Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia (NCT NCT02226198)
NCT ID: NCT02226198
Last Updated: 2016-07-04
Results Overview
Change in low density lipoprotein cholesterol (LDL C) following 6 weeks of rosuvastatin 20 mg compared to 6 weeks of placebo treatment
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
20 participants
Primary outcome timeframe
Samples taken on Day 42 (week 6) and on day 84 (week 12)
Results posted on
2016-07-04
Participant Flow
Twenty HoFH patients were recruited to 9 centers within 8 countries (Belgium, Canada, Denmark, Israel, Malaysia, Sweden, Taiwan, and The Netherlands) that participated in the study. FSI date: 03-Nov-2014
20 enrolled, 3 withdrew consent, 3 did not fulfil eligibility criteria. Among the 14 left, ten went through 4 week lead-in phase and 4 did not.
Participant milestones
| Measure |
Overall
Relevant for the lead-in and maintenance phases
|
Rosuva First Then Placebo
Relevant for the cross-over phase
|
Placebo First Then Rosuva
Relevant for the cross-over phase
|
|---|---|---|---|
|
Lead-in Phase
STARTED
|
11
|
0
|
0
|
|
Lead-in Phase
COMPLETED
|
10
|
0
|
0
|
|
Lead-in Phase
NOT COMPLETED
|
1
|
0
|
0
|
|
Cross-Over: First Intervention (6 Weeks)
STARTED
|
0
|
7
|
7
|
|
Cross-Over: First Intervention (6 Weeks)
COMPLETED
|
0
|
6
|
7
|
|
Cross-Over: First Intervention (6 Weeks)
NOT COMPLETED
|
0
|
1
|
0
|
|
Cross-Over: Second Interv. (6 Weeks)
STARTED
|
0
|
6
|
7
|
|
Cross-Over: Second Interv. (6 Weeks)
COMPLETED
|
0
|
6
|
7
|
|
Cross-Over: Second Interv. (6 Weeks)
NOT COMPLETED
|
0
|
0
|
0
|
|
Maintenance Phase (12 Weeks)
STARTED
|
13
|
0
|
0
|
|
Maintenance Phase (12 Weeks)
COMPLETED
|
13
|
0
|
0
|
|
Maintenance Phase (12 Weeks)
NOT COMPLETED
|
0
|
0
|
0
|
Reasons for withdrawal
| Measure |
Overall
Relevant for the lead-in and maintenance phases
|
Rosuva First Then Placebo
Relevant for the cross-over phase
|
Placebo First Then Rosuva
Relevant for the cross-over phase
|
|---|---|---|---|
|
Lead-in Phase
Protocol Violation
|
1
|
0
|
0
|
|
Cross-Over: First Intervention (6 Weeks)
No possibility to draw blood
|
0
|
1
|
0
|
Baseline Characteristics
A Study to Evaluate the Efficacy and Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
Baseline characteristics by cohort
| Measure |
Cross-over
n=14 Participants
Cross-over phase
|
|---|---|
|
Age, Continuous
|
10.9 Years
STANDARD_DEVIATION 2.70 • n=5 Participants
|
|
Sex: Female, Male
Female
|
7 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
7 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Samples taken on Day 42 (week 6) and on day 84 (week 12)Population: 6-17 years HoFH
Change in low density lipoprotein cholesterol (LDL C) following 6 weeks of rosuvastatin 20 mg compared to 6 weeks of placebo treatment
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-Cholesterol (mg/dL)
|
396.0 mg/dL
Standard Deviation 195.99
|
481.4 mg/dL
Standard Deviation 184.90
|
—
|
—
|
PRIMARY outcome
Timeframe: Samples taken on Day 42 (week 6) and on day 84 (week 12)Population: 6-17 years HoFH
Change in low density lipoprotein cholesterol (LDL C) following 6 weeks of rosuvastatin 20 mg compared to 6 weeks of placebo treatment
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-Cholesterol (mmol/L)
|
10.26 mmol/L
Standard Deviation 5.076
|
12.47 mmol/L
Standard Deviation 4.790
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of total cholesterol (TC)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
TC (mg/dL)
|
447.6 mg/dL
Standard Deviation 195.46
|
539.0 mg/dL
Standard Deviation 184.91
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of total cholesterol (TC)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
TC (mmol/L)
|
11.59 mmol/L
Standard Deviation 5.063
|
13.96 mmol/L
Standard Deviation 4.790
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of non-high density lipoprotein cholesterol (non-HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Non-HDL C (mg/dL)
|
412.1 mg/dL
Standard Deviation 198.62
|
505.3 mg/dL
Standard Deviation 186.39
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of non-high density lipoprotein cholesterol (non-HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Non-HDL C (mmol/L)
|
10.67 mmol/L
Standard Deviation 5.144
|
13.09 mmol/L
Standard Deviation 4.826
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of apolipoprotein B (ApoB)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
ApoB (mg/dL)
|
234.9 mg/dL
Standard Deviation 107.02
|
267.9 mg/dL
Standard Deviation 86.33
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of apolipoprotein B (ApoB)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
ApoB (g/L)
|
2.35 g/L
Standard Deviation 1.070
|
2.68 g/L
Standard Deviation 0.863
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of high density lipoprotein cholesterol (HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
HDL-C (mg/dL)
|
35.5 mg/dL
Standard Deviation 7.29
|
33.7 mg/dL
Standard Deviation 8.47
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of high density lipoprotein cholesterol (HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
HDL-C (mmol/L)
|
0.92 mmol/L
Standard Deviation 0.189
|
0.87 mmol/L
Standard Deviation 0.218
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Population: Patients not treated with apheresis
Efficacy in terms of low density lipoprotein cholesterol (LDL C) following 6 weeks rosuvastatin 20 mg or placebo treatment in patients not treated with Apheresis
