MedPath

Study to Assess the Safety, Pharmacokinetics, and Pharmacodynamics of Multiple Doses of COR-001

Phase 1
Completed
Conditions
Anemia
Interventions
Other: Placebo
Registration Number
NCT02868229
Lead Sponsor
Novo Nordisk A/S
Brief Summary

This is a randomized, double-blind, placebo-controlled trial designed to evaluate the safety, pharmacokinetics, and pharmacodynamic effects of multiple doses of COR-001 or placebo

Detailed Description

Not available

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
61
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
PARALLEL
Arm && Interventions
GroupInterventionDescription
COR-001COR-001-
PlaceboPlacebo-
Primary Outcome Measures
NameTimeMethod
Characterization of Maximum Tolerated Dose (MTD)Weeks 0-24

The MTD assessment was based on safety data. If more than 2 of 8 active participants in a cohort experience a Dose-Limiting Toxicities (DLT), the MTD was considered to have been exceeded. The DLT threshold was defined using a threshold of greater than or equal to (\>=) Grade 3 events, which includes severe: infusion-related reactions, cardiopulmonary infusion reactions, anaphylaxis, or hypersensitivity.

DLTs are defined as follows:

1. Confirmed Grade 3 neutropenia and representing a decline of \> 25% from baseline

2. Serious adverse events (SAEs) of infection in the presence of confirmed Grade 2 or higher new onset lymphopenia or new onset neutropenia.

3. ≥ Grade 3 ALT (Alanine transaminase) or AST(Aspartate transaminase)

4. ≥ Grade 4 hematologic toxicity

5. ≥ Grade 3 non-hematologic toxicity

Change in High-sensitivity C-reactive Protein (hsCRP): Week 4From baseline (mean of screening and day 1) to week 4

Change from the baseline in hsCRP values to week 4 are presented.

Change in Serum Amyloid A (SAA): Week 4From baseline (mean of screening and day 1) to week 4

Change from the baseline in serum amyloid A (SAA) values to week 4 are presented.

Secondary Outcome Measures
NameTimeMethod
Change in Serum Pre-albumin: Mean of 10-12 WeeksFrom baseline (mean of screening and day 1), week 10, week 12

Change from baseline in serum pre-albumin to the mean of 10-12 weeks is presented.

Change in Albumin: Week 12From baseline (mean of screening and day 1) to week 12

Change from baseline in albumin to week 12 is presented.

Number of Treatment Emergent Adverse Events (TEAEs)Week 0-24

An AE (adverse event) is any undesirable event or any untoward medical occurrence that occurs to a participant during the course of a study, or the protocol-defined time after study termination, whether or not that event is considered Study Drug-related. A TEAE was defined as an AE that initiated or worsened on or after the date of first dose of study drug up to the end of study. Number of TEAEs from baseline until the end of safety follow up (week 24) were presented.

Electrocardiogram (ECG)At baseline, week 6, week 12, week 18 and week 24

A summary of the overall ECG interpretation and clinical significance from baseline until the end of the safety follow up period (week 24) is presented and categorized as Normal, Abnormal clinically significant (CS), Abnormal non clinically significant (NCS) and Missing.

Number of Participants Who Developed Anti-drug Antibodies (ADAs)Weeks 0-35

Number of participants who developed ADAs from baseline until the end of the extended follow up period (week 35) were reported. Samples with detectable ADAs were classified as positive for ADAs. Samples without detectable ADAs were classified as negative for ADAs.

Number of Participants With ADA TitersWeeks 0-35

Number of participants with ADA titers for ADA-positive samples from baseline to week 35 is presented.

Number of Adverse Events of Special InterestWeeks 0-24

Frequency of events of interest by treatment group and dose from baseline until the end of the safety follow-up period (week 24) were reported. Adverse events of special interest included severe infusion-related reactions, hypersensitivity reaction during study drug infusion, anaphylaxis and neutropenia events of grade 2 or higher (i.e., absolute neutrophil count \<1500/mm\^3 and decline by at least 25% from baseline).

Change in Transferrin Saturation (TSAT): Week 4From baseline (mean of screening and day 1) to week 4

Change from baseline in TSAT to week 4 is presented.

Number of Participants With Neutralizing ADAsFrom baseline (mean of screening and week 1) to week 35

Number of participants with neutralizing ADAs from baseline to week 35 are presented.

