Hereditary angioedema(HAE)
Conditions
Interventions
Donidalorsen 40 mg, 80 mg, or placebo will be administered subcutaneously on the upper arm once every four weeks for a total of four times.
Sponsors
Tsunoda Takeshi
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1) Body mass index (BMI) of >=18.5 kg/m2 to <25.0 kg/m2 at screening 2)Must have a systolic blood pressure between 90 to 140 mmHg, inclusive, diastolic blood pressure between 50 to 90 mmHg, inclusive, and heart rate between 50 to 100 bpm, inclusive, at Screening
Exclusion criteria
Exclusion criteria: 1) Clinically-significant abnormalities in medical history including acute coronary syndrome, major surgery within 6 months of Screening, planned surgery that would occur during the study or physical examination or other screening results such as ECGs at Screening 2) Clinically-significant abnormalities in the following screening laboratory values including the following: - Urine protein/creatinine (P/C) ratio >= 0.2 mg/mg - Alanine aminotransferase, aspartate aminotransferase, bilirubin, alkaline phosphatase, serum creatinine, or blood urea nitrogen >upper limit of normal - Glycated hemoglobin >=6.5% - Platelet count < 140,000/uL 3) Body weight of <41 kg at Screening 4) Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to the first day Study Drug product is administered to the subject (Day 1) 5) Known history or positive test for human immunodeficiency virus (HIV), hepatitis C, chronic hepatitis B, or syphilis 6) Malignancy within 5 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated 7) History of bleeding diathesis or coagulopathy
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Pharmacokinetic Endpoints: -plasma donidalorsen concentration ,plasma PK parameter ,and cumulative parameter Pharmacodynamic Endpoint: -plasma PKK concentration Immunogenicity Endpoint: -plasma ADA measurement result Safety Endpoints: -Body weight -Physical examination findings -Vital signs (blood pressure, pulse rate ,body temperature) -12-Lead ECG -Clinical laboratory tests (hematology ,blood chemistry ,urinalysis) -Inflammatory (Hs-CRP) -Complement (C5a ,Bb) -Coagulation (PT ,INR ,APTT) -AEs | — |
Contacts
Public ContactDrug Information Center
Otsuka Pharmaceutical Co., LTD.
Outcome results
None listed