Hyperlipoproteinemia Type IIPhase 3REALIZENCT01524289

Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)

A Phase 3 study testing Anacetrapib in people with Hyperlipoproteinemia Type II and Hypercholesterolemia, Familial, run by Merck Sharp & Dohme LLC.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

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Trial summary

The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH). Read more about this study

Who is it for?: Ages 18 to 80 years
Conditions the study is for: Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
What is being tested?: Anacetrapib
Type of treatment: Pill or oral medicine
Could I receive a placebo?: Yes, some participants may receive a placebo
How many people: 306 participants
Where: No sites listed yet
Study dates: 3 Feb 2012to 13 Nov 2018
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 18 to 80 years
All sexes
No healthy volunteers
Adult, Older adult

Who may be able to join

If of reproductive potential, must agree to remain abstinent or use (or have their partner use) 2 acceptable methods of birth control for the duration of the study
Diagnosed with Heterozygous Familial Hypercholesterolemia (HeFH)
Have been treated with an optimal dose of statin for at least 6 weeks

Who cannot join

Received treatment with low-density lipoprotein (LDL) apheresis within 4 weeks of screening or expect to undergo treatment with LDL apheresis during the course of the study
Homozygous familial hypercholesterolemia
Severe chronic heart failure
Uncontrolled hypertension
Uncontrolled cardiac arrhythmias, myocardial infarction (MI), percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG), unstable angina, or stroke within 3 months
Uncontrolled endocrine or metabolic disease known to influence serum lipids or lipoproteins

Show all 16Show fewer

Active or chronic hepatobiliary, hepatic, or gall bladder disease
Pregnant or breast-feeding, or plans to become pregnant during the study or within 2 years after stopping study medication
History of ileal bypass, gastric bypass, or other significant condition associated with malabsorption
Human immunodeficiency virus (HIV) positive
History of malignancy ≤5 years
Donated blood products or has had phlebotomy of >300 mL within 8 weeks or intends to donate 250 mL of blood products or receive blood products within the projected duration of the study
Currently taking medications that are potent inhibitors or inducers of cytochrome P450 3A4 (CYP3A) (including but not limited to cyclosporine, systemic itraconazole or ketoconazole, erythromycin, clarithromycin, or telithromycin, nefazodone, protease inhibitors, carbamazepine, phenobarbital, phenytoin, rifabutin, rifampin, St John's wort) or has discontinued treatment <3 weeks prior
Consumes more than 2 alcoholic drinks per day
Currently participating or has participated in a study with an investigational compound or device within 3 months
Receiving treatment with systemic corticosteroids or taking systemic anabolic agents

About this study

Official title: A 1-Year, Worldwide, Multicenter, Double-Blind, Randomized, Parallel, Placebo-Controlled Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Statin Therapy With or Without Other Lipid Modifying Medication(s) in Patients With Heterozygous Familial Hypercholesterolemia

The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).

Conditions: Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Being tested: Anacetrapib, Placebo
Medical subject headings: Hyperlipoproteinemia Type II

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

AnacetrapibDrug
Also called MK-0859
One oral tablet, orally once daily for 52 weeks
PlaceboDrug
One oral tablet once daily for 52 weeks

Study groups in NCT01524289
Study group	Role	What they receive
Anacetrapib	Experimental	Participants were administered one tablet of 100 mg anacetrapib orally once daily with a meal for 52 weeks during the treatment period. Receives: Drug: Anacetrapib
Placebo	Placebo comparator	Participants were administered one matching placebo tablet orally once daily with a meal for 52 weeks during the treatment period. Receives: Drug: Placebo

What is being measured

The primary outcome is the main question the study is designed to answer.

Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase
LDL-C levels were measured at baseline and week 52 (or at discontinuation) using a beta quantification method. The Treatment Phase was the period from the date of the participant's first dose of study treatment (randomization visit, Visit 3) to the participant's last visit on treatment (discontinuation visit or Visit 8 [Week 52]).
Measured: Baseline and Week 52
Percentage of Participants With Any Adverse Event - Treatment Phase
An adverse event (AE) or experience was any unfavorable and unintended change in the structure (signs), function (symptoms), or chemistry (laboratory data) of the body temporally associated with any use of a study treatment, whether or not considered related to the use of the study treatment. Any worsening of a preexisting condition which was temporally associated with the use of the study treatment is also an AE. The percentage of participants with any adverse event during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Any Treatment-Related Adverse Event - Treatment Phase
An AE was defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study treatment, whether or not considered related to the use of the treatment. Any worsening of a preexisting condition which was temporally associated with the use of the study treatment was also an AE. AEs reported by the investigator as definitely, probably or possibly related to study treatment were considered treatment-related. The percentage of participants with any treatment-related adverse event during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Any Serious Adverse Event - Treatment Phase
A serious adverse experience (SAE) was any adverse event that occurred at any dose that resulted in death or was life threatening, resulted in a persistent or significant disability/incapacity, resulted in or prolonged an existing inpatient hospitalization, or was a congenital anomaly/birth defect. The percentage of participants with any serious adverse event during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants Discontinuing Study Treatment Due to an Adverse Event - Treatment Phase
An adverse event (AE) was defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study treatment, whether or not considered related to the use of the drug. Any worsening of a preexisting condition which was temporally associated with the use of the study drug was also an AE. The percentage of participants who discontinued study treatment due to an AE during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Changes in Systolic Blood Pressure (SBP) >= 10 mm Hg
Participants had SBP assessed at baseline and throughout the 52-week treatment period. Percentage of participants who had a SBP reading that was >= 10 mm Hg higher than their baseline SBP for any assessment performed during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Changes in SBP >= 15 mm Hg
Participants had SBP assessed at baseline and throughout the 52-week treatment period. The percentage of participants who had a SBP reading that was >= 15 mm Hg higher than their baseline SBP for any assessment performed during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Changes in Diastolic Blood Pressure (DBP) >= 10 mm Hg
Participants had DBP assessed at baseline and throughout the 52-week treatment period. The percentage of participants who had a DBP reading that was >= 10 mm Hg higher than their baseline DBP for any assessment performed during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Sodium Levels > Upper Limit of Normal (ULN)
Participants had sodium levels assessed throughout the 52-week treatment period. The percentage of participants who had any sodium level that was greater than the ULN of 145 mEq/L during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Chloride Levels > ULN
Participants had chloride levels assessed throughout the 52-week treatment period. The percentage of participants who had any chloride level that was > the ULN of 110 mEq/L during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Potassium Levels < Lower Limit of Normal (LLN)
Participants had potassium levels assessed throughout the 52-week treatment period. The percentage of participants who had any potassium level that was < the LLN of 3.5 mEq/L during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Bicarbonate Levels > ULN
Participants had bicarbonate levels assessed throughout the 52-week treatment period. The percentage of participants who had any bicarbonate level that was > the ULN of 33 mEq/L during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Consecutive Changes in Alanine Aminotransferase (ALT) and/or Aspartate Aminotransferase (AST) of >=3 x ULN
Participants had AST and ALT levels assessed throughout the 52-week treatment period. The percentage of participants who had 2 consecutive assessments of either AST or ALT that were 3 x ULN or greater during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With Creatine Kinase (CK) Level >=10 x ULN
Participants had CK levels assessed throughout the 52-week treatment period. The percentage of participants who had any CK level that was >=10 x ULN during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants With CK Level >=10 x ULN With Muscle Spasms
Participants had CK levels assessed throughout the 52-week treatment period. The percentage of participants who had any CK level that was >=10 x ULN and had associated muscle spasms during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants Adjudicated Cardiovascular (CV) SAE
An AE or suspected adverse reaction was considered an SAE if it resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. All events were adjudicated by an expert committee independent of the Sponsor. The percentage of participants that experienced adjudicated SAEs of CV death, non-fatal stroke, non-fatal myocardial infarction, or unstable angina during the treatment phase is presented.
Measured: Up to 52 weeks
Percentage of Participants Who Died From Any Cause - Treatment Phase
The percentage of participants who died from any cause during the treatment phase is presented. All deaths were adjudicated by an expert committee independent of the Sponsor.
Measured: Up to 52 weeks

Secondary outcomes (5)