Change in High Sensitivity C-reactive Protein (Hs-CRP): Mean of 10-12 WeeksFrom baseline (mean of screening and day 1), week 10, week 12

Change from baseline in hs-CRP to the mean of 10-12 weeks is presented.

Change in SAA: Mean of Weeks 10-12From baseline (mean of screening and day 1), week 10, week 12

Change from baseline in SAA to the mean of weeks 10-12 is presented.

Change in TSAT: Mean of Weeks 10-12From baseline (mean of screening and day 1), week 10, week 12

Change from baseline in TSAT to the mean of weeks 10-12 is presented.

Change in Reticulocyte Hemoglobin (CHr): Week 4From baseline (mean of screening and day 1) to week 4

Change from baseline in CHr to week 4 is presented.

Change in Erythropoietin Resistance Index (ERI): Week 4From baseline (weekly mean of screening) to week 4

Change from baseline in ERI to week 4 is presented. ERI is defined as the epoetin alfa-equivalent dose (weight-based per week in U/kg) divided by the serum hemoglobin (in g/dL).

Change in ERI: Mean of Weeks 8-12From baseline (weekly mean of screening), week 8, week 12

Change from baseline in ERI to the mean of weeks 8-12 is presented. ERI is defined as the epoetin alfa-equivalent dose (weight-based per week in U/kg) divided by the serum hemoglobin (in g/dL).

Change in ERI: Mean of Weeks 10-12From baseline (weekly mean of screening), week 10, week 12

Change from baseline in ERI to the mean of weeks 10-12 is presented. ERI is defined as the epoetin alfa-equivalent dose (weight-based per week in U/kg) divided by the serum hemoglobin (in g/dL).

Change in CHr: Mean of Weeks 10-12From baseline (mean of screening and day 1), week 10, week 12

Change from baseline in CHr to the mean of weeks 10-12 is presented.

Change in Hemoglobin: Week 4From baseline (mean of screening and day 1) to week 4

Change from baseline in hemoglobin to week 4 is presented.

Change in Hemoglobin: Mean of Weeks 10-12From baseline (mean of screening and day 1), week 10, week 12

Change from baseline in hemoglobin to weeks 10-12 is presented.

Change in Hemoglobin: Mean of Weeks 10-12, Excluding Hemoglobin Values Following a Change in the Total Weekly ESA DoseFrom baseline (mean of screening and day 1), week 10, week 12

Change from baseline in hemoglobin to weeks 10-12, excluding hemoglobin values following a change in the total weekly ESA (erythropoiesis stimulating agent) dose is presented. A change is defined as the first time when the ESA weekly dose goes up by \>25% or down by \>25% relative to the previous week's dose.

Change in ERI: Week 12From baseline (weekly mean of screening) to week 12

Change from baseline in ERI to week 12 is presented. ERI is defined as the epoetin alfa-equivalent dose (weight-based per week in U/kg) divided by the serum hemoglobin (in g/dL).

Change in ERI: Mean of Weeks 9-12From baseline (weekly mean of screening), week 9, week 12

Change from baseline in ERI to the mean of weeks 9-12 is presented. ERI is defined as the epoetin alfa-equivalent dose (weight-based per week in U/kg) divided by the serum hemoglobin (in g/dL).

Change in Hemoglobin: Week 12From baseline (mean of screening and day 1) to week 12

Change from baseline in hemoglobin to week 12 is presented.

Change in Hemoglobin: Week 24From baseline (mean of screening and day 1) to week 24

Change from baseline in hemoglobin to week 24 is presented.

Change in Hemoglobin From Screening to Peak Hemoglobin: Week 4From screening to week 4

Change in hemoglobin from screening to peak hemoglobin at week 4 is presented.

Erythrocyte Mean Corpuscular Volume: Week 24At week 24

The observed values of erythrocyte mean corpuscular volume at week 24 are presented.

Basophils: Week 12At week 12

The observed values of basophils at week 12 are presented.

Basophil: Week 24At week 24

The observed values of basophils at week 24 are presented.

Erythrocytes: Week 12At week 12

The observed values of erythrocytes at week 12 are presented.

Erythrocytes: Week 24At week 24

The observed values of erythrocytes at week 24 are presented.

Hematocrit: Week 12At week 12

The observed values of hematocrit at week 12 are presented.