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=6 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=6 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-C, Not on Apheresis (mg/dL)
|
479.8 mg/dL
Standard Deviation 239.07
|
594.7 mg/dL
Standard Deviation 203.60
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Population: Patients not treated with apheresis
Efficacy in terms of low density lipoprotein cholesterol (LDL C) following 6 weeks rosuvastatin 20 mg or placebo treatment in patients not treated with Apheresis
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=6 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=6 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-C, Not on Apheresis (mmol/L)
|
12.43 mmol/L
Standard Deviation 6.191
|
15.40 mmol/L
Standard Deviation 5.274
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6), Day 84 (week 12), Day 126 (week 18) and Day 168 (week 24)Change in low density lipoprotein cholesterol (LDL C) from end of placebo period to 6, 12, and 18 weeks of therapy with rosuvastatin 20 mg
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=6 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-C From End of Placebo (mg/dL)
6 weeks after end of Placebo
|
399.4 mg/dL
Standard Deviation 214.28
|
421.7 mg/dL
Standard Deviation 224.49
|
—
|
—
|
|
LDL-C From End of Placebo (mg/dL)
12 weeks after end of Placebo
|
345.8 mg/dL
Standard Deviation 214.69
|
394.7 mg/dL
Standard Deviation 178.45
|
—
|
—
|
|
LDL-C From End of Placebo (mg/dL)
18 weeks after end of Placebo
|
NA mg/dL
Standard Deviation NA
No measurements available since taken only at 6 and 12 weeks after end of placebo
|
441.8 mg/dL
Standard Deviation 260.31
|
—
|
—
|
|
LDL-C From End of Placebo (mg/dL)
End of Placebo
|
472.9 mg/dL
Standard Deviation 171.77
|
491.3 mg/dL
Standard Deviation 215.46
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6), Day 84 (week 12), Day 126 (week 18) and Day 168 (week 24)Change in low density lipoprotein cholesterol (LDL C) from end of placebo period to 6, 12, and 18 weeks of therapy with rosuvastatin 20 mg
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=6 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL-C From End of Placebo (mmol/L)
End of Placebo
|
12.25 mmol/L
Standard Deviation 4.449
|
12.73 mmol/L
Standard Deviation 5.583
|
—
|
—
|
|
LDL-C From End of Placebo (mmol/L)
6 weeks after end of Placebo
|
10.34 mmol/L
Standard Deviation 5.551
|
10.92 mmol/L
Standard Deviation 5.813
|
—
|
—
|
|
LDL-C From End of Placebo (mmol/L)
18 weeks after end of Placebo
|
NA mmol/L
Standard Deviation NA
No measurements available since taken only at 6 and 12 weeks after end of placebo
|
11.44 mmol/L
Standard Deviation 6.740
|
—
|
—
|
|
LDL-C From End of Placebo (mmol/L)
12 weeks after end of Placebo
|
8.96 mmol/L
Standard Deviation 5.558
|
10.22 mmol/L
Standard Deviation 4.621
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken 24 hours post-dose at Day 42 (week 6), Day 84 (week 12), Day 126 (week 18)Pharmacokinetic profile in terms of trough concentrations. Cross-over phase results based on measurements taken after 6 weeks active treatment (rosuvastatin) in the cross-over phase. Maintenance phase results based on measurements taken after 6 weeks active treatment (rosuvastatin) in the maintenance phase.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=11 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Trough Concentrations
|
7.387 ng/mL
Standard Deviation 8.11
|
4.482 ng/mL
Standard Deviation 3.50
|
—
|
—
|
SECONDARY outcome
Timeframe: From screening (5-6weeks before dose) up to the last visit Day 168 (approximately 30 weeks after screening)Safety and tolerability will be described in terms of frequency and severity of adverse events
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=11 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=14 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
n=13 Participants
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Adverse Events
Any AE
|
4 adverse events
|
5 adverse events
|
1 adverse events
|
—
|
|
Adverse Events
Any SAE
|
0 adverse events
|
0 adverse events
|
0 adverse events
|
—
|
|
Adverse Events
Any TEAE
|
4 adverse events
|
5 adverse events
|
1 adverse events
|
—
|
|
Adverse Events
Any AE leading to death
|
0 adverse events
|
0 adverse events
|
0 adverse events
|
—
|
SECONDARY outcome
Timeframe: From screening (5-6weeks before dose) up to the last visit Day 168 (approximately 30 weeks after screening)Safety and tolerability will be described in terms of rate of discontinuations due to adverse events
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=11 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=14 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
n=13 Participants
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
AE's Leading to Discontinuation
|
0 adverse events
|
0 adverse events
|
0 adverse events
|
—
|
SECONDARY outcome
Timeframe: From screening (5-6weeks before dose) up to the last visit Day 168 (approximately 30 weeks after screening)Safety and tolerability will be described in terms of abnormal serum laboratory values. The reported parameters are not the only ones measured, but rather those for which abnormailities were found
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
n=7 Participants
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
n=7 Participants
