Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.

Phase 3

Completed

• Conditions: Dyslipidemias; Hyperlipoproteinemias; Familial Hypercholesterolemia; Lipid Metabolism Disorder; Metabolic Disease; Lipid Metabolism, Inborn Errors; Genetic Disease, Inborn; Hypercholesterolemia; High Cholesterol
• Interventions: Drug: Placebo; Drug: Obicetrapib

• Registration Number: NCT05425745
• Lead Sponsor: NewAmsterdam Pharma

Brief Summary

This study will be a placebo-controlled, double-blind, randomized, phase 3 study to Evaluate the Efficacy, Safety, and Tolerability of Obicetrapib in Participants with a History of Heterozygous Familial Hypercholesterolemia (HeFH).

Detailed Description

This study will be a placebo-controlled, double-blind, randomized, phase 3 study in participants with HeFH to evaluate the efficacy, safety, and tolerability of obicetrapib as an adjunct to diet and maximally tolerated lipid-lowering therapy. The screening period for this study will take up to 14 days. Afterwards, patients will be randomized to placebo or 10 mg obicetrapib for an 365-day treatment period. After the treatment period, participants will have an end-of-study follow-up visit.

Study Timeline

• Study First Submitted: 2022-06-15
• Study First Submitted QC: 2022-06-15
• Study First Posted: 2022-06-21
• Study Start: 2022-07-25
• Primary Completion: 2024-05-28
• Study Completion: 2024-05-28
• Status Verified: 2024-11
• Last Update Submitted: 2024-11-06
• Last Update Posted: 2024-11-07

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 354

Inclusion Criteria

• Have a history of heterozygous familial hypercholesterolemia (HeFH) by 1) Genotyping (not a screening assessment), WHO Criteria/Dutch Lipid Clinical Network Criteria with a score of > 8 points; and/or Simon Broome Register Diagnostic Criteria for definite or possible Familial Hypercholesterolemia (FH)
• Maximally tolerated lipid Modifying therapy for at least 8 weeks prior to screening such as: ATV (40 or 80), or (ROS 20 or 40 mg), Ezetimide, Bempedoic Acid, PCSK9 targeted therapy for at least 4 doses
• Fasting serum LDL-C ≥70 mg/dL (≥1.80 mmol/L)

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Exclusion Criteria

• New York Heart Association class II or IV heart failure or last known left ventricular ejection fraction < 30%;
• Hospitalized for heart failure within 5 years prior to Screening
• Major adverse cardiac event (MACE) within 3 months prior to Screening;
• HbA1c ≥10%, or fasting glucose
• Formal diagnosis of homozygous familial hypercholesterolemia (HoFH)
• Uncontrolled severe hypertension, defined as either systolic blood pressure ≥ 160 mmHg or diastolic blood pressure ≥100 mmHg prior to Randomization

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	one placebo tablet once daily
Obicetrapib 10 mg	Obicetrapib	one 10 mg Obicetrapib tablet once daily

Primary Outcome Measures

Name	Time	Method
Low Density Lipoprotein-Cholesterol (LDL-C)	Percent change from baseline to Day 84 in Low Density Lipoprotein-Cholesterol (LDL-C)	Obicetrapib compared to placebo on Low Density Lipoprotein-Cholesterol (LDL-C)

Secondary Outcome Measures

Name	Time	Method
Apolipoprotein B (ApoB)	Percent change from baseline to Day 84, 180, and 365 in ApoB	obicetrapib compared to placebo on ApoB
non-HDL-C	Percent change from baseline to Day 84, 180, and 364 in non-HDL-C	obicetrapib compared to placebo on non-HDL-C
Triglycerides	Percent change from baseline to Day 84, 180, and 365 in Triglycerides	obicetrapib compared to placebo on Triglycerides
Lp(a)	Percent change from baseline to Day 84 and 365 in Lp(a)	obicetrapib compared to placebo on Lp(a)
Low Density Lipoprotein-Cholesterol (LDL-C)	Percent change from baseline to Day 180, and 365 in Low Density Lipoprotein-Cholesterol (LDL-C)	obicetrapib compared to placebo on Low Density Lipoprotein-Cholesterol (LDL-C)
HDL-C	Percent change from baseline to Day 84, 180, and 365 in HDL-C	obicetrapib compared to placebo on HDL-C
Total Cholesterol	Percent change from baseline to Day 84, 180, and 365 in Total Cholesterol	obicetrapib compared to placebo on Total Cholesterol

Trial Locations

Locations (96)

