Clinical Trials Register

Summary
EudraCT Number:	2021-005064-22
Sponsor's Protocol Code Number:	TA-8995-301
National Competent Authority:	Czechia - SUKL
Clinical Trial Type:	EEA CTA
Trial Status:	Ongoing
Date on which this record was first entered in the EudraCT database:	2022-07-28
Trial results

Index
A. PROTOCOL INFORMATION
B. SPONSOR INFORMATION
C. APPLICANT IDENTIFICATION
D. IMP IDENTIFICATION
D.8 INFORMATION ON PLACEBO
E. GENERAL INFORMATION ON THE TRIAL
F. POPULATION OF TRIAL SUBJECTS
G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
P. END OF TRIAL

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A. Protocol Information

A.1

Member State Concerned

Czechia - SUKL

A.2

EudraCT number

2021-005064-22

A.3

Full title of the trial

Obicetrapib on Top of Maximum Tolerated Lipid-Modifying
Therapies (BROOKLYN): A Placebo-Controlled, Double-Blind,
Randomized, Phase 3 Study to Evaluate the Effect of 10 mg
Obicetrapib in Participants With a History of HeFH and LDL-C
≥70 mg/dL Who are Not Adequately Controlled by Their
Lipid-Modifying Therapies

Obicetrapib v kombinaci s maximální tolerovanou dávkou při léčbě modifikující hladiny lipidů (BROOKLYN): placebem kontrolovaná, dvojitě zaslepená, randomizovaná studie fáze III hodnotící účinek 10 mg obicetrapibu u pacientů s heterozygotní familiární hypercholesterolémií (HeFH) a LDL-C ≥ 70 mg/dl, kteří dostatečně nereagují na svoji léčbu modifikující hladiny lipidů

A.3.1

Title of the trial for lay people, in easily understood, i.e. non-technical, language

A.4.1

Sponsor's protocol code number

TA-8995-301

A.7

Trial is part of a Paediatric Investigation Plan

A.8

EMA Decision number of Paediatric Investigation Plan

B. Sponsor Information
B.Sponsor: 1
B.1.1	Name of Sponsor	NewAmsterdam Pharma B.V.
B.1.3.4	Country	Netherlands
B.3.1 and B.3.2	Status of the sponsor	Commercial
B.4 Source(s) of Monetary or Material Support for the clinical trial:
B.4.1	Name of organisation providing support	NewAmsterdam Pharma BV
B.4.2	Country	Netherlands
B.5 Contact point designated by the sponsor for further information on the trial
B.5.1	Name of organisation	NewAmsterdam Pharma B.V.
B.5.2	Functional name of contact point	Clinical Operations
B.5.3	Address:
B.5.3.1	Street Address	Gooimeer 2-35
B.5.3.2	Town/ city	Naarden
B.5.3.3	Post code	1411 DC
B.5.3.4	Country	Netherlands
B.5.4	Telephone number	+31 35 2062971
B.5.5	Fax number	+31 35 2062971
B.5.6	E-mail	marc.ditmarsch@newamsterdampharma.com

D. IMP Identification
D.IMP: 1
D.1.2 and D.1.3	IMP Role	Test
D.2	Status of the IMP to be used in the clinical trial
D.2.1	IMP to be used in the trial has a marketing authorisation	No
D.2.5	The IMP has been designated in this indication as an orphan drug in the Community	No
D.2.5.1	Orphan drug designation number
D.3 Description of the IMP
D.3.1	Product name	obicetrapib
D.3.2	Product code	TA-8995
D.3.4	Pharmaceutical form	Film-coated tablet
D.3.4.1	Specific paediatric formulation	No
D.3.7	Routes of administration for this IMP	Oral use
D.3.8 to D.3.10 IMP Identification Details (Active Substances)
D.3.8	INN - Proposed INN	OBICETRAPIB
D.3.9.1	CAS number	866399-89-5
D.3.9.2	Current sponsor code	TA-8995
D.3.9.4	EV Substance Code	SUB188624
D.3.10	Strength
D.3.10.1	Concentration unit	mg milligram(s)
D.3.10.2	Concentration type	equal
D.3.10.3	Concentration number	10
D.3.11 The IMP contains an:
D.3.11.1	Active substance of chemical origin	Yes
D.3.11.2	Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP)	No
	The IMP is a:
D.3.11.3	Advanced Therapy IMP (ATIMP)	No
D.3.11.3.1	Somatic cell therapy medicinal product	No
D.3.11.3.2	Gene therapy medical product	No
D.3.11.3.3	Tissue Engineered Product	No
D.3.11.3.4	Combination ATIMP (i.e. one involving a medical device)	No
D.3.11.3.5	Committee on Advanced therapies (CAT) has issued a classification for this product	No
D.3.11.4	Combination product that includes a device, but does not involve an Advanced Therapy	No
D.3.11.5	Radiopharmaceutical medicinal product	No
D.3.11.6	Immunological medicinal product (such as vaccine, allergen, immune serum)	No
D.3.11.7	Plasma derived medicinal product	No
D.3.11.8	Extractive medicinal product	No
D.3.11.9	Recombinant medicinal product	No
D.3.11.10	Medicinal product containing genetically modified organisms	No
D.3.11.11	Herbal medicinal product	No
D.3.11.12	Homeopathic medicinal product	No
D.3.11.13	Another type of medicinal product	No