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Abnormal Serum Levels
Hemoglobin week 12
|
1 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Alanine Aminotransferase week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Alanine Aminotransferase week 6
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Albumin week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Albumin week 18
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Bicarbonate week 6
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Bicarbonate week 12
|
1 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Bicarbonate week 24
|
1 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Blood Urea Nitrogen week 18
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Creatine Kinase week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Creatine Kinase week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Glucose week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Potassium week 6
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Potassium week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Protein week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Protein week 24
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Sodium week 6
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Sodium week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Sodium week 24
|
0 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Total Bilirubin week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Total Bilirubin week 6
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Total Bilirubin week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Urate week 12
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular HGB Concentration week 12
|
3 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular HGB Concentration week 18
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular HGB Concentration week 24
|
2 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Hemoglobin week 0
|
1 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Volume week 24
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Erythrocytes week 0
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Erythrocytes week 6
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Erythrocytes week 12
|
0 participants
|
0 participants
|
1 participants
|
1 participants
|
|
Abnormal Serum Levels
Erythrocytes week 18
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Erythrocytes week 24
|
0 participants
|
2 participants
|
0 participants
|
2 participants
|
|
Abnormal Serum Levels
Hemoglobin week 0
|
2 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Hemoglobin week 6
|
2 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Hemoglobin week 24
|
0 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Lymphocytes/Leukocytes week 0
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Lymphocytes/Leukocytes week 6
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Lymphocytes/Leukocytes week 12
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Lymphocytes/Leukocytes week 18
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Lymphocytes/Leukocytes week 24
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Neutrophils, Segmented/Leukocytes week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils, Segmented/Leukocytes week 6
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils/Leukocytes week 6
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils/Leukocytes week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Platelets week 0
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Platelets week 6
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Platelets week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Platelets week 24
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Urate week 0
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Urate week 6
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Urate week 18
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Urate week 24
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular HGB Concentration week 0
|
2 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular HGB Concentration week 6
|
0 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Hemoglobin week 6
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Hemoglobin week 12
|
2 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Hemoglobin week 18
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Hemoglobin week 24
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Volume week 0
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Volume week 6
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Volume week 12
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Ery. Mean Corpuscular Volume week 18
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Alanine Aminotransferase week 12
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Alanine Aminotransferase week 18
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Alanine Aminotransferase week 24
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Albumin week 0
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Albumin week 6
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Albumin week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Bicarbonate week 0
|
1 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Hematocrit week 0
|
2 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Hematocrit week 6
|
2 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Hematocrit week 12
|
1 participants
|
0 participants
|
2 participants
|
0 participants
|
|
Abnormal Serum Levels
Hematocrit week 18
|
1 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Hematocrit week 24
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Hemoglobin week 18
|
1 participants
|
0 participants
|
3 participants
|
0 participants
|
|
Abnormal Serum Levels
Bicarbonate week 18
|
0 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Blood Urea Nitrogen week 0
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Blood Urea Nitrogen week 6
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Blood Urea Nitrogen week 12
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils, Segmented/Leukocytes week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils, Segmented/Leukocytes week 18
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils, Segmented/Leukocytes week 24
|
1 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils/Leukocytes week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils/Leukocytes week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Neutrophils/Leukocytes week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Platelets week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Blood Urea Nitrogen week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Chloride week 0
|
0 participants
|
2 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Chloride week 6
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Chloride week 12
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Chloride week 18
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Chloride week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Creatine Kinase week 6
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Creatine Kinase week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Creatine Kinase week 24
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Glucose week 0
|
0 participants
|
1 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Glucose week 6
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Glucose week 18
|
0 participants
|
1 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Glucose week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Potassium week 0
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Potassium week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Potassium week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Protein week 0
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Protein week 6
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Protein week 12
|
0 participants
|
0 participants
|
0 participants
|
1 participants
|
|
Abnormal Serum Levels
Sodium week 0
|
2 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Sodium week 18
|
3 participants
|
0 participants
|
1 participants
|
0 participants
|
|
Abnormal Serum Levels
Total Bilirubin week 18
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
|
Abnormal Serum Levels
Total Bilirubin week 24
|
0 participants
|
0 participants
|
0 participants
|
0 participants
|
SECONDARY outcome
Timeframe: Week 0 (start of cross-over), weeks 6, week 12 and week 18Safety and tolerability will be described in terms of growth, including height (linear growth \[cm and standard deviation (SD) score\]), and weight.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Height
Height (cm) week 0
|
144.0 cm
Standard Deviation 17.82
|
140.9 cm
Standard Deviation 15.70
|
—
|
—
|
|
Height
Height (cm) week 6
|
144.9 cm
Standard Deviation 17.70
|
141.1 cm
Standard Deviation 16.11
|
—
|
—
|
|
Height
Height (cm) week 12
|
145.7 cm
Standard Deviation 18.07
|
142.0 cm
Standard Deviation 17.17
|
—
|
—
|
|
Height
Height (cm) week 18
|
146.9 cm
Standard Deviation 17.68
|
142.3 cm
Standard Deviation 16.99
|
—
|
—
|
|
Height
Height (cm) week 24
|
147.1 cm
Standard Deviation 17.94
|
143.5 cm
Standard Deviation 17.58
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 (start of cross-over), weeks 6, week 12 and week 18Safety and tolerability will be described in terms of growth, including height (linear growth \[cm and standard deviation (SD) score\]), and weight.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Height Z-score
Height z-score week 18
|
0.00 ratio
Standard Deviation 1.721
|
-0.65 ratio
Standard Deviation 1.757
|
—
|
—
|
|
Height Z-score
Height z-score week 24
|
0.04 ratio
Standard Deviation 1.752
|
-0.49 ratio
Standard Deviation 1.758
|
—
|
—
|
|
Height Z-score
Height z-score week 0
|
-0.42 ratio
Standard Deviation 1.697
|
-0.75 ratio
Standard Deviation 1.555
|
—
|
—
|
|
Height Z-score
Height z-score week 6
|
-0.29 ratio
Standard Deviation 1.703
|
-0.71 ratio
Standard Deviation 1.644
|
—
|
—
|
|
Height Z-score
Height z-score week 12
|
-0.18 ratio
Standard Deviation 1.742
|
-0.70 ratio
Standard Deviation 1.776
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 (start of cross-over), weeks 6, week 12 and week 18Safety and tolerability will be described in terms of growth, including height (linear growth \[cm and standard deviation (SD) score\]), and weight.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Weight
Weight (kg) week 0
|
37.39 kg
Standard Deviation 12.731
|
40.19 kg
Standard Deviation 16.520
|
—
|
—
|
|
Weight
Weight (kg) week 6
|
38.14 kg
Standard Deviation 13.577
|
40.36 kg
Standard Deviation 16.462
|
—
|
—
|
|
Weight
Weight (kg) week 12