Site 01015; 🇺🇸 Toluca Lake, California, United States

Site 018004; 🇿🇦 Parow, South Africa

Site 01009; 🇺🇸 Sarasota, Florida, United States

Site 01023; 🇺🇸 Boise, Idaho, United States

Site 022005; 🇬🇪 Tbilisi, Georgia

Site 05005; 🇵🇱 Zabrze, Poland

Site 01010; 🇺🇸 New Providence, New Jersey, United States

Site 06001; 🇨🇦 Vancouver, Canada

Site 01004; 🇺🇸 Norfolk, Nebraska, United States

Site 01002; 🇺🇸 Morristown, New Jersey, United States

Site 022004; 🇬🇪 Tbilisi, Georgia

Site 022010; 🇬🇪 Tbilisi, Georgia

Site 022006; 🇬🇪 Tbilisi, Georgia

Site 018008; 🇿🇦 Umhlanga, South Africa

Site 018003; 🇿🇦 Somerset West, South Africa

Site 018007; 🇿🇦 Tongaat, South Africa

Site 018005; 🇿🇦 Somerset West, South Africa

Site 01007; 🇺🇸 Baton Rouge, Louisiana, United States

Site 01006; 🇺🇸 Saint Louis, Missouri, United States

Site 01018; 🇺🇸 Chicago, Illinois, United States

Site 01016; 🇺🇸 El Paso, Texas, United States

Site 01001; 🇺🇸 North Massapequa, New York, United States

Site 06005; 🇨🇦 Halifax, Canada

Site 06009; 🇨🇦 Montreal, Canada

Site 01013; 🇺🇸 Houston, Texas, United States

Site 01020; 🇺🇸 Morganton, North Carolina, United States

Site 01014; 🇺🇸 Suffolk, Virginia, United States

Site 06007; 🇨🇦 Brampton, Canada

Site 06004; 🇨🇦 Québec, Canada

Site 06006; 🇨🇦 Sherbrooke, Canada

Site 02003; 🇨🇿 Praha, Czechia

Site 02002; 🇨🇿 Hradec Králové, Czechia

Site 022001; 🇬🇪 Batumi, Georgia

Site 02001; 🇨🇿 Uherské Hradiště, Czechia

Site 02005; 🇨🇿 Praha, Czechia

Site 02004; 🇨🇿 Praha, Czechia

Site 022003; 🇬🇪 Tbilisi, Georgia

Site 04001; 🇳🇱 Amsterdam, Netherlands

Site 04002; 🇳🇱 Deventer, Netherlands

Site 04005; 🇳🇱 Roosendaal, Netherlands

Site 023003; 🇳🇴 Bodø, Norway

Site 04006; 🇳🇱 Rotterdam, Netherlands

Site 023002; 🇳🇴 Oslo, Norway

Site 018002; 🇿🇦 Cape Town, South Africa

Site 018009; 🇿🇦 Centurion, South Africa

Site 018006; 🇿🇦 Centurion, South Africa

Site 17017; 🇪🇸 Huelva, Spain

Site 17018; 🇪🇸 Figueras, Spain

Site 17011; 🇪🇸 Granada, Spain

Site 17004; 🇪🇸 La Coruña, Spain

Site 17010; 🇪🇸 Madrid, Spain

Site 17008; 🇪🇸 Las Palmas De Gran Canaria, Spain

Site 014002; 🇬🇧 London, United Kingdom

Site 014006; 🇬🇧 Birmingham, United Kingdom

Site 014009; 🇬🇧 Cardiff, United Kingdom

Site 17006; 🇪🇸 Sevilla, Spain

Site 17005; 🇪🇸 Zaragoza, Spain

Site 014001; 🇬🇧 Dundee, United Kingdom

Site 014010; 🇬🇧 Chichester, United Kingdom

Site 014003; 🇬🇧 Manchester, United Kingdom

Site 17002; 🇪🇸 Barcelona, Spain

Site 17003; 🇪🇸 Córdoba, Spain

Site 017001; 🇪🇸 Barcelona, Spain

Site 17012; 🇪🇸 Huesca, Spain

Site 17007; 🇪🇸 Málaga, Spain

Site 17016; 🇪🇸 Madrid, Spain

Site 17013; 🇪🇸 Sabadell, Spain

Site 17009; 🇪🇸 Sevilla, Spain

Site 17014; 🇪🇸 Valencia, Spain

Site 01019; 🇺🇸 Winston-Salem, North Carolina, United States

Site 01012; 🇺🇸 Iowa City, Iowa, United States

Site 01008; 🇺🇸 Chattanooga, Tennessee, United States

Site 02006; 🇨🇿 Brno, Czechia

Site 06002; 🇨🇦 Victoria, Canada

Site 022007; 🇬🇪 Tbilisi, Georgia

Site 17015; 🇪🇸 Santiago De Compostela, Spain

Site 014012; 🇬🇧 Bristol, United Kingdom

Site 014011; 🇬🇧 Penzance, United Kingdom

Site 014005; 🇬🇧 Stevenage, United Kingdom

Site 014004; 🇬🇧 West Bromwich, United Kingdom

Site 014008; 🇬🇧 Wirral, United Kingdom

Site 05002; 🇵🇱 Białystok, Poland

Site 05004; 🇵🇱 Łódź, Poland

Site 01022; 🇺🇸 Jonesboro, Arkansas, United States

Site 04003; 🇳🇱 Arnhem, Netherlands

Site 01011; 🇺🇸 Lincoln, Nebraska, United States

Site 022008; 🇬🇪 Tbilisi, Georgia

Site 022009; 🇬🇪 Tbilisi, Georgia

Site 05003; 🇵🇱 Łódź, Poland

Site 018001; 🇿🇦 Bloemfontein, South Africa

Site 06008; 🇨🇦 Chicoutimi, Canada

Site 06003; 🇨🇦 Montréal, Canada

Site 022002; 🇬🇪 Tbilisi, Georgia

Site 04004; 🇳🇱 Eindhoven, Netherlands

Site 05001; 🇵🇱 Zamosc, Poland

Site 01005; 🇺🇸 Port Gibson, Mississippi, United States