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Protocol Details

An Intravenous, Open-Label, Single-Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of rhLCAT in Patients with Stable Coronary Artery Disease

This study is NOT currently recruiting participants.

Summary | Eligibility | Citations | Contacts




Sponsoring Institute

National Heart, Lung and Blood Institute (NHLBI)

Recruitment Detail

Type: Completed Study; data analyses ongoing
Gender: Male & Female
Min Age: 30
Max Age: 75

Referral Letter Required


Population Exclusion(s)



Recombinant LCAT;
Enzyme Replacement Therapy;
Lecithin cholesterol acltransferase

Recruitment Keyword(s)



Coronary Artery Disease (CAD)

Investigational Drug(s)


Investigational Device(s)



Drug: rhLCAT

Supporting Site

National Heart, Lung and Blood InstituteAlpha Core Pharma


- HDL cholesterol is known as the good cholesterol because it transports or moves cholesterol from the artery walls back to the liver. The liver processes the cholesterol so it can be removed from the body. Improving a person's HDL levels can decrease their risk of heart disease.

- An enzyme called LCAT helps make HDL in the blood which may enable HDL to transport it to the liver. Increasing LCAT should improve the body's ability to process cholesterol from arteries. However, there are no drugs that increase LCAT. An artificial form of human LCAT, called rhLCAT, is being tested to see if is safe and effective in humans with existing heart disease. rhLCAT may be able to improve HDL levels and help decrease risk of worsening heart disease.


- To see if rhLCAT is a safe and effective method of improving HDL levels in people with heart disease.


- Individuals between 30 and 75 years of age who have coronary artery disease.

- Participants must be stable with no recent heart damage.


- Participants will be screened with a physical exam and medical history. Blood and urine samples will also be collected.

- At the start of the study, participants will enter the hospital and have additional blood and urine tests. An electrocardiogram (ECG) will be obtained.

- In the hospital, participants will receive a single infusion of rhLCAT over about 1 hour. They will be monitored closely for 24 hours afterward, with frequent blood tests.

- Participants will have follow-up visits 3, 4, 5, and 8 days after leaving the hospital. They will have blood and urine tests. They will also have an electrocardiogram (ECG) and answer questions about any side effects of the treatment.

- A final follow-up visit will take place 28 days after the rhLCAT infusion.

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Subjects must meet all of the following inclusion criteria to be eligible for enrollment into the study:

A history of stable documented CAD as indicated by prior myocardial infarction, prior revascularization (e.g., percutaneous coronary intervention [PCI], coronary artery bypass graft [CABG]), angiographically proven coronary atherosclerosis, or non-invasive (e.g., MRI, CT) evidence of coronary artery disease. A subject presenting with angina must meet all the following criteria:

- Symptom complex has remained stable for at least 60 days with no significant change in frequency, duration, precipitating causes or ease of relief of angina

- No evidence of recent myocardial damage

Currently non-smoking males and females ages 30 to 85 years inclusive.

- Female subjects of child-bearing potential (neither pregnant nor breast-feeding) must be using adequate birth control during study conduct.

Chronic concomitant medications must be stable for at least 2 months prior to screening (for example, aspirin, lipid-altering drugs, ACE-inhibitors, B-blockers and nitrates used for the treatment CAD).

HDL-C < 50 mg/dL for men and < 55 mg/dL for women at the screening visit to enhance the ability to obtain PD information.

Body Mass Index (BMI) of approximately 18 to 35 kg/m2; and a total body weight greater than or equal to 50 kg (110 lbs) .

Willing to return for all clinic visits and complete all study-related procedures and able to understand and provide informed consent.


Subjects presenting with any of the following will not be included in the study:

Myocardial infarction, stroke, or coronary intervention/revascularization procedure within 6 months prior to dosing.

Chronic heart failure (> NYHA Class II).

Ventricular tachyarrhythmias.

Uncontrolled Type 2 (HbA1c > 8.5%) or Type 1 diabetes mellitus.

History of febrile illness within 5 days prior to dosing.

History of regular alcohol consumption exceeding 10 drinks per week.

12-lead ECG demonstrating QTc >500 msec at screening.

Blood donation of approximately 1 pint (500 mL) within 56 days prior to dosing.

Treatment with an investigational drug within 28 days prior to dosing.

Known hypersensitivity to heparin or IV infusion equipment, plastics, adhesive or silicone or known history of hypotension or infusion site reactions with IV administration.

Other severe acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with trial participation or investigational product administration or may interfere with the interpretation of trial results and, in the judgment of the investigator, would make the subject inappropriate for entry into this trial.

-Total bilirubin > 2.0 times the upper limit of normal; creatinine greater than or equal to 2.0 mg/dL; AST (SGOT) or ALT (SGPT) > 1.5 times the upper limit of normal; alkaline phosphatase > 1.5 times the upper limit of normal; or hemoglobin < 11 g/dL (<110 g/L).

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Shamburek RD, Bakker-Arkema R, Shamburek AM, Freeman LA, Amar MJ, Auerbach B, Krause BR, Homan R, Adelman SJ, Collins HL, Sampson M, Wolska A, Remaley AT. Safety and Tolerability of ACP-501, a Recombinant Human Lecithin:Cholesterol Acyltransferase, in a Phase 1 Single-Dose Escalation Study. Circ Res. 2016 Jan 8;118(1):73-82. doi: 10.1161/CIRCRESAHA.115.306223. Epub 2015 Dec 1.

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Principal Investigator

Referral Contact

For more information:

Robert D. Shamburek, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CRC BG RM 5-3581
(301) 496-3460

Robert D. Shamburek, M.D.
National Heart, Lung and Blood Institute (NHLBI)
NIHBC 10 - CRC BG RM 5-3581
(301) 496-3460

Office of Patient Recruitment
National Institutes of Health Clinical Center (CC)
Building 61, 10 Cloister Court
Bethesda, Maryland 20892
Toll Free: 1-800-411-1222
Local Phone: 301-451-4383
TTY: TTY Users Dial 7-1-1

Clinical Trials Number:


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