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Clinical Trials/NCT03380780
NCT03380780CompletedPhase 1

A Single-Center, Open-Label, Single Dose Study to Investigate the Pharmacokinetics, Safety, and Tolerability of Emicizumab in Healthy Chinese Volunteers

Hoffmann-La Roche1 site in 1 country16 target enrollmentStarted: March 12, 2018Last updated:
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Conditions
Interventions
Drugs

Trial Snapshot

Phase
Phase 1
Status
Completed
Enrollment
16
Locations
1
Primary Endpoint
Maximum Observed Plasma Concentration (Cmax) of Emicizumab
Study OverviewStudy DesignEligibility CriteriaArms & InterventionsOutcomesInvestigatorsSitesRecords & IdentifiersSimilar Trials

Study Overview

Brief Summary

This single-center, open-label study will evaluate the pharmacokinetics, safety, and tolerability of emicizumab following a single subcutaneous (SC) administration to healthy Chinese subjects.

Study Design

Study Type
Interventional
Allocation
Na
Intervention Model
Single Group
Primary Purpose
Treatment
Masking
None

Eligibility Criteria

Ages
20 Years to 45 Years (Adult)
Sex
Male
Accepts Healthy Volunteers
Yes

Inclusion Criteria

  • •Healthy Chinese male subjects, aged 20-45 years inclusive at the time of screening
  • •Chinese subjects must have Chinese parents and grandparents, all of whom were born in China
  • •A body mass index (BMI) between 19 and 24 kilograms per height in meters squared (kg/m^2), inclusive
  • •Able to participate and willing to give written informed consent and to comply with the study requirements

Exclusion Criteria

  • •Any history or presence of a clinically significant disorder, or any other condition or disease which in the judgement of the investigator would place the subject at undue risk; interfere with absorption, distribution, metabolism, and excretion of emicizumab; or interfere with the ability of the subject to complete the study
  • •Major illness within 1 month prior to dosing, and/or any condition which could relapse during or immediately after the study
  • •Use of any prescribed or over-the-counter (OTC) medication or herbal medicine taken within 14 days prior to dosing or within 5 times the elimination half-life of the medication prior to dosing (whichever is longer), with some exceptions
  • •Any significant donation/loss of blood or plasma (greater than 450 milliliters) within the 3 months prior to dosing
  • •Regular smoker with consumption of more than 10 cigarettes per day or the equivalent amount of tobacco
  • •Participation within a clinical study with an investigational drug or device within the last 3 months prior to dosing
  • •Any clinically relevant history of hypersensitivity or allergic reactions, either spontaneous or following drug administration or exposure to foods or environmental agents
  • •Previous or concomitant thromboembolic disease such as deep vein thrombosis (DVT) or signs of thromboembolic disease, or family history of thromboembolic disorder such as serious DVT
  • •At high risk for thrombotic microangiopathy (e.g., have a previous medical or family history of thrombotic microangiopathy), in the investigator's judgment
  • •Previous or concomitant autoimmune or connective tissue disease
  • •History of tuberculosis or active tuberculosis with positive test result at screening
  • •Any other reason that, in the judgment of the investigator, would render the subject unsuitable for study participation

Arms & Interventions

Emicizumab

Experimental

Intervention: Emicizumab (Drug)

Outcomes

Primary Outcomes

Maximum Observed Plasma Concentration (Cmax) of Emicizumab

Time Frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

Area Under the Plasma Concentration Versus Time Curve (AUC) Between Time Zero Extrapolated to Infinity (AUC0-inf) of Emicizumab

Time Frame: Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113

Plasma concentrations of emicizumab were analyzed using a validated enzyme-linked immunosorbent assay (ELISA). Pharmacokinetics (PK) parameters were estimated using standard non-compartmental methods.

Secondary Outcomes

  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Monocytes, Absolute Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Total Neutrophils, Absolute Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Number of Participants by Test Results for Blood in Urine by Timepoint(Baseline and Days 2, 8, 29, 57, 85, and 113)
  • Change From Baseline in Vital Signs by Timepoint: Diastolic Blood Pressure(Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Platelet Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: QTcB Duration (Bazett's Correction Formula)(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Number of Participants With Concomitant Medications(From screening to study completion (20 weeks))
  • Change From Baseline in Vital Signs by Timepoint: Pulse Rate(Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Vital Signs by Timepoint: Respiratory Rate(Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Vital Signs by Timepoint: Systolic Blood Pressure(Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Vital Signs by Timepoint: Temperature (Axillary)(Baseline and Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Electrocardiogram (ECG) Results by Timepoint: Heart Rate(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: PR Duration(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: QRS Duration(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: QT Duration(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • AUC Between Time Zero and the Time of Last Quantifiable Concentration (AUC0-last) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Time to Cmax (Tmax) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Apparent Clearance (CL/F) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Apparent Volume of Distribution (Vz/F) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Number of Participants Testing Negative or Positive for the Presence of Anti-Drug Antibodies (ADA) Against Emicizumab by Timepoint and for the Overall Study(Predose at Baseline (Day 1) and postdose on Days 57 and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: C-Reactive Protein Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: QTcF Duration (Fridericia's Correction Formula)(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in ECG Results by Timepoint: RR Duration(Baseline and Days 2, 4, 6, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Apparent Terminal Half-Life (t1/2) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Mean Residence Time (MRT) of Emicizumab(Predose on Day 1 and postdose on Days 2, 4, 6, 8, 11, 15, 22, 29, 36, 43, 50, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Bilirubin Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Blood Glucose Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Blood Urea Nitrogen Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Cholesterol Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time(Baseline and Days 2, 11, 29, 57 and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Eosinophils, Absolute Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Number of Participants With Laboratory Test Abnormalities(Baseline and Days 2, 4, 8, 11, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Albumin Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatine Kinase Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Potassium Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Fasting Triglycerides Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Uric Acid Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Alkaline Phosphatase Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Chloride Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Gamma Glutamyl Transferase Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Total Protein Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGOT/AST Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: SGPT/ALT Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Basophils, Absolute Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Lymphocytes, Absolute Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Creatinine Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Lactate Dehydrogenase Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Sodium Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Prothrombin Time/International Normalized Ratio (INR)(Baseline and Days 2, 11, 29, 57 and 113)
  • Number of Participants by Test Results for Glucose in Urine by Timepoint(Baseline and Days 2, 8, 29, 57, 85, and 113)
  • Number of Participants by Test Results for Protein in Urine by Timepoint(Baseline and Days 2, 8, 29, 57, 85, and 113)
  • Change From Baseline in Clinical Chemistry Laboratory Test Results by Timepoint: Direct Bilirubin Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Volume(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hematocrit (as a Fraction of 1)(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Red Blood Cell Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: White Blood Cell Count(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Number of Participants With Adverse Events by Highest World Health Organization (WHO) Toxicity Grade(From screening to study completion (20 weeks))
  • Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Activated Partial Thromboplastin Time(Baseline and Days 2, 11, 29, 57, and 113)
  • Change From Baseline in Coagulation Laboratory Test Results by Timepoint: Fibrinogen Concentration(Baseline and Days 2, 11, 29, 57 and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Erythrocyte Mean Corpuscular Hemoglobin Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)
  • Change From Baseline in Hematology Laboratory Test Results by Timepoint: Hemoglobin Concentration(Baseline and Days 2, 4, 8, 15, 29, 43, 57, 71, 85, and 113)

Investigators

Sponsor Class
Industry
Responsible Party
Sponsor

Study Sites (1)

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