Percent Change From Baseline in High-Density Lipoprotein Cholesterol Levels
The efficacy of adding anacetrapib 100 mg relative to placebo on plasma concentrations of high-density lipoprotein cholesterol (HDL-C) was evaluated at Week 0 (start of treatment phase) and Week 52 (end of treatment phase) or at discontinuation.
Measured: Baseline and Week 52
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol Levels
The efficacy of adding anacetrapib 100 mg was evaluated relative to placebo on plasma concentrations of non-high-density lipoprotein cholesterol (HDL-C) for the FAS population at Week 0 (start of treatment phase) and Week 52 (end of treatment phase) or at discontinuation.
Measured: Baseline and Week 52
Percent Change From Baseline in Apolipoprotein (Apo) B Levels
The efficacy of adding anacetrapib 100 mg was evaluated relative to placebo on plasma concentrations of apolipoprotein (Apo) B for the FAS population at Week 0 (start of treatment phase) and Week 52 (end of treatment phase) or at discontinuation.
Measured: Baseline and Week 52
Percent Change From Baseline in Apolipoprotein (Apo) A-1 Levels
The efficacy of adding anacetrapib 100 mg was evaluated relative to placebo on plasma concentrations of Apo A-1 for the FAS population at Week 0 (start of treatment phase) and Week 52 (end of treatment phase) or at discontinuation.
Measured: Baseline and Week 52
Percent Change From Baseline in Lipoprotein(a) (Lp[a]) Levels
The efficacy of adding anacetrapib 100 mg was evaluated relative to placebo on plasma concentrations of lipoprotein(a) (Lp[a]) for the FAS population at Week 0 (start of treatment phase) and Week 52 (end of treatment phase) or at discontinuation.
Measured: Baseline and Week 52

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

3 Feb 201213 Nov 2018

First posted1 Feb 2012 (estimated)
Study start3 Feb 2012
Primary completion12 Feb 2014
Study completion13 Nov 2018

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.
Publications (1)Papers the sponsor has linked to this study.
Other trials for Hyperlipoproteinemia Type IIStudies of the same condition, open and under way.
More trials run by Merck Sharp & Dohme LLCThe 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: Randomised
Intervention model: Parallel groups
Masking: Double blind
Who is masked: Participant, Investigator
Enrolment: 306 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT01524289
Sponsor's protocol ID: 0859-020
Other IDs: 2011-004525-27, MK-0859-020
Acronym: REALIZE
Status last verified: Sep 2019
First submitted: 30 Jan 2012
Last updated: 14 Oct 2019
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: No
Individual data sharing: Yes: http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf

Publications

Kastelein JJ, Besseling J, Shah S, Bergeron J, Langslet G, Hovingh GK, Al-Saady N, Koeijvoets M, Hunter J, Johnson-Levonas AO, Fable J, Sapre A, Mitchel Y. Anacetrapib as lipid-modifying therapy in patients with heterozygous familial hypercholesterolaemia (REALIZE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet. 2015 May 30;385(9983):2153-61. doi: 10.1016/S0140-6736(14)62115-2. Epub 2015 Mar 3.
PubMed 25743173

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

Official record

Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Enrolling by invitationEarly Phase 1
Truway Diagnostic Tools in Primary Care
Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
Hypercholesterolaemia, Familial Hypercholesterolaemia
Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)

All Hyperlipoproteinemia Type II trials

Study at a glance

From the registry record, last updated by the sponsor on 14 Oct 2019. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A 1-Year, Worldwide, Multicenter, Double-Blind, Randomized, Parallel, Placebo-Controlled Study to Assess the Efficacy and Tolerability of Anacetrapib When Added to Ongoing Statin Therapy With or Without Other Lipid Modifying Medication(s) in Patients With Heterozygous Familial Hypercholesterolemia
ClinicalTrials.gov ID: NCT01524289
Acronym / sponsor ID: REALIZE / 0859-020
Conditions: Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Phase: Phase 3
Study type: Interventional
Recruitment status: Completed
Enrolment: 306 participants (actual)
Lead sponsor: Merck Sharp & Dohme LLCIndustry (pharma, biotech, devices)
Study start: 3 Feb 2012 (actual)
Primary completion: 12 Feb 2014 (actual)
Study completion: 13 Nov 2018 (actual)
First posted: 1 Feb 2012 (estimated)
Results: Posted 28 Aug 2019

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: Double blind (participant, investigator)
Number of arms: 2

Arm	Type	Receives
Anacetrapib	Experimental	Drug: AnacetrapibParticipants were administered one tablet of 100 mg anacetrapib orally once daily with a meal for 52 weeks during the treatment period.
Placebo	Placebo comparator	Drug: PlaceboParticipants were administered one matching placebo tablet orally once daily with a meal for 52 weeks during the treatment period.

