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

NewAmsterdam Pharma

Status and phase: Completed; Phase 3

Conditions: Dyslipidemias; Hypercholesterolemia; Lipid Metabolism Disorder; Lipid Metabolism, Inborn Errors; Metabolic Disease; Hyperlipoproteinemias; High Cholesterol; Genetic Disease, Inborn; Familial Hypercholesterolemia

Treatments: Drug: Placebo; Drug: Obicetrapib

Study type: Interventional

Funder types: Industry

Identifiers

NCT05425745

TA-8995-301

Details and patient eligibility

About

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).

Full 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.

Enrollment: 354 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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354 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

one placebo tablet once daily

Treatment:

Drug: Placebo

Obicetrapib 10 mg

Experimental group

Description:

one 10 mg Obicetrapib tablet once daily

Treatment:

Drug: Obicetrapib