Hypochromatic Red Cells: Week 24At week 24

The observed values of hypochromatic red cells at week 24 are presented.

Basophils to Leukocytes Ratio: Week 12At week 12

Basophils to leukocytes ratio at week 12 is presented.

Basophils to Leukocytes Ratio: Week 24At week 24

Basophils to leukocytes ratio at week 24 is presented.

Eosinophils: Week 12At week 12

The observed values of eosinophils at week 12 are presented.

Eosinophils: Week 24At week 24

The observed values of eosinophils at week 24 are presented.

Eosinophils to Leukocytes Ratio: Week 12At week 12

Eosinophils to leukocytes ratio at week 12 is presented.

Hematocrit: Week 24At week 24

The observed values of hematocrit at week 24 are presented.

Hemoglobin: Week 12At week 12

The observed values of hemoglobin at week 12 are presented.

Hemoglobin: Week 24At week 24

The observed values of hemoglobin at week 24 are presented.

Eosinophils to Leukocytes Ratio: Week 24At week 24

The eosinophils to leukocytes ratio at week 24 is presented.

Erythrocyte Mean Corpuscular HGB Concentration: Week 24At week 24

The observed values of erythrocyte mean corpuscular HGB concentration at week 24 are presented.

Erythrocyte Mean Corpuscular Volume: Week 12At week 12

The observed values of erythrocyte mean corpuscular volume at week 12 are presented.

Erythrocyte Mean Corpuscular Hemoglobin: Week 12At week 12

The observed values of erythrocyte mean corpuscular hemoglobin at week 12 are presented.

Erythrocyte Mean Corpuscular Hemoglobin: Week 24At week 24

The observed values of erythrocyte mean corpuscular hemoglobin at week 24 are presented.

Erythrocyte Mean Corpuscular Hemoglobin (HGB) Concentration: Week 12At week 12

The observed values of erythrocyte mean corpuscular HGB concentration at week 12 are presented.

Hypochromatic Red Cells Week 12At week 12

The observed values of hypochromatic red cells at week 12 are presented.

Leukocytes: Week 12At week 12

The observed values of leukocytes at week 12 are presented.

Leukocytes: Week 24At week 24

The observed values of leukocytes at week 24 are presented.

Monocytes to Leukocytes Ratio: Week 24At week 24

Monocytes to leukocytes ratio at week 24 is presented.

Neutrophils: Week 12At week 12

The observed values of neutrophils at week 12 are presented.

Neutrophils: Week 24At week 24

The observed values of neutrophils at week 24 are presented.

Neutrophils to Leukocytes Ratio: Week 12At week 12

Neutrophils to leukocytes ratio at week 12 is presented.

Neutrophils to Leukocytes Ratio: Week 24At week 24

Neutrophils to leukocytes ratio at week 24 is presented.

Platelets: Week 12At week 12

The observed values of platelets at week 12 are presented.

Platelets: Week 24At week 24

The observed values of platelets at week 24 are presented.

Reticulocytes to Erythrocytes Ratio: Week 12At week 12

Reticulocytes to erythrocytes ratio at week 12 is presented.

Reticulocytes to Erythrocytes Ratio: Week 24At week 24

Reticulocytes to erythrocytes ratio at week 24 is presented.

Lymphocytes: Week 12At week 12

The observed values of lymphocytes at week 12 are presented.

Lymphocytes: Week 24At week 24

The observed values of lymphocytes at week 24 are presented.

Lymphocytes to Leukocytes Ratio: Week 12At week 12

Lymphocytes to leukocytes ratio at week 12 is presented.

Lymphocytes to Leukocytes Ratio at Week 24At week 24

Lymphocytes to leukocytes ratio at week 24 is presented.

Monocytes: Week 12At week 12

The observed values of monocytes at week 12 are presented.

Monocytes: Week 24At week 24

The observed values of monocytes at week 24 are presented.

Monocytes to Leukocytes Ratio: Week 12At week 12

Monocytes to leukocytes ratio at week 12 is presented.

Alanine Aminotransferase: Week 12At week 12

The observed values of alanine aminotransferase at week 12 are presented.

Alanine Aminotransferase: Week 24At week 24

The observed values of alanine aminotransferase at week 24 are presented.

Albumin: Week 12At week 12

The observed values of albumin at week 12 are presented.

Albumin: Week 24At week 24

The observed values of albumin at week 24 are presented.

