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INvestigating the Lowest Threshold of Vascular bENefits From LDL Lowering With a PCSK9 InhibiTor in healthY Volunteers

Not Applicable
Completed
Conditions
Atherosclerosis
Cardiovascular Diseases
Interventions
Other: Written Informed Consent
Other: Inclusion/Exclusion check
Other: Full Clinical Chemistry and Haematology Bloods
Other: Serum sample for systemic markers and lipid sub-fractions
Other: Pregnancy Test
Other: 12 Lead ECG
Other: Blood Pressure and Heart Rate
Other: Arterial Stiffness
Other: Central Haemodynamics
Other: Carotid Intima Media Thickness
Other: Forearm blood flow studies
Other: Concomitant medication check
Other: Medication compliance check (Pill count)
Other: Physical examination
Other: Medical history
Other: AE/SAE review & reporting
Drug: Dosing
Registration Number
NCT03273972
Lead Sponsor
Cambridge University Hospitals NHS Foundation Trust
Brief Summary

The INTENSITY-LOW study aims to answer if there are any limits to LDL reduction in relation to benefitting vascular health from a mechanistic viewpoint, and therefore potentially limitations to primary prevention in healthy volunteers by lowering LDL cholesterol using PCSK9 therapies. This research is being carried out because it is unclear what the lowest threshold of LDL cholesterol should be to attain significant reductions in CV risk in healthy individuals. It is also unknown whether there is a true limit of LDL cholesterol below which there is no further improvement in endothelial function in healthy people, and, whether this is associated with a reduction in markers of both systemic and vascular inflammation. This study will hope to provide evidence that the so-called pleiotropic effects of statins are actually mediated by a mechanism of LDL-cholesterol lowering per se and not necessarily a special therapeutic effect of statins.

Defining this may help identify individuals from the general population who may benefit from more aggressive lipid-lowering treatment than standard statin treatment in terms of CV morbidity and mortality.

This study will be conducted in healthy volunteers only, where participants will be randomized to either the Alirocumab arm (which is a therapy designed to lower cholesterol) or comparator arm. Both Alirocumab and comparator arms will be combined with Atorvastatin (another therapy designed to lower cholesterol) at the second dosing visit.

In total, thirty healthy individuals will be recruited to this single centre, randomized, single blind, parallel group, mechanistic physiological study which will be conducted at Cambridge University Hospitals NHS Foundation Trust/University of Cambridge. This study will be open to recruitment for one year and the total study duration for each participant will be approximately 10 weeks. There will be 4 study visits in total with a telephone follow up at the end of the study. Of these visits, two will be dosing visits and the total duration of treatment is approx. 4 weeks.

A series of non-invasive haemodynamic assessments and minimally invasive procedures including blood tests and forearm blood flow measurements will be conducted prior, during and post dosing to determine if there is an improvement of endothelia vascular function (as measured by nitric oxide bioavailability) and reduced systemic inflammation. This study is funded by JP Moulton Charitable Foundation.

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
30
Inclusion Criteria
  • Apparently healthy male or female individuals
  • Age 18-45 years old inclusive at screening
  • Body weight ≥ 45kg and BMI 18 -29.9 kg/m2
  • Fasting LDL-C < 4.1 mmol/l, TG <1.7 mmol/l and HDL-C ≥ 1.0 mmol for men and ≥1.3 mmol for women
  • Palpable brachial arterial pulse, as per study team assessment
  • Not currently eligible for statin therapy according to current treatment criteria
Exclusion Criteria
  • History of established CV disease (Coronary Heart Disease, Cerebrovascular Disease, Peripheral Vascular Disease or Abdominal Aortic Aneurysm)
  • Lipid lowering treatment at screening or within 6 weeks before screening
  • Pregnancy at any study visit
  • Ongoing anticoagulation therapy with warfarin or any of the DOAC/NOAC's
  • History of hypersensitivity to any of the study drugs/any known sensitivity to alirocumab or monoclonal antibodies
  • History of alcohol or drug abuse or dependence within 6 months of the study at screening
  • Current or previous history of regular smoking (defined as 1 pack-year) within the last 10 years.
  • History of hypertension or sustained BP ≥140/90 mmHg on repeated measurements at screening
  • History of type 1 or 2 diabetes or HbA1c ≥ 48 mmol/mol (6.5%) at screening
  • Chronic kidney disease defined as eGFR <60ml/min/1.73m2 at screening
  • Biological first-degree relatives who have experienced stroke, TIA, myocardial infarction or peripheral vascular disease (incident event at an age younger than: 55 for male and 65 for female relatives)
  • History of autoimmune inflammatory conditions
  • Lack of ability to provide informed consent
  • TSH >5.0 mu/l at screening
  • Clinically significant liver disease on the basis of screening bloods or history
  • History of myositis/rhabdomyolysis
  • Any concomitant condition that, at the discretion of the investigator, may affect the participants' ability to complete the study or the study results

