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Ezetimibe

Phase 3

Familial Hypercholesterolemia | Small molecule | Metabolic |Organon & Co.|Last Updated: May 10, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03884452Ezetimibe (SCH 58235) Taken With Either Atorvastatin or Simvastatin in Participants With Familial Hypercholesterolemia (MK-0653-018)PHASE3 COMPLETED 50May 3, 2000May 24, 2001May 10, 2024 -
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Study Endpoints
Primary Endpoints
Percent change from baseline in Low Density Lipoprotein Cholesterol (LDL-C) measured directly
Baseline and Up to Week 12
Percentage of participants with an Adverse Event (AE)
Up to Week 12
Secondary Endpoints
Percent change from baseline in calculated LDL-C
Baseline and Up to Week 12
Percent change from baseline in Total Cholesterol (TC)
Baseline and Up to Week 12
Percent change from baseline in Triglycerides (TG)
Baseline and Up to Week 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Atorvastatin 80 mgEXPERIMENTAL80 mg atorvastatin taken orally, once daily for 12 weeks
Ezetimibe + Atorvastatin 40 mgEXPERIMENTAL10 mg ezetimibe and 40 mg atorvastatin taken orally, once daily for 12 weeks
Ezetimibe + Atorvastatin 80 mgEXPERIMENTAL10 mg ezetimibe and 80 mg atorvastatin taken orally, once daily for 12 weeks
Simvastatin 80 mgEXPERIMENTAL80 mg simvastatin taken orally, once daily for 12 weeks
Ezetimibe + Simvastatin 40 mgEXPERIMENTAL10 mg ezetimibe and 40 mg simvastatin taken orally, once daily for 12 weeks
Ezetimibe + Simvastatin 80 mgEXPERIMENTAL10 mg ezetimibe and 80 mg simvastatin taken orally, once daily for 12 weeks
Interventions
NameTypeDescription
AtorvastatinDRUGTablets taken orally once daily in the morning
SimvastatinDRUGTablets taken orally once daily in the morning or evening
EzetimibeDRUGTablet taken orally once daily in the morning or evening
Placebo for EzetimibeDRUGTablets taken orally once daily in the morning or evening
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Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo

Inclusion Criteria: * With a diagnosis of homozygous familial hypercholesterolemia * All females must have a negative pregnancy test prior to study entry. Women of child bearing potential must agree to practice an effective barrier method of birth control for the duration of the study, until one mo...

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