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Phase 3 N=354 Randomized Quadruple-blind Treatment

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

Dyslipidemias · High Cholesterol · Hypercholesterolemia · Familial Hypercholesterolemia · Lipid Metabolism Disorder

Enrolled (actual)
354
Serious AEs
6.0%
Results posted
Jun 2025
Primary outcome: Primary: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 84 [PUC] — .25; -36.05 percent change from baseline — p=<.0001

Study Design & Population

Study type
Interventional
Phase
Phase 3
Interventions
Obicetrapib (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
NewAmsterdam Pharma
Primary completion
May 2024

Outcome Measures

OutcomeResultp-value
PRIMARY
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 84 [PUC]
.25; -36.05 <.0001 sig
SECONDARY
Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 180 [Martin/Hopkins]
5.98; -31.80 <.0001 sig
SECONDARY
Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 365 [PUC]
10.30; -31.14 <.0001 sig
SECONDARY
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 84
2.93; -21.45 <.0001 sig
SECONDARY
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 180
6.02; -18.30 <.0001 sig
SECONDARY
Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 365
8.15; -17.62 <.0001 sig
SECONDARY
Percent Change in Non-HDL-C From Baseline to Day 84
2.83; -31.62 <.0001 sig
SECONDARY
Percent Change in Non-HDL-C From Baseline to Day 180
5.92; -27.08 <.0001 sig
SECONDARY
Percent Change in Non-HDL-C From Baseline to Day 365
11.64; -25.84 <.0001 sig
SECONDARY
Percent Change in HDL-C From Baseline to Day 84
1.26; 139.92 <.0001 sig
SECONDARY
Percent Change in HDL-C From Baseline to Day 180
2.63; 133.83 <.0001 sig
SECONDARY
Percent Change in HDL-C From Baseline to Day 365
6.28; 127.67 <.0001 sig
SECONDARY
Percent Change in Lp(a) From Baseline to Day 84
10.52; -35.42 <.0001 sig
SECONDARY
Percent Change in Lp(a) From Baseline to Day 365
24.37; -29.93 0.1648
SECONDARY
Percent Change in Total Cholesterol From Baseline to Day 84
2.34; 12.09
SECONDARY
Percent Change in Total Cholesterol From Baseline to Day 180
4.44; 14.43
SECONDARY
Percent Change in Total Cholesterol From Baseline to Day 365
9.32; 13.92
SECONDARY
Percent Change in Triglycerides From Baseline to Day 84
10.16; -1.57
SECONDARY
Percent Change in Triglycerides From Baseline to Day 180
12.43; 4.49
SECONDARY
Precent Change in Triglycerides From Baseline to Day 365
7.39; 2.27

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).

Eligibility Criteria

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)

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
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT05425745). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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