Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
- Conditions
- DyslipidemiasHyperlipoproteinemiasFamilial HypercholesterolemiaLipid Metabolism DisorderMetabolic DiseaseLipid Metabolism, Inborn ErrorsGenetic Disease, InbornHypercholesterolemiaHigh Cholesterol
- Interventions
- Drug: Placebo
- 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.
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 354
- 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)
- 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
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 Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 84 [PUC] 84 Days LS mean percent change from baseline to Day 84 in Low-Density Lipoprotein Cholesterol (LDL-C) in the obicetrapib group compared to the placebo group \[PUC\]. LDL-C level was measured by preparative ultracentrifugation (PUC).
- Secondary Outcome Measures
Name Time Method Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 180 [Martin/Hopkins] 180 Days LS mean percent change from baseline to Day 180 in Low-Density Lipoprotein Cholesterol (LDL-C) in the obicetrapib group compared to the placebo group \[Martin/Hopkins\]. LDL-C value was calculated using the Martin/Hopkins equation unless TG \>= 400 mg/dL or LDL-C \<= 50 mg/dL; where, LDL-C value was measured directly by preparative ultracentrifugation (PUC).
Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 365 [PUC] 365 Days LS mean percent change from baseline to Day 365 in Low-Density Lipoprotein Cholesterol (LDL-C) in the obicetrapib group compared to the placebo group \[PUC\]. LDL-C level was measured by preparative ultracentrifugation (PUC).
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in apolipoprotein B (ApoB) in obicetrapib group compared to the placebo group.
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 180 180 Days LS mean percent change from baseline to Day 180 in apolipoprotein B (ApoB) in obicetrapib group compared to the placebo group
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 365 365 Days LS mean percent change from baseline to Day 365 in apolipoprotein B (ApoB) in obicetrapib group compared to the placebo group
Percent Change in Non-HDL-C From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in Non-high-density Lipoprotein Cholesterol (Non-HDL-C) in obicetrapib group compared to the placebo group
Percent Change in Non-HDL-C From Baseline to Day 180 180 Days LS mean percent change from baseline to Day 180 in Non-high-density Lipoprotein Cholesterol (non-HDL-C) in obicetrapib group compared to the placebo group
Percent Change in Non-HDL-C From Baseline to Day 365 365 Days LS mean percent change from baseline to Day 365 in Non-high-density Lipoprotein Cholesterol (non-HDL-C) in obicetrapib group compared to the placebo group
Percent Change in HDL-C From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in High-density Lipoprotein Cholesterol (HDL-C) in obicetrapib group compared to the placebo group
Percent Change in HDL-C From Baseline to Day 180 180 Days LS mean percent change from baseline to Day 180 in High-density Lipoprotein Cholesterol (HDL-C) in obicetrapib group compared to the placebo group
Percent Change in HDL-C From Baseline to Day 365 365 Days LS mean percent change from baseline to Day 365 in High-density Lipoprotein Cholesterol (HDL-C) in obicetrapib group compared to the placebo group
Percent Change in Lp(a) From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in Lipoprotein (a) \[Lp(a)\] in obicetrapib group compared to the placebo group
Percent Change in Lp(a) From Baseline to Day 365 365 Days LS mean percent change from baseline to Day 365 in Lipoprotein (a) \[Lp(a)\] in obicetrapib group compared to the placebo group
Percent Change in Total Cholesterol From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in Total Cholesterol (TC) in obicetrapib group compared to the placebo group
Percent Change in Total Cholesterol From Baseline to Day 180 180 Days LS mean percent change from baseline to Day 180 in Total Cholesterol in obicetrapib group compared to the placebo group
Percent Change in Total Cholesterol From Baseline to Day 365 365 Days LS mean percent change from baseline to Day 365 in Total Cholesterol in obicetrapib group compared to the placebo group
Percent Change in Triglycerides From Baseline to Day 84 84 Days LS mean percent change from baseline to Day 84 in Triglycerides in obicetrapib group compared to the placebo group
Percent Change in Triglycerides From Baseline to Day 180 180 Days LS mean percent change from baseline to Day 180 in Triglycerides in obicetrapib group compared to the placebo group
Precent Change in Triglycerides From Baseline to Day 365 365 days LS mean percent change from baseline to Day 365 in Triglycerides in obicetrapib group compared to the placebo group
Trial Locations
- Locations (96)
Site 01022
🇺🇸Jonesboro, Arkansas, United States
Site 01015
🇺🇸Toluca Lake, California, United States
Site 01009
🇺🇸Sarasota, Florida, United States
Site 01023
🇺🇸Boise, Idaho, United States
Site 01018
🇺🇸Chicago, Illinois, United States
Site 01012
🇺🇸Iowa City, Iowa, United States
Site 01007
🇺🇸Baton Rouge, Louisiana, United States
Site 01005
🇺🇸Port Gibson, Mississippi, United States
Site 01006
🇺🇸Saint Louis, Missouri, United States
Site 01011
🇺🇸Lincoln, Nebraska, United States
Scroll for more (86 remaining)Site 01022🇺🇸Jonesboro, Arkansas, United States