Clinical trial NCT05425745: Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
Official title: A Placebo-Controlled, Double-Blind, Randomized, Phase 3 Study to Evaluate the Effect of 10 mg Obicetrapib in Participants With a History of HeFH Who Are Not Adequately Controlled by Their Lipid Modifying Therapies
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Dyslipidemias
High Cholesterol
Hypercholesterolemia
Familial Hypercholesterolemia
Lipid Metabolism Disorder
Metabolic Disease
Lipid Metabolism, Inborn Errors
Genetic Disease, Inborn
Hyperlipoproteinemias
Keywords: Obicetrapib
BROOKLYN
Cholesteryl ester transfer protein (CETP) inhibitor
Heterozygous Familial Hypercholesterolemia (HeFH)
LDL-C
Lead sponsor: NewAmsterdam Pharma
Sponsor class: INDUSTRY
First posted: 2022-06-21
Enrollment: 354
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Placebo-Controlled, double-blind, randomized
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 84 [PUC]
timeFrame: 84 Days
Secondary outcomes:
measure: Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 180 [Martin/Hopkins]
timeFrame: 180 Days
measure: Percent Change in Low Density Lipoprotein-Cholesterol (LDL-C) From Baseline to Day 365 [PUC]
timeFrame: 365 Days
measure: Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 180
timeFrame: 180 Days
measure: Percent Change in Apolipoprotein B (ApoB) From Baseline to Day 365
timeFrame: 365 Days
measure: Percent Change in Non-HDL-C From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in Non-HDL-C From Baseline to Day 180
timeFrame: 180 Days
measure: Percent Change in Non-HDL-C From Baseline to Day 365
timeFrame: 365 Days
measure: Percent Change in HDL-C From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in HDL-C From Baseline to Day 180
timeFrame: 180 Days
measure: Percent Change in HDL-C From Baseline to Day 365
timeFrame: 365 Days
measure: Percent Change in Lp(a) From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in Lp(a) From Baseline to Day 365
timeFrame: 365 Days
measure: Percent Change in Total Cholesterol From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in Total Cholesterol From Baseline to Day 180
timeFrame: 180 Days
measure: Percent Change in Total Cholesterol From Baseline to Day 365
timeFrame: 365 Days
measure: Percent Change in Triglycerides From Baseline to Day 84
timeFrame: 84 Days
measure: Percent Change in Triglycerides From Baseline to Day 180
timeFrame: 180 Days
measure: Precent Change in Triglycerides From Baseline to Day 365
timeFrame: 365 days
Other outcomes:
Locations:
city: Jonesboro
country: United States
facility: Site 01022
state: Arkansas
city: Toluca Lake
country: United States
facility: Site 01015
state: California
city: Sarasota
country: United States
facility: Site 01009
state: Florida
city: Boise
country: United States
facility: Site 01023
state: Idaho
city: Chicago
country: United States
facility: Site 01018
state: Illinois
city: Iowa City
country: United States
facility: Site 01012
state: Iowa
city: Baton Rouge
country: United States
facility: Site 01007
state: Louisiana
city: Port Gibson
country: United States
facility: Site 01005
state: Mississippi
city: Saint Louis
country: United States
facility: Site 01006
state: Missouri
city: Lincoln
country: United States
facility: Site 01011
state: Nebraska
city: Norfolk
country: United States
facility: Site 01004
state: Nebraska
city: Morristown
country: United States
facility: Site 01002
state: New Jersey
city: New Providence
country: United States
facility: Site 01010
state: New Jersey
city: North Massapequa
country: United States
facility: Site 01001
state: New York
city: Morganton
country: United States
facility: Site 01020
state: North Carolina
city: Winston-Salem
country: United States
facility: Site 01019
state: North Carolina
city: Chattanooga
country: United States
facility: Site 01008
state: Tennessee
city: El Paso
country: United States
facility: Site 01016
state: Texas
city: Houston
country: United States
facility: Site 01013
state: Texas
city: Suffolk
country: United States
facility: Site 01014
state: Virginia
city: Brampton
country: Canada
facility: Site 06007
city: Chicoutimi
country: Canada