Alkaline Phosphatase: Week 12At week 12

The observed values of alkaline phosphatase at week 12 are presented.

Alkaline Phosphatase: Week 24At week 24

The observed values of alkaline phosphatase at week 24 are presented.

Aspartate Aminotransferase: Week 12At week 12

The observed values of aspartate aminotransferase at week 12 are presented.

Aspartate Aminotransferase: Week 24At week 24

The observed values of aspartate aminotransferase at week 24 are presented.

Bicarbonate: Week 12At week 12

The observed values of bicarbonate at week 12 are presented.

Bicarbonate: Week 24At week 24

The observed values of bicarbonate at week 24 are presented.

Bilirubin: Week 12At week 12

The observed values of bilirubin at week 12 are presented.

Bilirubin: Week 24At week 24

The observed values of bilirubin at week 24 are presented.

Calcium: Week 12At week 12

The observed values of calcium at week 12 are presented.

Calcium: Week 24At week 24

The observed values of calcium at week 24 are presented.

Chloride: Week 12At week 12

The observed values of chloride at week 12 are presented.

Chloride: Week 24At week 24

The observed values of chloride at week 24 are presented.

Cholesterol: Week 12At week 12

The observed values of cholesterol at week 12 are presented.

Cholesterol: Week 24At week 24

The observed values of cholesterol at week 24 are presented.

Creatinine: Week 12At week 12

The observed values of creatinine at week 12 are presented.

Creatinine: Week 24At week 24

The observed values of creatinine at week 24 are presented.

Direct Bilirubin: Week 12At week 12

The observed values of direct bilirubin at week 12 are presented.

Direct Bilirubin: Week 24At week 24

The observed values of direct bilirubin at week 24 are presented.

Glucose: Week 12At week 12

The observed values of glucose at week 12 are presented.

Glucose: Week 24At week 24

The observed values of glucose at week 24 are presented.

High-density Lipoprotein (HDL) Cholesterol: Week 12At week 12

The observed values of HDL cholesterol at week 12 are presented.

HDL Cholesterol: Week 24At week 24

The observed values of HDL cholesterol at week 24 are presented.

Hepcidin-25: Week 12At week 12

The observed values of hepcidin-25 at week 12 are presented.

Hepcidin-25: Week 24At week 24

The observed values of hepcidin-25 at week 24 are presented.

Low-density Lipoproteins (LDL) Cholesterol: Week 12At week 12

The observed values of LDL cholesterol at week 12 are presented.

LDL Cholesterol: Week 24At week 24

The observed values of LDL cholesterol at week 24 are presented.

Lipoprotein-a: Week 12At week 12

The observed values of lipoprotein-a at week 12 are presented.

Lipoprotein-a: Week 24At week 24

The observed values of lipoprotein-a at week 24 are presented.

Phosphate: Week 12At week 12

The observed values of phosphate at week 12 are presented.

Phosphate: Week 24At week 24

The observed values of phosphate at week 24 are presented.

Potassium: Week 12At week 12

The observed values of potassium at week 12 are presented.

Potassium: Week 24At week 24

The observed values of potassium at week 24 are presented.

Sodium: Week 12At week 12

The observed values of sodium at week 12 are presented.

Sodium: Week 24At week 24

The observed values of sodium at week 24 are presented.

Triglycerides: Week 12At week 12

The observed values of triglyceride at week 12 are presented.

Pre-infusion Systolic Blood Pressure: Week 11At week 11

Pre-infusion systolic blood pressure values at week 11 are presented.

Triglycerides: Week 24At week 24

The observed values of triglycerides at week 24 are presented.

Urea Nitrogen: Week 12At week 12

The observed values of urea nitrogen at week 12 are presented.

Urea Nitrogen: Week 24At week 24

The observed values of urea nitrogen at week 24 are presented.

Pre-dialysis Body Mass Index (BMI): Week 12At week 12

Pre-dialysis BMI values at week 12 are presented.

Pre-infusion BMI: Week 11At week 11

Pre-infusion BMI values at week 11 are presented.

Pre-dialysis Diastolic Blood Pressure: Week 12At week 12

Pre-dialysis diastolic blood pressure values at week 12 are presented.

Pre-dialysis Diastolic Blood Pressure: Week 24At week 24

Pre-dialysis diastolic blood pressure values at week 24 are presented.

