| Primary | The Number of Participants Who Experianced Adverse Events and Serious Adverse Events | | | Posted | | Number | | participants | | 96 weeks | | | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
| | |
| |
| Primary | Safety and Tolerability in Terms of Number of Participants Who Had Adverse Events, Discontinuations Due to Adverse Events | | | Posted | | Number | | participants | | 96 weeks | | | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
|
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Basophils/Leukocytes (%) >Upper Limite of Normal (ULN) | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Growth, Height | | | Posted | | Mean | Standard Deviation | cm | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormalitites in Sexual Maturation | | | Posted | | Number | | participants | | 96 weeks | | | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
|
| Primary | Safety and Tolerability in Terms of Growth, Height SD-score (or Z-score) | Height z-score is a dimensionless quantity derived by subtracting the population mean from the individual raw score, and then deviding the difference by the pouulation SD of the reference population. This indicates how many SDs and observation is above or below the general population mean. | | Posted | | Mean | Standard Deviation | standard deviations | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Growth, Weight | | | Posted | | Mean | Standard Deviation | kg | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Alanine Aminotransferase (U/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Albumin (g/dL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Aspartate Aminotransferase (U/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB Concentration (g/dL) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB (pg) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hematocrit (%) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hemoglobin (g/dL) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Leukocytes >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Monocytes/Leukocytes (%) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Platelets (10^9/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Secondary | Percent Change in LDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline LDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
| Secondary | Percent Change in HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline HDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
| Secondary | Percent Change in Total Cholesterol (TC) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline TC | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Percent Change in Triglycerides (TG) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline TG | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Percent Change in Non-HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline Non-HDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Percent Change in LDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline LDL-C/HDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Percent Change in TC/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline TC/HDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
| Secondary | Percent Change in Non-HDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline Non-HDL-C/HDL-C | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Percent Change in ApoB From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline ApoB | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
| Secondary | Percent Change in ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline ApoA-1 | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
| Secondary | Percent Change in ApoB/ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis | | | Posted | | Geometric Mean | 95% Confidence Interval | % change from baseline ApoB/ApoA-1 | | Up to 22 months | participants | participants | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | |
| Secondary | Pharmacokinetic Profile in Terms of Trough Concentrations in Pediatric HoFH Taking a Daily Dose of Rosuvastatin 40mg | | | Posted | | Number | | ng/mL | | Up to 22 months | | | | ID | Title | Description |
|---|
| OG000 | Full Analysis Set | Full analysis set, comprised of patient who received at least one dose of study drug. |
| | | Title | Denominators | Categories |
|---|
|
|
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Blood Urea Nitrogen (mg/dL) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Chloride (mmol/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Creatine Kinase (U/L) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Glucose (mg/dL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lactate Dehydrogenase (U/L) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Phosphate (mg/dL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Protein (g/dL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Sodium (mmol/L) <LLN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Urate (mg/dL) >ULN | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Ketones | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Occult Blood | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Protein | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal ECG, Abnormalities | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Physical Exams, Cardiovascular | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Physical Exams, General Appearance | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Physical Exams, Head and Neck | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Physical Exams, Musculoskeletal/Extremities | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Physical Exams, Skin | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |
| Primary | Safety and Tolerability in Terms of Abnormal Vital Signs | | | Posted | | Number | | participants | | 96 weeks | participants | participants | | ID | Title | Description |
|---|
| OG000 | Sequence A | Rosuva/Placebo in the D3561C00004 cross-over phase | | OG001 | Sequence B | Placebo/Rosuva in the D3561C00004 cross-over phase |
| |