Trial Outcomes & Findings for MDCO-216 Infusions Leading to Changes in Atherosclerosis: A Novel Therapy in Development to Improve Cardiovascular Outcomes - Proof of Concept Intravascular Ultrasound (IVUS), Lipids, and Other Surrogate Biomarkers Trial (NCT NCT02678923)

NCT ID: NCT02678923

Last Updated: 2017-07-13

Results Overview

Change from Baseline to Day 36 post-randomization in PAV in a targeted (imaged) coronary artery for all anatomically comparable slices, as determined by IVUS. The change is calculated by subtracting the value at Baseline from the value at Day 36, with positive numbers to represent increases and negative numbers to represent decreases. Change in PAV was analyzed using an analysis of covariance (ANCOVA) model that included Baseline PAV as a covariate and treatment group as factor. Least Squares (LS) mean was adjusted for stratification factors of country and prior statin use.

Recruitment status

COMPLETED

Study phase

PHASE1/PHASE2

Target enrollment

126 participants

Primary outcome timeframe

Baseline, Day 36

Results posted on

2017-07-13

Participant Flow

Participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up intravascular ultrasound (IVUS) assessment were included in the modified intent-to-treat (mITT) population (primary/secondary analyses).

Participant milestones

Participant milestones
Measure
MDCO-216
20 milligrams/kilogram (mg/kg) of MDCO-216 administered intravenously (IV) as a 360 milliliter (mL) infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
360 mL of placebo (0.9% sodium chloride \[NaCl\] solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Overall Study
STARTED
59
67
Overall Study
Received at Least 1 Dose of Study Drug
58
64
Overall Study
mITT Population
52
61
Overall Study
COMPLETED
53
62
Overall Study
NOT COMPLETED
6
5

Reasons for withdrawal

Reasons for withdrawal
Measure
MDCO-216
20 milligrams/kilogram (mg/kg) of MDCO-216 administered intravenously (IV) as a 360 milliliter (mL) infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
360 mL of placebo (0.9% sodium chloride \[NaCl\] solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Overall Study
Protocol Deviation (Not Dosed)
1
3
Overall Study
Withdrawal by Subject
3
2
Overall Study
Lost to Follow-up
1
0
Overall Study
Did Not Return for Study Visits
1
0

Baseline Characteristics

MDCO-216 Infusions Leading to Changes in Atherosclerosis: A Novel Therapy in Development to Improve Cardiovascular Outcomes - Proof of Concept Intravascular Ultrasound (IVUS), Lipids, and Other Surrogate Biomarkers Trial

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure
MDCO-216
n=58 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=64 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Total
n=122 Participants
Total of all reporting groups
Age, Continuous
62.2 years
STANDARD_DEVIATION 10.6 • n=5 Participants
61.4 years
STANDARD_DEVIATION 10.3 • n=7 Participants
61.8 years
STANDARD_DEVIATION 10.4 • n=5 Participants
Sex: Female, Male
Female
13 Participants
n=5 Participants
16 Participants
n=7 Participants
29 Participants
n=5 Participants
Sex: Female, Male
Male
45 Participants
n=5 Participants
48 Participants
n=7 Participants
93 Participants
n=5 Participants
Race (NIH/OMB)
American Indian or Alaska Native
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Asian
0 Participants
n=5 Participants
1 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
1 Participants
n=5 Participants
0 Participants
n=7 Participants
1 Participants
n=5 Participants
Race (NIH/OMB)
Black or African American
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
White
56 Participants
n=5 Participants
63 Participants
n=7 Participants
119 Participants
n=5 Participants
Race (NIH/OMB)
More than one race
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Race (NIH/OMB)
Unknown or Not Reported
0 Participants
n=5 Participants
0 Participants
n=7 Participants
0 Participants
n=5 Participants
Region of Enrollment
Canada
3 Participants
n=5 Participants
3 Participants
n=7 Participants
6 Participants
n=5 Participants
Region of Enrollment
Czech Republic
3 Participants
n=5 Participants
2 Participants
n=7 Participants
5 Participants
n=5 Participants
Region of Enrollment
Hungary
6 Participants
n=5 Participants
8 Participants
n=7 Participants
14 Participants
n=5 Participants
Region of Enrollment
Netherlands
8 Participants
n=5 Participants
8 Participants
n=7 Participants
16 Participants
n=5 Participants
Region of Enrollment
Poland
38 Participants
n=5 Participants
43 Participants
n=7 Participants
81 Participants
n=5 Participants

PRIMARY outcome

Timeframe: Baseline, Day 36

Population: mITT population included all participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up IVUS assessment.