D.8 Information on Placebo
D.8 Placebo: 1
D.8.1	Is a Placebo used in this Trial?	Yes
D.8.3	Pharmaceutical form of the placebo	Film-coated tablet
D.8.4	Route of administration of the placebo	Oral use

E. General Information on the Trial

E.1 Medical condition or disease under investigation

E.1.1

Medical condition(s) being investigated

dyslipidemia
heterozygous familial hypercholesterolemia (HeFH)

dyslipidémie
heterozygotní familiární hypercholesterolémie (HeFH)

E.1.1.1

Medical condition in easily understood language

treatment of high cholesterol

léčba vysokého cholesterolu

E.1.1.2

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

MedDRA Classification

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.1
E.1.2	Level	LLT
E.1.2	Classification code	10020049
E.1.2	Term	High cholesterol
E.1.2	System Organ Class	100000004848

E.1.2 Medical condition or disease under investigation

E.1.2	Version	20.0
E.1.2	Level	LLT
E.1.2	Classification code	10057079
E.1.2	Term	Heterozygous familial hypercholesterolemia
E.1.2	System Organ Class	100000004850

E.1.3

Condition being studied is a rare disease

E.2 Objective of the trial

E.2.1

Main objective of the trial

To evaluate the effect of obicetrapib on LDL-C levels at Day 84.

E.2.2

Secondary objectives of the trial

- To evaluate the effect of obicetrapib on fasting apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non HDL C), high-density lipoprotein cholesterol (HDL-C), total cholesterol (TC), and triglycerides (TG) at Days 84, 180, and 365;
- To evaluate the effect of obicetrapib on fasting LDL-C levels at Days 180 and 365;
- To evaluate the effect of obicetrapib on fasting lipoprotein (a) (Lp[a]) at Days 84 and 365; and
- To evaluate the safety and tolerability profile of obicetrapib in a representative population of adult males and females with HeFH, assessed by adverse events (AEs), events of special interest (ESIs), vital signs (including blood pressure), electrocardiogram (ECG) measurements, and clinical laboratory values.

E.2.3

Trial contains a sub-study

E.3

Principal inclusion criteria

1. Are willing and able to give written informed consent before initiation of any study-related procedures and willing to comply with all required study procedures;
2. Are male or female and ≥18 years of age at Screening;
o Females may be enrolled if all 3 of the following criteria are met:
-They are not pregnant;
-They are not breastfeeding; and
-They do not plan on becoming pregnant during the study.
o Females of childbearing potential must have a negative urine pregnancy test at Screening.
3. Have a history of HeFH as defined by at least 1 of the following:
o Genotyping by a central laboratory;
o Clinical assessment using the WHO Criteria/Dutch Lipid Clinical Network Criteria with a score of >8 points; and/or
o Meet the Simon Broome Register Diagnostic Criteria for definite or possible Familial Hypercholesterolemia (FH).
4. Are on maximally tolerated lipid-modifying therapy, as an adjunct to diet, defined as follows:
o A statin at a maximally tolerated stable dose;
o Ezetimibe for at least 8 weeks with or without maximally tolerated statin prior to Screening;
o Bempedoic acid for at least 8 weeks in combination with maximally tolerated statin prior to Screening;
o A proprotein convertase subtilisin/kexin type 9 (PCSK9)-targeted therapy alone or in combination with other lipid-modifying therapy for at least 4 stable doses prior to Screening;
5. Have a fasting serum LDL-C ≥70 mg/dL (≥1.81 mmol/L) at Screening;
6. Have fasting TG <400 mg/dL (<4.52 mmol/L) at Screening; and
7. Have an estimated glomerular filtration rate ≥30 mL/min/1.73 m2
calculated using the Chronic Kidney Disease Epidemiology Collaboration equation at Screening.