Who may qualify

Ages: 18 to 80 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

Key inclusion criteria (3 listed)

If of reproductive potential, must agree to remain abstinent or use (or have their partner use) 2 acceptable methods of birth control for the duration of the study
Diagnosed with Heterozygous Familial Hypercholesterolemia (HeFH)
Have been treated with an optimal dose of statin for at least 6 weeks

Key exclusion criteria (16 listed)

Received treatment with low-density lipoprotein (LDL) apheresis within 4 weeks of screening or expect to undergo treatment with LDL apheresis during the course of the study
Homozygous familial hypercholesterolemia
Severe chronic heart failure
Uncontrolled hypertension
Uncontrolled cardiac arrhythmias, myocardial infarction (MI), percutaneous coronary intervention (PCI), coronary artery bypass graft (CABG), unstable angina, or stroke within 3 months

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
AnacetrapibAlso: MK-0859One oral tablet, orally once daily for 52 weeks	Drug	Anacetrapib
PlaceboOne oral tablet once daily for 52 weeks	Drug	Placebo

Primary outcome

Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase
Time frame: Baseline and Week 52
LDL-C levels were measured at baseline and week 52 (or at discontinuation) using a beta quantification method. The Treatment Phase was the period from the date of the participant's first dose of study treatment (randomization visit, Visit 3) to the participant's last visit on treatment (discontinuation visit or Visit 8 [Week 52]).
Percentage of Participants With Any Adverse Event - Treatment Phase
Time frame: Up to 52 weeks
An adverse event (AE) or experience was any unfavorable and unintended change in the structure (signs), function (symptoms), or chemistry (laboratory data) of the body temporally associated with any use of a study treatment, whether or not considered related to the use of the study treatment. Any worsening of a preexisting condition which was temporally associated with the use of the study treatment is also an AE. The percentage of participants with any adverse event during the treatment phase is presented.
Percentage of Participants With Any Treatment-Related Adverse Event - Treatment Phase
Time frame: Up to 52 weeks
An AE was defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study treatment, whether or not considered related to the use of the treatment. Any worsening of a preexisting condition which was temporally associated with the use of the study treatment was also an AE. AEs reported by the investigator as definitely, probably or possibly related to study treatment were considered treatment-related. The percentage of participants with any treatment-related adverse event during the treatment phase is presented.
Percentage of Participants With Any Serious Adverse Event - Treatment Phase
Time frame: Up to 52 weeks
A serious adverse experience (SAE) was any adverse event that occurred at any dose that resulted in death or was life threatening, resulted in a persistent or significant disability/incapacity, resulted in or prolonged an existing inpatient hospitalization, or was a congenital anomaly/birth defect. The percentage of participants with any serious adverse event during the treatment phase is presented.
Percentage of Participants Discontinuing Study Treatment Due to an Adverse Event - Treatment Phase
Time frame: Up to 52 weeks
An adverse event (AE) was defined as any unfavorable and unintended change in the structure, function, or chemistry of the body temporally associated with the use of the study treatment, whether or not considered related to the use of the drug. Any worsening of a preexisting condition which was temporally associated with the use of the study drug was also an AE. The percentage of participants who discontinued study treatment due to an AE during the treatment phase is presented.
Percentage of Participants With Changes in Systolic Blood Pressure (SBP) >= 10 mm Hg
Time frame: Up to 52 weeks
Participants had SBP assessed at baseline and throughout the 52-week treatment period. Percentage of participants who had a SBP reading that was >= 10 mm Hg higher than their baseline SBP for any assessment performed during the treatment phase is presented.
Percentage of Participants With Changes in SBP >= 15 mm Hg
Time frame: Up to 52 weeks
Participants had SBP assessed at baseline and throughout the 52-week treatment period. The percentage of participants who had a SBP reading that was >= 15 mm Hg higher than their baseline SBP for any assessment performed during the treatment phase is presented.
Percentage of Participants With Changes in Diastolic Blood Pressure (DBP) >= 10 mm Hg
Time frame: Up to 52 weeks
Participants had DBP assessed at baseline and throughout the 52-week treatment period. The percentage of participants who had a DBP reading that was >= 10 mm Hg higher than their baseline DBP for any assessment performed during the treatment phase is presented.
Percentage of Participants With Sodium Levels > Upper Limit of Normal (ULN)
Time frame: Up to 52 weeks
Participants had sodium levels assessed throughout the 52-week treatment period. The percentage of participants who had any sodium level that was greater than the ULN of 145 mEq/L during the treatment phase is presented.
Percentage of Participants With Chloride Levels > ULN
Time frame: Up to 52 weeks
Participants had chloride levels assessed throughout the 52-week treatment period. The percentage of participants who had any chloride level that was > the ULN of 110 mEq/L during the treatment phase is presented.
Percentage of Participants With Potassium Levels < Lower Limit of Normal (LLN)
Time frame: Up to 52 weeks
Participants had potassium levels assessed throughout the 52-week treatment period. The percentage of participants who had any potassium level that was < the LLN of 3.5 mEq/L during the treatment phase is presented.
Percentage of Participants With Bicarbonate Levels > ULN
Time frame: Up to 52 weeks
Participants had bicarbonate levels assessed throughout the 52-week treatment period. The percentage of participants who had any bicarbonate level that was > the ULN of 33 mEq/L during the treatment phase is presented.
Percentage of Participants With Consecutive Changes in Alanine Aminotransferase (ALT) and/or Aspartate Aminotransferase (AST) of >=3 x ULN
Time frame: Up to 52 weeks
Participants had AST and ALT levels assessed throughout the 52-week treatment period. The percentage of participants who had 2 consecutive assessments of either AST or ALT that were 3 x ULN or greater during the treatment phase is presented.
Percentage of Participants With Creatine Kinase (CK) Level >=10 x ULN
Time frame: Up to 52 weeks
Participants had CK levels assessed throughout the 52-week treatment period. The percentage of participants who had any CK level that was >=10 x ULN during the treatment phase is presented.
Percentage of Participants With CK Level >=10 x ULN With Muscle Spasms
Time frame: Up to 52 weeks
Participants had CK levels assessed throughout the 52-week treatment period. The percentage of participants who had any CK level that was >=10 x ULN and had associated muscle spasms during the treatment phase is presented.
Percentage of Participants Adjudicated Cardiovascular (CV) SAE
Time frame: Up to 52 weeks
An AE or suspected adverse reaction was considered an SAE if it resulted in any of the following outcomes: death, a life-threatening adverse event, inpatient hospitalization or prolongation of existing hospitalization, a persistent or significant incapacity or substantial disruption of the ability to conduct normal life functions, or a congenital anomaly/birth defect. All events were adjudicated by an expert committee independent of the Sponsor. The percentage of participants that experienced adjudicated SAEs of CV death, non-fatal stroke, non-fatal myocardial infarction, or unstable angina during the treatment phase is presented.
Percentage of Participants Who Died From Any Cause - Treatment Phase
Time frame: Up to 52 weeks
The percentage of participants who died from any cause during the treatment phase is presented. All deaths were adjudicated by an expert committee independent of the Sponsor.