Change from Baseline to Day 36 post-randomization in PAV in a targeted (imaged) coronary artery for all anatomically comparable slices, as determined by IVUS. The change is calculated by subtracting the value at Baseline from the value at Day 36, with positive numbers to represent increases and negative numbers to represent decreases. Change in PAV was analyzed using an analysis of covariance (ANCOVA) model that included Baseline PAV as a covariate and treatment group as factor. Least Squares (LS) mean was adjusted for stratification factors of country and prior statin use.

Outcome measures

Outcome measures
Measure
MDCO-216
n=52 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=61 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Change From Baseline In Percent Atheroma Volume (PAV) At Day 36
-0.21 change in percent
Standard Error 0.386
-0.94 change in percent
Standard Error 0.382

SECONDARY outcome

Timeframe: Baseline, Day 36

Population: mITT population included all participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up IVUS assessment.

Change from Baseline to Day 36 post-randomization in normalized TAV in a targeted (imaged) coronary artery for all anatomically comparable slices, as determined by IVUS. The change is calculated by subtracting the value at Baseline from the value at Day 36, with positive numbers to represent increases and negative numbers to represent decreases. Change in TAV was analyzed using an analysis of covariance (ANCOVA) model that included Baseline TAV as a covariate and treatment group as factor. LS mean was adjusted for stratification factors of country and prior statin use.

Outcome measures

Outcome measures
Measure
MDCO-216
n=52 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=61 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Change From Baseline In Total Atheroma Volume (TAV) At Day 36
-6.33 cubic millimeter (mm^3)
Standard Error 3.425
-7.89 cubic millimeter (mm^3)
Standard Error 3.354

SECONDARY outcome

Timeframe: Baseline, Day 36

Population: mITT population included all participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up IVUS assessment.

Change in TAV from Baseline to Day 36 post-randomization of the most diseased 10-mm subsegment, as determined by IVUS. The change is calculated by subtracting the value at Baseline from the value at Day 36, with positive numbers to represent increases and negative numbers to represent decreases. Change in TAV was analyzed using an analysis of covariance (ANCOVA) model that included Baseline TAV for the most diseased 10-mm subsegment as a covariate and treatment group as factor. LS mean was adjusted for stratification factors of country and prior statin use.

Outcome measures

Outcome measures
Measure
MDCO-216
n=52 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=61 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Change From Baseline In TAV For The 10 Millimeters (mm) Subsegment With The Greatest Disease Burden At Day 36
-2.16 mm^3
Standard Error 1.809
-1.74 mm^3
Standard Error 1.908

SECONDARY outcome

Timeframe: Baseline through Day 36

Population: mITT population included all participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up IVUS assessment.

The number of participants with regression of coronary atherosclerosis is presented. For this Outcome Measure, the regression of coronary atherosclerosis is defined as a reduction in PAV from Baseline to Day 36 of greater than 2 standard deviations of the test-retest variability.

Outcome measures

Outcome measures
Measure
MDCO-216
n=52 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=61 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Participants With Regression Of Coronary Atherosclerosis As Measured By A PAV Change Greater Than 2 Standard Deviations Of Test-Retest Measurement Variability
NA participants
Test-retest requires IVUS to be done twice at the same time point and retest IVUS was not done.
NA participants
Test-retest requires IVUS to be done twice at the same time point and retest IVUS was not done.

SECONDARY outcome

Timeframe: Baseline through Day 36

Population: mITT population included all participants who were screened, enrolled, randomized, received at least one infusion of study drug, and who had an evaluable Baseline and Follow-up IVUS assessment.

