyspepsia
|
1.5%
3/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Flatulence
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Gastritis
|
2.0%
4/197 • Number of events 6 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Intestinal obstruction
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Irritable bowel syndrome
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Mouth ulceration
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Nausea
|
9.1%
18/197 • Number of events 24 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Rectal spasm
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Toothache
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Gastrointestinal disorders
Vomiting
|
9.6%
19/197 • Number of events 24 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
General disorders
Asthenia
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
General disorders
Fatigue
|
3.6%
7/197 • Number of events 8 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
General disorders
Influenza like illness
|
8.1%
16/197 • Number of events 19 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
General disorders
Malaise
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
General disorders
Pyrexia
|
5.1%
10/197 • Number of events 10 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Hepatobiliary disorders
Hepatic pain
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Immune system disorders
Allergy to animal
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Immune system disorders
Hypersensitivity
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Immune system disorders
Seasonal allergy
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Amoebic dysentery
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Appendicitis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Bronchitis
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Conjunctivitis infective
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Cystitis
|
1.5%
3/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Ear infection
|
2.0%
4/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Eye infection
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Furuncle
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Gastritis viral
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Gastroenteritis
|
6.1%
12/197 • Number of events 15 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Gastroenteritis viral
|
9.1%
18/197 • Number of events 24 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Gingival infection
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Groin infection
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Herpes zoster
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Impetigo
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Infectious mononucleosis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Influenza
|
10.2%
20/197 • Number of events 28 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Laryngitis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Lower respiratory tract infection
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Mononucleosis syndrome
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Nail infection
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Nasopharyngitis
|
44.7%
88/197 • Number of events 150 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Otitis externa
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Otitis media
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pertussis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pharyngitis
|
4.1%
8/197 • Number of events 10 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pharyngitis streptococcal
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pilonidal cyst
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pneumonia
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Pyelonephritis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Respiratory tract infection
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Rhinitis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Sinusitis
|
1.0%
2/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Tonsillitis
|
2.0%
4/197 • Number of events 6 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Tooth infection
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Toxic shock syndrome streptococcal
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Upper respiratory tract infection
|
5.6%
11/197 • Number of events 12 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Urinary tract infection
|
3.6%
7/197 • Number of events 7 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Viral infection
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Viral pericarditis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Infections and infestations
Viral upper respiratory tract infection
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Arthropod bite
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Concussion
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Contusion
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Excoriation
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Face injury
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Fall
|
1.5%
3/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Femur fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Foot fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Forearm fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Hand fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Joint injury
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Laceration
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Ligament rupture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Ligament sprain
|
2.5%
5/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Limb injury
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Lower limb fracture
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Multiple fractures
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Periorbital haematoma
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Post procedural complication
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Post procedural swelling
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Post-traumatic pain
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Procedural pain
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Road traffic accident
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Scratch
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Tendon injury
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Tendon rupture
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Traumatic haematoma
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Urinary retention postoperative
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Injury, poisoning and procedural complications
Wrist fracture
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Blood bilirubin increased
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Blood creatine phosphokinase increased
|
2.0%
4/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Cardiac murmur
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Haemoglobin decreased
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Hepatic enzyme increased
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Lymphocyte count abnormal
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Renal bruit
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Serum ferritin decreased
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Investigations
Weight decreased
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Metabolism and nutrition disorders
Hypoglycaemia
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.1%
12/197 • Number of events 15 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
3.0%
6/197 • Number of events 6 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Bone disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Exostosis
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Flank pain
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Growing pains
|
1.0%
2/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Joint stiffness
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Joint swelling
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Muscle spasms
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Muscular weakness
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal stiffness
|
2.0%
4/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.6%
11/197 • Number of events 12 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Neck pain
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Osteochondrosis
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
3.6%
7/197 • Number of events 7 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Pain in jaw
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Scoliosis
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Tendon disorder
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Tendon pain
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Musculoskeletal and connective tissue disorders
Tendonitis
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Skin papilloma
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Dizziness
|
3.0%
6/197 • Number of events 8 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Epilepsy
|
0.51%
1/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Headache
|
23.4%
46/197 • Number of events 76 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Migraine
|
1.5%
3/197 • Number of events 11 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Paraesthesia
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Post-traumatic headache
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Presyncope
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Psychomotor hyperactivity
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Nervous system disorders
Syncope
|
3.0%
6/197 • Number of events 11 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Attention deficit/hyperactivity disorder
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Autism spectrum disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Depressed mood
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Depression
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Insomnia
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Intentional self-injury
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Psychiatric disorders
Sleep disorder
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Renal and urinary disorders
Haematuria
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Renal and urinary disorders
Pollakiuria
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Renal and urinary disorders
Polyuria
|
0.51%
1/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Renal and urinary disorders
Pyuria
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Amenorrhoea
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Dysmenorrhoea
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Menstrual disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Menstruation irregular
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Polycystic ovaries
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Reproductive system and breast disorders
Scrotal pain
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Asthma
|
1.0%
2/197 • Number of events 2 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
5.6%
11/197 • Number of events 11 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
3.0%
6/197 • Number of events 9 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
1.0%
2/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal obstruction
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
6.6%
13/197 • Number of events 13 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Rhinorrhoea
|
2.5%
5/197 • Number of events 5 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus congestion
|
1.5%
3/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Respiratory, thoracic and mediastinal disorders
Sinus disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Acne
|
3.0%
6/197 • Number of events 8 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Alopecia
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
2.0%
4/197 • Number of events 4 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Nail disorder
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Rash
|
1.5%
3/197 • Number of events 3 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Rash vesicular
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Skin lesion
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Skin and subcutaneous tissue disorders
Xanthoma
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
|
Vascular disorders
Haematoma
|
0.51%
1/197 • Number of events 1 • Treatment Phase
One patient received 1 dose of study drug but was not included in the efficacy and safety analyses due to a lack of follow-up data.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place