Other protocol-defined criteria apply.

E.4

Principal exclusion criteria

1. Have current or any previous history of New York Heart Association class III or IV heart failure (HF) or left ventricular ejection fraction <30%;
2. Have been hospitalized for HF within 5 years prior to Screening;
3. Have had any of the following clinical events within 3 months prior to Screening:
o Non-fatal myocardial infarction;
o Non-fatal stroke;
o Non-elective coronary revascularization; and/or
o Hospitalization for unstable angina and/or chest pain.
4. Have uncontrolled severe hypertension, defined as either systolic blood pressure ≥160 mmHg or diastolic blood pressure ≥100 mmHg prior to Randomization taken as the average of triplicate measurements. One triplicate retest will be allowed during the same visit, at which point if the retest result is no longer exclusionary, the participant may be randomized;
5. Have a formal diagnosis of homozygous FH;
6. Have active liver disease, defined as any known current infectious, neoplastic, or metabolic pathology of the liver; unexplained elevations in alanine aminotransferase (ALT) or aspartate
aminotransferase (AST) >3 x upper limit of normal (ULN); or total bilirubin >2 x ULN at Screening;
7. Have glycosylated hemoglobin ≥10.0% (≥0.100 hemoglobin fraction) or a fasting glucose ≥270 mg/dL (≥15.0 mmol/L) at Screening;
8. Have thyroid-stimulating hormone >1.5 X ULN at Screening;
9. Have creatine kinase >3 x ULN at Screening;
10. Have a history of a malignancy that required surgery (excluding local and wide local excision), radiation therapy, and/or systemic therapy during the 3 years prior to Randomization;
11. Have a known history of alcohol and/or drug abuse within 5 years prior to Randomization;
12. Have received treatment with other investigational products or devices within 30 days of Screening or 5 half-lives of the previous investigational product, whichever is longer;
13.Are taking gemfibrozil;
14. Have planned use of other investigational products or devices during the course of the study;
15. Have participated in any clinical study evaluating obicetrapib;
16. Have a known allergy or hypersensitivity to the study drug, placebo, or any of the excipients in the study drug or placebo; or
17. Have any participant condition that, according to the Investigator, could interfere with the conduct of the study, such as, but not limited to, the following:
o Are unable to communicate or to cooperate with the Investigator;
o Are unable to understand the protocol requirements, instructions and study-related restrictions, and the nature, scope, and possible consequences of the study (including participants whose cooperation is doubtful due to drug abuse or alcohol dependency);
o Are unlikely to comply with the protocol requirements, instructions, and study-related restrictions (eg, uncooperative attitude, inability to return for follow-up visits, and improbability of completing the study);
o Have any medical or surgical condition which, in the opinion of the Investigator, would put the participant at increased risk from participating in the study; or
o Are directly involved in the conduct of the study.

Other protocol-defined criteria apply.

E.5 End points

E.5.1

Primary end point(s)

The percent change from Baseline to Day 84 in fasting LDL-C in
the obicetrapib group compared to the placebo group.

E.5.1.1

Timepoint(s) of evaluation of this end point

Baseline, Day 84

E.5.2

Secondary end point(s)

1. Percent change from Baseline to Days 180 and 365 in fasting LDL-C in the obicetrapib group compared to the placebo group;
2. Percent change from Baseline to Days 84, 180, and 365 in fasting ApoB in the obicetrapib group compared to the placebo group;
3. Percent change from Baseline to Days 84, 180, and 365 in fasting non-HDL-C in the obicetrapib group compared to the placebo group;
4. Percent change from Baseline to Days 84, 180, and 365 in fasting HDL-C in the obicetrapib group compared to the placebo group;
5. Percent change from Baseline to Days 84 and 365 in fasting Lp(a) in the obicetrapib group compared to the placebo group;
6. Percent change from Baseline to Days 84, 180, and 365 in fasting TC in the obicetrapib group compared to the placebo group; and
7. Percent change from Baseline to Days 84, 180, and 365 in fasting TG in the obicetrapib group compared to the placebo group.
8. Trough levels of obicetrapib from Baseline to Day 365 in the obicetrapib group.