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
Alirocumab Treatment ArmMedical historyV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmDosingV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment Arm12 Lead ECGV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmBlood Pressure and Heart RateV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmForearm blood flow studiesV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmMedical historyV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmWritten Informed ConsentV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmFull Clinical Chemistry and Haematology BloodsV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmArterial StiffnessV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmDosingV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmInclusion/Exclusion checkV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmSerum sample for systemic markers and lipid sub-fractionsV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmPregnancy TestV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmBlood Pressure and Heart RateV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmInclusion/Exclusion checkV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmCarotid Intima Media ThicknessV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmMedication compliance check (Pill count)V2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmAE/SAE review & reportingV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment Arm12 Lead ECGV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmForearm blood flow studiesV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmSerum sample for systemic markers and lipid sub-fractionsV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmPregnancy TestV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmCentral HaemodynamicsV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmConcomitant medication checkV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Alirocumab Treatment ArmPhysical examinationV2: Day 1 - Alirocumab 150mg (subcutaneous injection) V3: Day 15 +/- 3 days - Alirocumab 150mg (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmFull Clinical Chemistry and Haematology BloodsV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmCentral HaemodynamicsV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmWritten Informed ConsentV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmCarotid Intima Media ThicknessV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmAE/SAE review & reportingV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmArterial StiffnessV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmConcomitant medication checkV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmMedication compliance check (Pill count)V2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Comparator Treatment ArmPhysical examinationV2: Day 1 - Placebo (subcutaneous injection) V3: Day 15 +/- 3 days - Placebo (subcutaneous injection) plus Atorvastatin 20mg (oral) approx. 14 days prescription.
Primary Outcome Measures
NameTimeMethod
Forearm Blood Flow (FBF) - In response to intra-arterial Acetylcholine infusion, comparing Alirocumab to placebo.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Secondary Outcome Measures
NameTimeMethod
Carotid IMT4 weeks

Change in Carotid IMT between visits and different treatment regimes.

Augmentation Index (an indicator of arterial stiffness)4 weeks

Change in Augmentation Index between visits and different treatment regimes.

Forearm blood flow in response to intra-arterial Sodium Nitroprusside, comparing Alirocumab with statin to statin alone.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Forearm blood flow in response to intra-arterial Acetylcholine infusion, comparing Alirocumab with statin to statin alone.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Forearm blood flow in response to intra-arterial Sodium Nitroprusside, comparing Alirocumab to placebo.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Forearm blood flow in response to intra-arterial L-NMMA infusion, comparing Alirocumab with statin to statin alone.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Total and LDL-cholesterol4 weeks

Correlation of change in responses to Acetylcholine between groups.

Forearm blood flow in response to intra-arterial Sodium Nitroprusside, comparing Alirocumab to statin.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Forearm blood flow in response to intra-arterial L-NMMA infusion, comparing Alirocumab to placebo.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Forearm blood flow in response to intra-arterial L-NMMA infusion, comparing Alirocumab to statin.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Physical examination, vital signs, ECG, laboratory tests and adverse event assessment to determine Safety and tolerability parameters4 weeks

Including physical examination, blood pressure, heart rate, 12-lead electrocardiograms (ECGs), clinical laboratory tests side effects and adverse event reporting.

Forearm Blood Flow in response to intra-arterial Acetylcholine infusion, comparing Alirocumab to statin.4 weeks

Change in FBF ratio, as measured by absolute \& percent change in venous occlusion plethysmography

Pulse Wave Velocity4 weeks

Change in aortic Pulse Wave Velocity between visits and different treatment regimes.

Systemic inflammation4 weeks

Change in lipid profile, hsCRP and other markers of systemic inflammation between visits and different treatment regimes.

Trial Locations

Locations (1)

Addenbrooke's Hospital

🇬🇧

Cambridge, Cambridgeshire, United Kingdom

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