UMIN-CTR Clinical Trial

BACK TOP
UMIN-CTR English Home Glossary (Simple) FAQ Search clinical trials

Name:
UMIN ID:

Recruitment status Open public recruiting
Unique ID issued by UMIN UMIN000034869
Receipt No. R000039604
Scientific Title A Phase 3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of two dose levels of BCX7353 as an oral treatment for the prevention of attacks in subjects with hereditary angioedema
Date of disclosure of the study information 2018/11/13
Last modified on 2019/05/14

* This page includes information on clinical trials registered in UMIN clinical trial registed system.
* We don't aim to advertise certain products or treatments


Basic information
Public title A Phase 3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of two dose levels of BCX7353 as an oral treatment for the prevention of attacks in subjects with hereditary angioedema
Acronym Evaluate study between two doses levels of BCX7353 and placebo for prevention of attacks in subjects with hereditary angioedema
Scientific Title A Phase 3, randomized, double-blind, placebo-controlled, parallel-group study to evaluate the efficacy and safety of two dose levels of BCX7353 as an oral treatment for the prevention of attacks in subjects with hereditary angioedema
Scientific Title:Acronym Evaluate study between two doses levels of BCX7353 and placebo for prevention of attacks in subjects with hereditary angioedema
Region
Japan

Condition
Condition Hereditary angioedema
Classification by specialty
Medicine in general Gastroenterology Clinical immunology
Dermatology Oto-rhino-laryngology Emergency medicine
Classification by malignancy Others
Genomic information YES

Objectives
Narrative objectives1 To determine the efficacy of BCX7353 110 and 150 mg administered once daily (QD) for 24 weeks compared to placebo in the prevention of angioedema events in subjects with hereditary angioedema (HAE)
Basic objectives2 Safety,Efficacy
Basic objectives -Others
Trial characteristics_1 Confirmatory
Trial characteristics_2 Pragmatic
Developmental phase Phase III

Assessment
Primary outcomes The rate of expert-confirmed angioedema events during dosing in the entire 24 week treatment period (Days 1 to 168)
Key secondary outcomes -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)

Base
Study type Interventional

Study design
Basic design Parallel
Randomization Randomized
Randomization unit Individual
Blinding Double blind -all involved are blinded
Control Placebo
Stratification YES
Dynamic allocation YES
Institution consideration Institution is not considered as adjustment factor.
Blocking YES
Concealment Central registration

Intervention
No. of arms 3
Purpose of intervention Prevention
Type of intervention
Medicine
Interventions/Control_1 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:BCX7353 capsules,to be administered orally for 28 weeks.
Interventions/Control_2 Drug:BCX7353 capsules, to be administered orally
-Treatment Group 2 (150 mg QD) Parts 1 and 2: two 75 mg capsules of BCX7353
-Part1:BCX7353 capsules,to be administered orally for 24 weeks.
-Part2:BCX7353 capsules,to be administered orally for 28 weeks.
Interventions/Control_3 Drug:BCX7353 capsules, to be administered orally
-Treatment Group 3a (110 mg QD) Part 2: two 55 mg capsules of BCX7353/ Group 3b (150 mg QD) Part 2: two 75 mg capsules of BCX7353
-Part1:two Placebo capsules,to be administered orally for 24 weeks.
-Part2:BCX7353 capsules,to be administered orally for 28 weeks.
Interventions/Control_4
Interventions/Control_5
Interventions/Control_6
Interventions/Control_7
Interventions/Control_8
Interventions/Control_9
Interventions/Control_10

Eligibility
Age-lower limit
12 years-old <=
Age-upper limit

Not applicable
Gender Male and Female
Key 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.
Key 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
Target sample size 24

Research contact person
Name of lead principal investigator
1st name Sylvia Dobo, MD
Middle name
Last name Sylvia Dobo, MD
Organization BioCryst Pharmaceuticals, Inc.
Division name BioCryst Medical Monitoring team
Zip code 27703
Address 4505 Emperor Boulevard, Suite 200 Durham, NC 27703, USA
TEL +1-919-859-7905
Email mmj@biocryst.com

Public contact
Name of contact person
1st name Miki
Middle name
Last name Kikuchi
Organization Integrated Development Associates Co., Ltd.
Division name Department of Clinical Operations
Zip code 106-6006
Address Kasumigaseki Building 6F, 3-2-5 Kasumigaseki, Chiyoda-ku, Tokyo
TEL 03-6811-2332
Homepage URL
Email miki.kikuchi@i-d-a.com

Sponsor
Institute BioCryst Pharmaceuticals, Inc.
Institute
Department

Funding Source
Organization BioCryst Pharmaceuticals, Inc.
Organization
Division
Category of Funding Organization Outside Japan
Nationality of Funding Organization Unites States of America

Other related organizations
Co-sponsor
Name of secondary funder(s)

IRB Contact (For public release)
Organization Shimane University Hospital Institutional Review Board
Address 89-1, Enya-cho, Izumo-shi, Shimane
Tel 0853-23-2111
Email tiken@med.shimane-u.ac.jp

Secondary IDs
Secondary IDs NO
Study ID_1
Org. issuing International ID_1
Study ID_2
Org. issuing International ID_2
IND to MHLW

Institutions
Institutions

Other administrative information
Date of disclosure of the study information
2018 Year 11 Month 13 Day

Related information
URL releasing protocol
Publication of results Unpublished

Result
URL related to results and publications
Number of participants that the trial has enrolled
Results
Results date posted
Results Delayed
Results Delay Reason
Date of the first journal publication of results
Baseline Characteristics
Participant flow
Adverse events
Outcome measures
Plan to share IPD
IPD sharing Plan description

Progress
Recruitment status Open public recruiting
Date of protocol fixation
2018 Year 10 Month 22 Day
Date of IRB
2018 Year 11 Month 14 Day
Anticipated trial start date
2018 Year 12 Month 01 Day
Last follow-up date
2020 Year 06 Month 30 Day
Date of closure to data entry
Date trial data considered complete
Date analysis concluded

Other
Other related information

Management information
Registered date
2018 Year 11 Month 12 Day
Last modified on
2019 Year 05 Month 14 Day


Link to view the page
URL(English) https://upload.umin.ac.jp/cgi-open-bin/ctr_e/ctr_view.cgi?recptno=R000039604

Research Plan
Registered date File name

Research case data specifications
Registered date File name

Research case data
Registered date File name


Contact us.