5 secondary outcomes

Percent Change From Baseline in High-Density Lipoprotein Cholesterol Levels
Time frame: Baseline and Week 52
Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol Levels
Time frame: Baseline and Week 52
Percent Change From Baseline in Apolipoprotein (Apo) B Levels
Time frame: Baseline and Week 52
Percent Change From Baseline in Apolipoprotein (Apo) A-1 Levels
Time frame: Baseline and Week 52
Percent Change From Baseline in Lipoprotein(a) (Lp[a]) Levels
Time frame: Baseline and Week 52

Results and safety

Results posted to the registry on 28 Aug 2019, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Anacetrapib 100 mg	204	174
Placebo	102	88

Primary outcome: Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase

Time frame: Baseline and Week 52. Least squares mean (95% Confidence Interval). Unit: Percent Change

Measure	Anacetrapib 100 mg n = 165	Placebo n = 85
Value	-36.0 (-39.5 to -32.5)	3.7 (-1.2 to 8.6)

Anacetrapib 100 mg vs Placebo: Difference in Least Squares (LS) Means: -39.7 (95% CI -45.7 to -33.7), p <0.001, Constrained Longitudinal Data Analysis

Population analysed: The full analysis set (FAS) population consisted of all randomized participants who received at least one dose of study treatment, had at least one post randomization observation for the analysis endpoint subsequent to at least one dose of study treatment and had baseline data for those analyses that require baseline data.

