Homozygous Familial Hypercholesterolemia (HoFH)Phase 3NCT02434497
A Study to Evaluate the Safety of Rosuvastatin in Children and Adolescents With Homozygous Familial Hypercholesterolemia
A Phase 3 study testing Rosuvastatin 20mg in people with Homozygous Familial Hypercholesterolemia (HoFH), run by AstraZeneca at 6 sites in 6 countries.
Trial summary
The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia. Read more about this study
- Who is it for?
- Ages 6 to 18 years
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia (HoFH)
- What is being tested?
- Rosuvastatin 20mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 6 sitesin 6 countries
- Study dates
- 6 Jun 2015to 17 Nov 2016
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Prior to any study related procedures being performed, provision of written informed consent from a parent/both parents or guardian and statement of assent from the child or adolescent (if required by Institutional Review Board [IRB] or Independent Ethics Committee [IEC] according to local regulations and guidelines). Study D3561C00004 participants who have had their 18th birthday (adults) will be required to provide written informed consent. Communication should take place between the Investigator, patient/guardian and child/adolescent to confirm understanding and required compliance with the requirements of the study.
- Male and female children and adolescents who were aged 6 to <18 years at the onset of Study D3561C00004 (even if they had their 18th birthday during that study) with:
- Documentation of genetic testing confirming 2 mutated alleles of the LDL receptor gene locus; and/or
- Documented untreated LDL C >500 mg/dL (12.9 mmol/L) and TG <400 mg/dL (4.5 mmol/L) and at least 1 of the following criteria:
- Tendinous and/or cutaneous xanthoma prior to 10 years of age; or
- Documentation of HeFH in both parents by:
Show all 12Show fewer
- genetic and/or
- clinical criteria
- Negative pregnancy test (b human chorionic gonadotropin analysis) prior to baseline in females of child bearing potential:
- Female patients of child bearing potential must adhere to a pregnancy prevention method (abstinence, chemical, or mechanical) during the study and 3 months following the last dose;
- Male patients should refrain from fathering a child (including sperm donation) during the study and up to 3 months following the last dose; and
- Were taking study drug at the end of Study D3561C00004 and are willing to follow all study procedures including adherence to dietary guidelines, study visits, fasting blood draws, and compliance with study treatment regimens.
Who cannot join
- History of statin inducted myopathy or serious hypersensitivity reaction to other HMG CoA reductase inhibitors (statins), including rosuvastatin, at Visit 1 of Study D3561C00004.
- Fasting serum glucose of >9.99 mmol/L (180 mg/dL) or glycosylated hemoglobin >9% during Study D3561C00004 or patients with a history of diabetic ketoacidosis within the past year.
- Uncontrolled hypothyroidism defined as thyroid stimulating hormone >1.5 times the upper limit of normal (ULN) at any time during Study D3561C00004.
- Evidence of active liver disease or hepatic dysfunction (except a confirmed diagnosis of Gilbert's disease) as defined as non-transient elevations of ALT or AST elevations ≥3 times the ULN or non-transient total bilirubin ≥2 times the ULN during the Study D3561C00004.
- Definite or suspected personal history or family history of clinically significant adverse drug reactions (ADRs), or hypersensitivity to drugs with a similar chemical structure to rosuvastatin as well as other statins.
Trial locations and contacts
6 sites in 6 countries.
6 sites in 6 countries
About this study
Official title: An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH)
The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.
Read the detailed description
This is a long-term extension (LTE) to the randomized, double-blind, cross-over study of rosuvastatin 20 mg once daily (QD) versus placebo QD in children and adolescents (aged from 6 to <18 years) with homozygous familial hypercholesterolemia (HoFH) (Study D3561C00004).
The study is designed to assess the long-term safety and tolerability of rosuvastatin 20 mg in pediatric patients with HoFH.
In this study all patients will receive rosuvastatin 20 mg QD. Investigators will also be permitted to titrate the dose of rosuvastatin from 20 to 40 mg per day if they feel it is warranted to more aggressively treat patients' elevated LDL-C levels. This up-titration will not be permitted in Asian patients. Pharmacokinetic data of the trough plasma exposure of rosuvastatin will also be assessed for the pediatric patients with HoFH taking a daily dose of rosuvastatin 40 mg.
The primary outcome measures to be assessed include 1) Adverse events, including:
- The frequency and severity of adverse events,
- Rate of discontinuations due to adverse events,
- Abnormal serum and urine laboratory values, electrocardiograms (ECGs), physical examinations, and vital signs; and 2) Assessments of growth.