|
38.63 kg
Standard Deviation 14.426
|
42.30 kg
Standard Deviation 18.651
|
—
|
—
|
|
Weight
Weight (kg) week 18
|
39.31 kg
Standard Deviation 14.588
|
42.50 kg
Standard Deviation 17.990
|
—
|
—
|
|
Weight
Weight (kg) week 24
|
39.90 kg
Standard Deviation 14.550
|
43.00 kg
Standard Deviation 17.867
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0 (start of cross-over)Stages for fem (Pubic hair, Breasts): 1. (Preadol,Preadol) 2. (Sparse, lightly pigmented, medial border of labia,Breast and papilla elevated as small mound; areolar diam incr) 3. (Darker, beginning to curl, incr amount, Breast and areola enlarged, no contour separation) 4. (Course, curly, abundant but less amount in adult,Areola and papilla form secondary mound) 5. (Adult fem triangle, spread to medial surface of thighs,Mature, nipple projects, areola part of general breast contour) For males (Pubic hair, Penis, Testes) 1=(None,Preadol,Preadol) 2=(Scanty, long, light pigm,Slight enl,Enl scrotum, pink texture alt) 3=(Darker, starts to curl, small amount,Longer,Larger) 4=(Resembles adult type, but less in quant; course, curly,Larger; glans and breadth increased in size,Larger, scrotum dark) 5=(Adult distr, spread to medial thighs,Adult size,Adult size). Progr at a normal rate is preferred. Regr is not preferred.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Tanner Stage
Tanner stage week 0
|
2.0 stage
Standard Deviation 1.41
|
1.7 stage
Standard Deviation 1.11
|
—
|
—
|
|
Tanner Stage
Tanner stage week 24
|
2.3 stage
Standard Deviation 1.38
|
2.0 stage
Standard Deviation 0.2
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of triglycerides (TG)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
TG (mg/dL)
|
79.8 mg/dL
Standard Deviation 24.48
|
119.5 mg/dL
Standard Deviation 52.67
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of triglycerides (TG)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
TG (mmol/L)
|
0.90 mmol/L
Standard Deviation 0.277
|
1.35 mmol/L
Standard Deviation 0.595
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of low density lipoprotein cholesterol (LDL C) / high density lipoprotein cholesterol (HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
LDL C/HDL C
|
12.208 ratio
Standard Deviation 7.8638
|
15.600 ratio
Standard Deviation 9.1317
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of total cholesterol (TC) / high density lipoprotein cholesterol (HDL C)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
TC/HDL C
|
13.704 ratio
Standard Deviation 8.0414
|
17.416 ratio
Standard Deviation 9.5454
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of non-high density lipoprotein cholesterol (non-HDL C) / HDL C
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Non-HDL C/HDL C
|
12.704 ratio
Standard Deviation 8.0414
|
16.416 ratio
Standard Deviation 9.5454
|
—
|
—
|
SECONDARY outcome
Timeframe: Samples taken at Day 42 (week 6) and Day 84 (week 12)Efficacy in terms of apolipoprotein B (ApoB) / apolipoprotein A (ApoA)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=13 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=13 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
ApoB/ApoA
|
2.408 ratio
Standard Deviation 1.3259
|
2.873 ratio
Standard Deviation 1.4669
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0, week 6, week 12 and week 18Safety and tolerability will be described in terms of abnormal urine laboratory values
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Urinalysis Abnormalitites
Urine Protein week 0
|
3 participants
|
2 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Protein week 6
|
1 participants
|
2 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Protein week 12
|
3 participants
|
2 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Protein week 18
|
2 participants
|
1 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Protein week 24
|
2 participants
|
1 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Ketones week 0
|
1 participants
|
0 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Ketones week 6
|
0 participants
|
0 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Ketones week 12
|
0 participants
|
0 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Ketones week 18
|
0 participants
|
1 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Ketones week 24
|
0 participants
|
0 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Blood week 0
|
1 participants
|
2 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Blood week 6
|
2 participants
|
1 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Blood week 12
|
1 participants
|
2 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Blood week 18
|
1 participants
|
1 participants
|
—
|
—
|
|
Urinalysis Abnormalitites
Urine Blood week 24
|
1 participants
|
1 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: Week 0Safety and tolerability will be described in terms of abnormal electro cardio gram (ECG)
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=14 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
n=7 Participants
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
ECG Abnormalities
|
0 participants
|
0 participants
|
0 participants
|
—
|
SECONDARY outcome
Timeframe: Screening, Week 0, week 6, week 12 and week 18, week 24Safety and tolerability will be described in terms of abnormal physical examinations. Only parameters for which abnormalities were found are reported.