Pre-infusion Diastolic Blood Pressure: Week 11At week 11

Pre-infusion diastolic blood pressure values at week 11 are presented.

Pre-dialysis Heart Rate: Week 12At week 12

Pre-dialysis heart rate values at week 12 are presented.

Pre-dialysis Systolic Blood Pressure: Week 24At week 24

Pre-dialysis values of systolic blood pressure at week 24 are presented.

Pre-dialysis Weight: Week 12At week 12

Pre-dialysis weight values at week 12 are presented.

Pre-dialysis Heart Rate: Week 24At week 24

Pre-dialysis heart rate values at week 24 are presented.

Pre-infusion Heart Rate: Week 11At week 11

Pre-infusion heart rate values at week 11 are presented.

Pre-dialysis Respiration Rate: Week 12At week 12

Pre-dialysis respiration rate values at week 12 are presented.

Pre-dialysis Respiration Rate: Week 24At week 24

Pre-dialysis respiration rate values at week 24 are presented.

Pre-infusion Respiration Rate: Week 11At week 11

Pre-infusion respiration rate values at week 11 are presented.

Pre-dialysis Systolic Blood Pressure: Week 12At week 12

Pre-dialysis values of systolic blood pressure at week 12 are presented.

Pre-infusion Weight: Week 11At week 11

Pre-infusion weight values at week 11 are presented.

Pre-dialysis Temperature: Week 12At week 12

Pre-dialysis temperature values at week 12 are presented.

Pre-dialysis Temperature: Week 24At week 24

Pre-dialysis temperature values at week 24 are presented.

Pre-infusion Temperature: Week 11At week 11

Pre-infusion temperature values at week 11 are presented.

Area Under the Serum Concentration Time Curve From Time 0 to Infinity (AUC 0-α) of COR-001Week 1 (day 1, 3, and 5), Week 2 (day 8), Week 3 (day 15), Week 5 (day 29), Week 7 (day 43), Week 8 (day 50), Week 9 (day 57), Week 11 (day 71 and 75), Week 14 (day 92), Week 18 (day 120) and Week 35 (day 239).

Area under the serum concentration time curve from time 0 to infinity (AUC 0-α) of COR-001 is presented.

Elimination Half-life in the Initial Phase (t 1/2,α)Week 1 (day 1, 3, and 5), Week 2 (day 8), Week 3 (day 15), Week 5 (day 29), Week 7 (day 43), Week 8 (day 50), Week 9 (day 57), Week 11 (day 71 and 75), Week 14 (day 92), Week 18 (day 120) and Week 35 (day 239).

Elimination half-life in the initial phase (t 1/2,α) is presented from week 0 to week 35.

Elimination Half-life in the Terminal Phase (t 1/2, z)Week 1 (day 1, 3, and 5), Week 2 (day 8), Week 3 (day 15), Week 5 (day 29), Week 7 (day 43), Week 8 (day 50), Week 9 (day 57), Week 11 (day 71 and 75), Week 14 (day 92), Week 18 (day 120) and Week 35 (day 239).

Elimination half-life in the terminal phase(t 1/2, z) is presented from week 0 to week 35.

Maximum Serum Concentration (Cmax)Week 1 (day 1, 3, and 5), Week 2 (day 8), Week 3 (day 15), Week 5 (day 29), Week 7 (day 43), Week 8 (day 50), Week 9 (day 57), Week 11 (day 71 and 75), Week 14 (day 92), Week 18 (day 120) and Week 35 (day 239).

Maximum serum concentration (Cmax) of COR-001 from week 0 to week 35 is presented.

Impact of ADAs on PharmacokineticsWeek 1 (day 1, 3, and 5), Week 2 (day 8), Week 3 (day 15), Week 5 (day 29), Week 7 (day 43), Week 8 (day 50), Week 9 (day 57), Week 11 (day 71 and 75), Week 14 (day 92), Week 18 (day 120) and Week 35 (day 239).

Presence of ADA was a covariate for pharmacokinetics (PK) affecting V1 (volume of distribution for the central compartment). Impact of ADAs on V1 from baseline to week 35 is presented. In the below table, result presented is the bootstrap parameter estimate of the effect of ADA on V1 for a PK model that includes data for all arms combined.

Trial Locations

Locations (1)

Novo Nordisk Investigational Site

🇺🇸

San Antonio, Texas, United States

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