- Being tested
- Rosuvastatin 20mg
- Keywords
- LDL-C, HoFH, Hyperlipidemia
- Medical subject headings
- Homozygous Familial Hypercholesterolemia, Hyperlipidemias
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Rosuvastatin 20mgDrug
Active drug 1 or 2 tablets will be taken taken orally, QD, either in the morning or in the evening
| Study group | Role | What they receive |
|---|---|---|
| Single Arm | Experimental | One treatment period for all patients (<1 year and 10 months), with the possibility to up-titrate dose to 40 mg of rosuvastatin for non-Asian patients. Receives: Drug: Rosuvastatin 20mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Number of Participants Who Experianced Adverse Events and Serious Adverse Events
Measured: 96 weeks
Safety and Tolerability in Terms of Number of Participants Who Had Adverse Events, Discontinuations Due to Adverse Events
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Basophils/Leukocytes (%) >Upper Limite of Normal (ULN)
Measured: 96 weeks
Safety and Tolerability in Terms of Growth, Height
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormalitites in Sexual Maturation
Measured: 96 weeks
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.
Measured: 96 weeks
Safety and Tolerability in Terms of Growth, Weight
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Alanine Aminotransferase (U/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Albumin (g/dL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Aspartate Aminotransferase (U/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB Concentration (g/dL) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB (pg) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hematocrit (%) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hemoglobin (g/dL) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Leukocytes >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Monocytes/Leukocytes (%) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Platelets (10^9/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Blood Urea Nitrogen (mg/dL) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Chloride (mmol/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Creatine Kinase (U/L) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Glucose (mg/dL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lactate Dehydrogenase (U/L) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Phosphate (mg/dL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Protein (g/dL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Sodium (mmol/L) <LLN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Urate (mg/dL) >ULN
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Ketones
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Occult Blood
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Protein
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal ECG, Abnormalities
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Cardiovascular
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, General Appearance
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Head and Neck
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Musculoskeletal/Extremities
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Skin
Measured: 96 weeks
Safety and Tolerability in Terms of Abnormal Vital Signs
Measured: 96 weeks
Secondary outcomes (12)
Percent Change in LDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in Total Cholesterol (TC) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in Triglycerides (TG) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in Non-HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in LDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in TC/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in Non-HDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in ApoB From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Percent Change in ApoB/ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Measured: Up to 22 months
Pharmacokinetic Profile in Terms of Trough Concentrations in Pediatric HoFH Taking a Daily Dose of Rosuvastatin 40mg
Measured: Up to 22 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted5 May 2015 (estimated)
- Study start6 Jun 2015
- Primary completion17 Nov 2016
- Study completion17 Nov 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Homozygous Familial Hypercholesterolemia (HoFH)Studies of the same condition, open and under way.
- More trials run by AstraZenecaThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- AstraZeneca
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02434497
- Sponsor's protocol ID
- D356NC00001
- Status last verified
- Feb 2018
- First submitted
- 7 Apr 2015
- Last updated
- 27 Feb 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Feb 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Long-Term Extension to the Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (Aged 6 to <18 Years) With Homozygous Familial Hypercholesterolemia (HoFH)
- ClinicalTrials.gov ID
- NCT02434497
- Acronym / sponsor ID
- D356NC00001
- Keywords
- LDL-C, HoFH, Hyperlipidemia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- AstraZenecaIndustry (pharma, biotech, devices)
- Study start
- 6 Jun 2015 (actual)
- Primary completion
- 17 Nov 2016 (actual)
- Study completion
- 17 Nov 2016 (actual)
- First posted
- 5 May 2015 (estimated)
- Results
- Posted 27 Feb 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of the study is to evaluate the safety of Rosuvastatin in Children and Adolescents with Homozygous Familial Hypercholesterolemia.
Detailed description
This is a long-term extension (LTE) to the randomized, double-blind, cross-over study of rosuvastatin 20 mg once daily (QD) versus placebo QD in children and adolescents (aged from 6 to <18 years) with homozygous familial hypercholesterolemia (HoFH) (Study D3561C00004).
The study is designed to assess the long-term safety and tolerability of rosuvastatin 20 mg in pediatric patients with HoFH.
In this study all patients will receive rosuvastatin 20 mg QD. Investigators will also be permitted to titrate the dose of rosuvastatin from 20 to 40 mg per day if they feel it is warranted to more aggressively treat patients' elevated LDL-C levels. This up-titration will not be permitted in Asian patients. Pharmacokinetic data of the trough plasma exposure of rosuvastatin will also be assessed for the pediatric patients with HoFH taking a daily dose of rosuvastatin 40 mg.
The primary outcome measures to be assessed include 1) Adverse events, including:
- The frequency and severity of adverse events,
- Rate of discontinuations due to adverse events,
- Abnormal serum and urine laboratory values, electrocardiograms (ECGs), physical examinations, and vital signs; and 2) Assessments of growth.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single Arm | Experimental | Drug: Rosuvastatin 20mgOne treatment period for all patients (<1 year and 10 months), with the possibility to up-titrate dose to 40 mg of rosuvastatin for non-Asian patients. |
Who may qualify
- Ages
- 6 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (12 listed)
- Prior to any study related procedures being performed, provision of written informed consent from a parent/both parents or guardian and statement of assent from the child or adolescent (if required by Institutional Review Board [IRB] or Independent Ethics Committee [IEC] according to local regulations and guidelines). Study D3561C00004 participants who have had their 18th birthday (adults) will be required to provide written informed consent. Communication should take place between the Investigator, patient/guardian and child/adolescent to confirm understanding and required compliance with the requirements of the study.