E.5.2.1

Timepoint(s) of evaluation of this end point

Baseline, Days 84, 180 and 365

E.6 and E.7 Scope of the trial

E.6

Scope of the trial

E.6.1

Diagnosis

E.6.2

Prophylaxis

E.6.3

Therapy

Yes

E.6.4

Safety

Yes

E.6.5

Efficacy

Yes

E.6.6

Pharmacokinetic

Yes

E.6.7

Pharmacodynamic

E.6.8

Bioequivalence

E.6.9

Dose response

E.6.10

Pharmacogenetic

E.6.11

Pharmacogenomic

E.6.12

Pharmacoeconomic

E.6.13

Others

E.7

Trial type and phase

E.7.1

Human pharmacology (Phase I)

E.7.1.1

First administration to humans

E.7.1.2

Bioequivalence study

E.7.1.3

Other

E.7.1.3.1

Other trial type description

E.7.2

Therapeutic exploratory (Phase II)

E.7.3

Therapeutic confirmatory (Phase III)

Yes

E.7.4

Therapeutic use (Phase IV)

E.8 Design of the trial

E.8.1

Controlled

Yes

E.8.1.1

Randomised

Yes

E.8.1.2

Open

E.8.1.3

Single blind

E.8.1.4

Double blind

Yes

E.8.1.5

Parallel group

E.8.1.6

Cross over

E.8.1.7

Other

E.8.2

Comparator of controlled trial

E.8.2.1

Other medicinal product(s)

E.8.2.2

Placebo

Yes

E.8.2.3

Other

E.8.2.4

Number of treatment arms in the trial

E.8.3

The trial involves single site in the Member State concerned

E.8.4

The trial involves multiple sites in the Member State concerned

Yes

E.8.4.1

Number of sites anticipated in Member State concerned

E.8.5

The trial involves multiple Member States

Yes

E.8.5.1

Number of sites anticipated in the EEA

E.8.6 Trial involving sites outside the EEA

E.8.6.1

Trial being conducted both within and outside the EEA

Yes

E.8.6.2

Trial being conducted completely outside of the EEA

E.8.6.3

If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned

Canada

Czechia

Georgia

Netherlands

Norway

Poland

South Africa

Spain

United Kingdom

United States

E.8.7

Trial has a data monitoring committee

Yes

E.8.8

Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial

LSLV

E.8.9 Initial estimate of the duration of the trial

E.8.9.1

In the Member State concerned years

E.8.9.1

In the Member State concerned months

E.8.9.1

In the Member State concerned days

E.8.9.2

In all countries concerned by the trial years

E.8.9.2

In all countries concerned by the trial months

F. Population of Trial Subjects

F.1 Age Range

F.1.1

Trial has subjects under 18

F.1.1.1

In Utero

F.1.1.2

Preterm newborn infants (up to gestational age < 37 weeks)

F.1.1.3

Newborns (0-27 days)

F.1.1.4

Infants and toddlers (28 days-23 months)

F.1.1.5

Children (2-11years)

F.1.1.6

Adolescents (12-17 years)

F.1.2

Adults (18-64 years)

Yes

F.1.2.1

Number of subjects for this age range:

241

F.1.3

Elderly (>=65 years)

Yes

F.1.3.1

Number of subjects for this age range:

113

F.2 Gender

F.2.1

Female

Yes

F.2.2

Male

Yes

F.3 Group of trial subjects

F.3.1

Healthy volunteers

F.3.2

Patients

Yes

F.3.3

Specific vulnerable populations

Yes

F.3.3.1

Women of childbearing potential not using contraception

F.3.3.2

Women of child-bearing potential using contraception

Yes

F.3.3.3

Pregnant women

F.3.3.4

Nursing women

F.3.3.5

Emergency situation

F.3.3.6

Subjects incapable of giving consent personally

F.3.3.7

Others

F.4 Planned number of subjects to be included

F.4.1

In the member state

F.4.2

For a multinational trial

F.4.2.1

In the EEA

114

F.4.2.2

In the whole clinical trial

354

F.5

Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)

standard of care

G. Investigator Networks to be involved in the Trial
G.4 Investigator Network to be involved in the Trial: 1
G.4.1	Name of Organisation	Vascular Research Network (VRN)
G.4.3.4	Network Country	Netherlands
G.4 Investigator Network to be involved in the Trial: 2
G.4.1	Name of Organisation	WCN
G.4.3.4	Network Country	Netherlands

N. Review by the Competent Authority or Ethics Committee in the country concerned

Competent Authority Decision

Authorised

Date of Competent Authority Decision

2022-08-25

Ethics Committee Opinion of the trial application

Favourable

Ethics Committee Opinion: Reason(s) for unfavourable opinion

Date of Ethics Committee Opinion

2022-08-10

P. End of Trial
P.	End of Trial Status	Ongoing

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