The number of participants with regression of coronary atherosclerosis is presented. For this Outcome Measure, the regression of coronary atherosclerosis is defined as a change in PAV from Baseline to Day 36 of less than zero.

Outcome measures

Outcome measures
Measure
MDCO-216
n=52 Participants
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=61 Participants
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Participants With Regression Of Coronary Atherosclerosis As Measured By A PAV Change <0
29 Participants
41 Participants

Adverse Events

MDCO-216

Serious events: 10 serious events
Other events: 24 other events
Deaths: 0 deaths

Placebo

Serious events: 7 serious events
Other events: 12 other events
Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure
MDCO-216
n=58 participants at risk
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=64 participants at risk
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Cardiac disorders
Angina unstable
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Cardiac disorders
Atrial flutter
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Cardiac disorders
Coronary artery disease
5.2%
3/58 • Up to 59 days (±2 days) post randomization
3.1%
2/64 • Up to 59 days (±2 days) post randomization
Cardiac disorders
Ventricular extrasystoles
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Gastrointestinal disorders
Diarrhoea
0.00%
0/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
General disorders
Non-cardiac chest pain
0.00%
0/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
General disorders
Vessel puncture site haemorrhage
0.00%
0/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
Hepatobiliary disorders
Cholelithiasis
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Hepatobiliary disorders
Hepatitis acute
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Infections and infestations
Urinary tract infection
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Injury, poisoning and procedural complications
Coronary artery restenosis
0.00%
0/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
Investigations
Echocardiogram abnormal
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Nervous system disorders
Syncope
1.7%
1/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Respiratory, thoracic and mediastinal disorders
Pulmonary oedema
0.00%
0/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
Vascular disorders
Deep vein thrombosis
1.7%
1/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization

Other adverse events

Other adverse events
Measure
MDCO-216
n=58 participants at risk
20 mg/kg of MDCO-216 administered IV as a 360 mL infusion over 2 hours on Days 1, 8, 15, 22, and 29
Placebo
n=64 participants at risk
360 mL of placebo (0.9% NaCl solution) infusion, IV, over 2 hours on Days 1, 8, 15, 22, and 29
Blood and lymphatic system disorders
Anaemia
5.2%
3/58 • Up to 59 days (±2 days) post randomization
6.2%
4/64 • Up to 59 days (±2 days) post randomization
Cardiac disorders
Bradycardia
5.2%
3/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
General disorders
Vessel puncture site haematoma
3.4%
2/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Infections and infestations
Nasopharyngitis
3.4%
2/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Investigations
Alanine aminotransferase increased
3.4%
2/58 • Up to 59 days (±2 days) post randomization
3.1%
2/64 • Up to 59 days (±2 days) post randomization
Investigations
Blood creatine phosphokinase increased
0.00%
0/58 • Up to 59 days (±2 days) post randomization
3.1%
2/64 • Up to 59 days (±2 days) post randomization
Metabolism and nutrition disorders
Gout
3.4%
2/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Nervous system disorders
Dizziness
6.9%
4/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
Nervous system disorders
Headache
6.9%
4/58 • Up to 59 days (±2 days) post randomization
1.6%
1/64 • Up to 59 days (±2 days) post randomization
Psychiatric disorders
Insomnia
3.4%
2/58 • Up to 59 days (±2 days) post randomization
0.00%
0/64 • Up to 59 days (±2 days) post randomization
Renal and urinary disorders
Haematuria
6.9%
4/58 • Up to 59 days (±2 days) post randomization
3.1%
2/64 • Up to 59 days (±2 days) post randomization
Vascular disorders
Hypertension
3.4%
2/58 • Up to 59 days (±2 days) post randomization
3.1%
2/64 • Up to 59 days (±2 days) post randomization

Additional Information

Global Health Science Center

The Medicines Company

Phone: 1-888-977-6326

Results disclosure agreements

  • Principal investigator is a sponsor employee
  • Publication restrictions are in place