- Male and female children and adolescents who were aged 6 to <18 years at the onset of Study D3561C00004 (even if they had their 18th birthday during that study) with:
- Negative pregnancy test (b human chorionic gonadotropin analysis) prior to baseline in females of child bearing potential:
- Were taking study drug at the end of Study D3561C00004 and are willing to follow all study procedures including adherence to dietary guidelines, study visits, fasting blood draws, and compliance with study treatment regimens.
Key exclusion criteria (5 listed)
- History of statin inducted myopathy or serious hypersensitivity reaction to other HMG CoA reductase inhibitors (statins), including rosuvastatin, at Visit 1 of Study D3561C00004.
- Fasting serum glucose of >9.99 mmol/L (180 mg/dL) or glycosylated hemoglobin >9% during Study D3561C00004 or patients with a history of diabetic ketoacidosis within the past year.
- Uncontrolled hypothyroidism defined as thyroid stimulating hormone >1.5 times the upper limit of normal (ULN) at any time during Study D3561C00004.
- Evidence of active liver disease or hepatic dysfunction (except a confirmed diagnosis of Gilbert's disease) as defined as non-transient elevations of ALT or AST elevations ≥3 times the ULN or non-transient total bilirubin ≥2 times the ULN during the Study D3561C00004.
- Definite or suspected personal history or family history of clinically significant adverse drug reactions (ADRs), or hypersensitivity to drugs with a similar chemical structure to rosuvastatin as well as other statins.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Rosuvastatin 20mgActive drug 1 or 2 tablets will be taken taken orally, QD, either in the morning or in the evening | Drug | Single Arm |
Primary outcome
The Number of Participants Who Experianced Adverse Events and Serious Adverse Events
Time frame: 96 weeks
Safety and Tolerability in Terms of Number of Participants Who Had Adverse Events, Discontinuations Due to Adverse Events
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Basophils/Leukocytes (%) >Upper Limite of Normal (ULN)
Time frame: 96 weeks
Safety and Tolerability in Terms of Growth, Height
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormalitites in Sexual Maturation
Time frame: 96 weeks
Safety and Tolerability in Terms of Growth, Height SD-score (or Z-score)
Time frame: 96 weeks
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.
Safety and Tolerability in Terms of Growth, Weight
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Alanine Aminotransferase (U/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Albumin (g/dL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Aspartate Aminotransferase (U/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Bicarbonate (Mol/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB Concentration (g/dL) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular HGB (pg) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Ery. Mean Corpuscular Volume (fL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Erythrocytes (10^12/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hematocrit (%) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Hemoglobin (g/dL) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Leukocytes >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lymphocytes/Leukocytes (%) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Monocytes/Leukocytes (%) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Platelets (10^9/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Blood Urea Nitrogen (mg/dL) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Chloride (mmol/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Creatine Kinase (U/L) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Glucose (mg/dL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Lactate Dehydrogenase (U/L) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Phosphate (mg/dL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Protein (g/dL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Sodium (mmol/L) <LLN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Serum Laboratory Values, Urate (mg/dL) >ULN
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Ketones
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Occult Blood
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Urine Laboratory Values, Urine Protein
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal ECG, Abnormalities
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Cardiovascular
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, General Appearance
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Head and Neck
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Musculoskeletal/Extremities
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Physical Exams, Skin
Time frame: 96 weeks
Safety and Tolerability in Terms of Abnormal Vital Signs
Time frame: 96 weeks
12 secondary outcomes
Percent Change in LDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in Total Cholesterol (TC) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in Triglycerides (TG) From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in Non-HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in LDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in TC/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in Non-HDL-C/HDL-C From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in ApoB From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Percent Change in ApoB/ApoA-1 From End of Placebo of D3561C00004 to the End of D356NC00001, Repeated Measures Analysis
Time frame: Up to 22 months
Pharmacokinetic Profile in Terms of Trough Concentrations in Pediatric HoFH Taking a Daily Dose of Rosuvastatin 40mg
Time frame: Up to 22 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Feb 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
6 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Rosuvastatin 20mg in people with Homozygous Familial Hypercholesterolemia (HoFH), run by AstraZeneca at 6 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 27 Feb 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Belgium, Canada, Denmark, Israel, Malaysia and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 27 Feb 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Number of Participants Who Experianced Adverse Events and Serious Adverse Events (time frame: 96 weeks). The study lists 44 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 27 Feb 2018.