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=7 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
n=7 Participants
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Physical Exam Abnormalitites
General Appearance week 0
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
General Appearance week 6
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin week 12
|
5 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin week 18
|
5 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin week 24
|
5 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
General Appearance screening
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
General Appearance week 12
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
General Appearance week 18
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
General Appearance week 24
|
1 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck screening
|
0 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck week 0
|
0 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck week 6
|
0 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck week 12
|
0 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck week 18
|
0 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Head and Neck week 24
|
0 participants
|
1 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes screening
|
0 participants
|
0 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes week 0
|
0 participants
|
1 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes week 6
|
0 participants
|
0 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes week 12
|
0 participants
|
0 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes week 18
|
0 participants
|
0 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Lymph Nodes week 24
|
0 participants
|
0 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin screening
|
5 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin week 0
|
5 participants
|
2 participants
|
—
|
—
|
|
Physical Exam Abnormalitites
Skin week 6
|
5 participants
|
2 participants
|
—
|
—
|
SECONDARY outcome
Timeframe: From screening (5-6weeks before dose) up to the last visit Day 168 (approximately 30 weeks after screening)Safety and tolerability will be described in terms of abnormal vital signs
Outcome measures
| Measure |
C-FAS Rosuvastatin
n=14 Participants
Cross-over Full Analysys Set, 6 weeks of Rosuvastatin
|
C-FAS Placebo
Cross-over Full Analysis Set, 6 weeks of Placebo
|
Maintenance
Maintenance Phase, Safety analysis set
|
Pla/Ros >ULN
Safety Analysis Set, starting cross-over phase with 6 weeks of placebo
|
|---|---|---|---|---|
|
Abnormal Vital Signs
|
0 participants
|
—
|
—
|
—
|
Adverse Events
Lead-in
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Cross-over
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Maintenance
Serious events: 0 serious events
Other events: 1 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Lead-in
n=11 participants at risk
Lead-in
|
Cross-over
n=14 participants at risk
Cross-over phase
|
Maintenance
n=13 participants at risk
Maintenance phase
|
|---|---|---|---|
|
General disorders
Influenza like illness
|
18.2%
2/11 • Number of events 2
|
14.3%
2/14 • Number of events 2
|
0.00%
0/13
|
|
General disorders
Peripheral swelling
|
9.1%
1/11 • Number of events 1
|
0.00%
0/14
|
0.00%
0/13
|
|
Gastrointestinal disorders
Abdominal pain upper
|
9.1%
1/11 • Number of events 1
|
0.00%
0/14
|
0.00%
0/13
|
|
Infections and infestations
Influenza
|
0.00%
0/11
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Infections and infestations
Nasopharyngitis
|
0.00%
0/11
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
|
Investigations
Blood bicarbonate decreased
|
0.00%
0/11
|
0.00%
0/14
|
7.7%
1/13 • Number of events 1
|
|
General disorders
Chest pain
|
0.00%
0/11
|
7.1%
1/14 • Number of events 1
|
0.00%
0/13
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee Upon completion of the Clinical Trial (CT), the Principal Investigator (PI) may prepare the data derived from the CT for publication or presentation. Material should be submitted to the Sponsor for review prior to review or submission for publication, or public dissemination. Publications from individual sites must not precede the primary manuscript, but if not published within twelve months after completion of the CT, the PI has the right to publish or present the methods and results of the CT.
- Publication restrictions are in place
Restriction type: OTHER