Hereditary angioedema
Conditions
Interventions
Drug:BCX7353 capsules, to be administered orally -Treatment Group 1 (110 mg QD) Parts 1 and 2: two 55 mg capsules of BCX7353 -Part1:BCX7353 capsules,to be administered orally for 24 weeks. -Part2:B
Sponsors
BioCryst Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: -Males and non-pregnant, non-lactating females more than 12 years of age. -Able to provide written, informed consent. Subjects aged 12 to 17 years must be able to read, understand, and be willing to sign an assent form in addition to a caregiver providing informed consent. -A clinical diagnosis of HAE Type 1 or Type 2, defined as having a C1 esterase inhibitor (C1 INH) functional level less than 50% and a complement 4 (C4) level below the lower limit of the normal (LLN) reference range, as assessed during the screening period. -Access to and ability to use an acute treatment for angioedema events approved by the Japan Ministry of Health, Labor, and Welfare (plasma-derived C1 INH or icatibant). -Subjects must be medically appropriate for on-demand treatment as the sole medicinal management for their HAE during the study, that is, subjects must be medically appropriate to be managed without prophylactic treatments for HAE. -In the opinion of the investigator, the subject is expected to adequately comply with all required study procedures for the duration of the study. The subject must demonstrate adequate compliance with all study procedures required from the screening visit through randomization, including e-diary recording of angioedema events beginning at the screening visit.
Exclusion criteria
Exclusion criteria: -Any clinically significant medical or psychiatric condition or medical history that would interfere with the subject's ability to participate in the study or increases the risk to the subject by participating in the study -Clinically significant abnormal ECG at the screening visit -Any clinically significant history of angina, myocardial infarction etc. or any other clinically significant cardiovascular abnormality such as poorly controlled hypertension -Known family history of sudden cardiac death. Family history of sudden death from HAE is not exclusionary -Any abnormal laboratory or urinalysis parameter at screening that is clinically significant and relevant for this study -Suspected C1-INH resistance -Pregnant or planning to become pregnant during the study -Currently breastfeeding -Use of androgens or tranexamic acid for prophylaxis of angioedema events within the 28 days prior to the screening visit or initiation during the study -Use of C1-INH for prophylaxis of angioedema events within the 14 days prior to the screening visit or initiation during the study -Use of concomitant medications that are metabolized by CYP2D6, CYP2C9, CYP2C19, and CYP3A4 and have a narrow therapeutic range, within 7 days of the baseline visit or planned initiation during the study -Use of a medication that is clinically known to prolong the QT interval and is metabolized by CYP2D6, CYP2C9, CYP2C19, and/or CYP3A4 7 days prior to the baseline visit or planned initiation during the study -Use of an angiotensin-converting enzyme inhibitor and a medication that is transported by P-glycoprotein and has a narrow therapeutic range, within 7 days of the baseline visit or planned initiation during the study -Initiation of an estrogen-containing hormonal contraceptive within 56 days of the screening visit or planned initiation during the study -Current participation in any other investigational drug study or received another investigational drug within 30 days of the screening visit
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| The rate of expert-confirmed angioedema events during dosing in the entire 24 week treatment period (Days 1 to 168) | — |
Secondary
| Measure | Time frame |
|---|---|
| -Change from baseline in Angioedema Quality of Life questionnaire (AE QoL) at Week 24 (total score) -Number and proportion of days with angioedema symptoms through 24 weeks -Rate of expert-confirmed angioedema events during dosing in the effective treatment period (beginning on Day 8 through 24 weeks) | — |
Countries
Japan
Contacts
Public ContactMasayuki Ono
Covance Japan Co., Ltd. Department of Clinical Operations
Outcome results
None listed