Primary outcome: Percentage of Participants With Any Adverse Event - Treatment Phase

Time frame: Up to 52 weeks. Number. Unit: Percentage of Participants

Measure	Anacetrapib 100 mg n = 203	Placebo n = 102
Value	76.4	78.4

Anacetrapib 100 mg vs Placebo: Difference in Percentages: -2.1 (95% CI -11.5 to 8.4)

Population analysed: The all participants as treated (APaT) population consisted of all randomized participants who received at least one dose of study treatment.

Primary outcome: Percentage of Participants With Any Treatment-Related Adverse Event - Treatment Phase

Time frame: Up to 52 weeks. Number. Unit: Percentage of Participants

Measure	Anacetrapib 100 mg n = 203	Placebo n = 102
Value	18.2	13.7

Anacetrapib 100 mg vs Placebo: Difference in Percentages: 4.5 (95% CI -4.8 to 12.6)

Population analysed: The APaT population consisted of all randomized participants who received at least one dose of study treatment.

Primary outcome: Percentage of Participants With Any Serious Adverse Event - Treatment Phase

Time frame: Up to 52 weeks. Number. Unit: Percentage of Participants

Measure	Anacetrapib 100 mg n = 203	Placebo n = 102
Value	8.9	9.8

Anacetrapib 100 mg vs Placebo: Difference in Percentages: -0.9 (95% CI -8.9 to 5.6)

Population analysed: The APaT population consisted of all randomized participants who received at least one dose of study treatment.

5 secondary outcomes are reported in the full results.

Adverse events

Time frame: Up to 64 weeks. Other adverse events reported at 5% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Anacetrapib 100 mg - Treatment Phase	18 of 203 (8.9%)	87 of 203 (42.9%)	0 of 203
Placebo - Treatment Phase	10 of 102 (9.8%)	41 of 102 (40.2%)	0 of 102
Anacetrapib 100 mg - Reversal Phase	3 of 196 (1.5%)	0 of 196 (0%)	0 of 196
Placebo - Reversal Phase	2 of 95 (2.1%)	0 of 95 (0%)	0 of 95

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 14 Oct 2019.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Background: PubMed 25743173Kastelein JJ, Besseling J, Shah S, Bergeron J, Langslet G, Hovingh GK, Al-Saady N, Koeijvoets M, Hunter J, Johnson-Levonas AO, Fable J, Sapre A, Mitchel Y. Anacetrapib as lipid-modifying therapy in patients with heterozygous familial hypercholesterolaemia (REALIZE): a randomised, double-blind, placebo-controlled, phase 3 study. Lancet. 2015 May 30;385(9983):2153-61. doi: 10.1016/S0140-6736(14)62115-2. Epub 2015 Mar 3.
Individual participant dataSharing planned: Yes. http://engagezone.msd.com/doc/ProcedureAccessClinicalTrialData.pdf

Related trials for Hyperlipoproteinemia Type II

Active, not recruitingObservational study
Early Detection of Familial Hypercholesterolaemia in Pre-school Children ( CzeCH-IT)
Familial Hypercholesterolaemia
Not yet recruiting
Penn Family Screening for Familial Hypercholesterolemia (NHLBI R33) Trial 3
Familial Hypercholesterolemia
Enrolling by invitationEarly Phase 1
Truway Diagnostic Tools in Primary Care
Chronic Disease, Type 2 Diabetes Mellitus (T2DM), Obesity, Overweight
Not yet recruitingObservational study
An Observational Study to Evaluate Treatment Patterns in High, Very High and Extreme Cardiovascular Risk Patients With Hypercholesterolemia, Including Familial Hypercholesterolemia
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Active, not recruitingObservational study
Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia (HoFH), Major Adverse Cardiovascular Events (MACE)

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.

Medical Director study director

Merck Sharp & Dohme LLC

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Anacetrapib in people with Hyperlipoproteinemia Type II and Hypercholesterolemia, Familial, run by Merck Sharp & Dohme LLC.

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 3 inclusion and 16 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 14 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. 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 28 Aug 2019). 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?

Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) - Treatment Phase (time frame: Baseline and Week 52). The study lists 17 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01524289, which also lists 1 linked publication.

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 14 Oct 2019.