facility: Site 06008
city: Halifax
country: Canada
facility: Site 06005
city: Montreal
country: Canada
facility: Site 06009
city: Montréal
country: Canada
facility: Site 06003
city: Québec
country: Canada
facility: Site 06004
city: Sherbrooke
country: Canada
facility: Site 06006
city: Vancouver
country: Canada
facility: Site 06001
city: Victoria
country: Canada
facility: Site 06002
city: Brno
country: Czechia
facility: Site 02006
city: Hradec Králové
country: Czechia
facility: Site 02002
city: Praha
country: Czechia
facility: Site 02003
city: Praha
country: Czechia
facility: Site 02005
city: Praha
country: Czechia
facility: Site 02004
city: Uherské Hradiště
country: Czechia
facility: Site 02001
city: Batumi
country: Georgia
facility: Site 022001
city: Tbilisi
country: Georgia
facility: Site 022003
city: Tbilisi
country: Georgia
facility: Site 022004
city: Tbilisi
country: Georgia
facility: Site 022010
city: Tbilisi
country: Georgia
facility: Site 022006
city: Tbilisi
country: Georgia
facility: Site 022007
city: Tbilisi
country: Georgia
facility: Site 022008
city: Tbilisi
country: Georgia
facility: Site 022002
city: Tbilisi
country: Georgia
facility: Site 022005
city: Tbilisi
country: Georgia
facility: Site 022009
city: Amsterdam
country: Netherlands
facility: Site 04001
city: Arnhem
country: Netherlands
facility: Site 04003
city: Deventer
country: Netherlands
facility: Site 04002
city: Eindhoven
country: Netherlands
facility: Site 04004
city: Roosendaal
country: Netherlands
facility: Site 04005
city: Rotterdam
country: Netherlands
facility: Site 04006
city: Bodø
country: Norway
facility: Site 023003
city: Oslo
country: Norway
facility: Site 023002
city: Białystok
country: Poland
facility: Site 05002
city: Zabrze
country: Poland
facility: Site 05005
city: Zamosc
country: Poland
facility: Site 05001
city: Łódź
country: Poland
facility: Site 05003
city: Łódź
country: Poland
facility: Site 05004
city: Bloemfontein
country: South Africa
facility: Site 018001
city: Cape Town
country: South Africa
facility: Site 018002
city: Centurion
country: South Africa
facility: Site 018009
city: Centurion
country: South Africa
facility: Site 018006
city: Parow
country: South Africa
facility: Site 018004
city: Somerset West
country: South Africa
facility: Site 018003
city: Somerset West
country: South Africa
facility: Site 018005
city: Tongaat
country: South Africa
facility: Site 018007
city: Umhlanga
country: South Africa
facility: Site 018008
city: Barcelona
country: Spain
facility: Site 017001
city: Barcelona
country: Spain
facility: Site 17002
city: Córdoba
country: Spain
facility: Site 17003
city: Figueras
country: Spain
facility: Site 17018
city: Granada
country: Spain
facility: Site 17011
city: Huelva
country: Spain
facility: Site 17017
city: Huesca
country: Spain
facility: Site 17012
city: La Coruña
country: Spain
facility: Site 17004
city: Las Palmas De Gran Canaria
country: Spain
facility: Site 17008
city: Madrid
country: Spain
facility: Site 17010
city: Madrid
country: Spain
facility: Site 17016
city: Málaga
country: Spain
facility: Site 17007
city: Sabadell
country: Spain
facility: Site 17013
city: Santiago De Compostela
country: Spain
facility: Site 17015
city: Sevilla
country: Spain
facility: Site 17009
city: Sevilla
country: Spain
facility: Site 17006
city: Valencia
country: Spain
facility: Site 17014
city: Zaragoza
country: Spain
facility: Site 17005
city: Birmingham
country: United Kingdom
facility: Site 014006
city: Bristol
country: United Kingdom
facility: Site 014012
city: Cardiff
country: United Kingdom
facility: Site 014009
city: Chichester
country: United Kingdom
facility: Site 014010
city: Dundee
country: United Kingdom
facility: Site 014001
city: London
country: United Kingdom
facility: Site 014002
city: Manchester
country: United Kingdom
facility: Site 014003
city: Penzance
country: United Kingdom
facility: Site 014011
city: Stevenage
country: United Kingdom
facility: Site 014005
city: West Bromwich
country: United Kingdom
facility: Site 014004
city: Wirral
country: United Kingdom
facility: Site 014008
Study officials:
affiliation: NewAmsterdam Pharma
name: Marc Ditmarsch
role: STUDY_DIRECTOR
References: