Flag of the European Union EU Clinical Trials Register Help

Clinical trials

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   43861   clinical trials with a EudraCT protocol, of which   7284   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     

    < Back to search results

    Download PDF

    Clinical Trial Results:
    A randomized, double-blind, placebo-controlled, dose-ranging, parallel-group study to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics of BCX7353 as a preventative treatment to reduce the frequency of attacks in subjects with hereditary angioedema

    Summary
    EudraCT number
    		 2016-001272-29
    Trial protocol
    		 GB   DE   HU   ES   AT   DK   GR  
    Global end of trial date
    08 Aug 2017

    Results information
    Results version number
    		 v1(current)
    This version publication date
    07 Nov 2020
    First version publication date
    07 Nov 2020
    Other versions

    Trial information

    		 Close Top of page
    Trial identification
    Sponsor protocol code
    BCX7353-203
    Additional study identifiers
    ISRCTN number
    		 -
    US NCT number
    		 NCT02870972
    WHO universal trial number (UTN)
    		 -
    Other trial identifiers
    		 205417: IRAS
    Sponsors
    Sponsor organisation name
    BioCryst Pharmaceuticals Inc
    Sponsor organisation address
    4505 Emperor Blvd., Suite 200, Durham, United States, 27703
    Public contact
    Study Director, BioCryst Pharmaceuticals Inc, +1 919-859-1302, clinicaltrials@biocryst.com
    Scientific contact
    Study Director, BioCryst Pharmaceuticals Inc, +1 919-859-1302, clinicaltrials@biocryst.com
    Paediatric regulatory details
    Is trial part of an agreed paediatric investigation plan (PIP)
    No
    Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial?
    No
    Results analysis stage
    Analysis stage
    Final
    Date of interim/final analysis
    30 Jul 2019
    Is this the analysis of the primary completion data?
    Yes
    Primary completion date
    08 Aug 2017
    Global end of trial reached?
    Yes
    Global end of trial date
    08 Aug 2017
    Was the trial ended prematurely?
    No
    General information about the trial
    Main objective of the trial
    To evaluate the efficacy of once-daily prophylactic berotralstat at up to 5 dose levels, as measured by the number of attacks of hereditary angioedema (HAE) observed in patients with HAE enrolled in each treatment group.
    Protection of trial subjects
    This trial was designed and monitored in accordance with sponsor procedures, which comply with the ethical principles of Good Clinical Practice (GCP) as required by the major regulatory authorities, and in accordance with the Declaration of Helsinki. A signed ICF was obtained from each subject before performing any study-related procedures; subjects were not screened or treated until the subject had signed an approved ICF in a language in which the subject was fluent. Each subject was given both verbal and written information describing the nature and duration of the clinical study. The investigator or designee explained to potential subjects the aims, methods, objectives, reasonably anticipated benefits, and potential hazards of the study prior to performing any study-related procedures. Subjects were informed that they were free not to participate in the trial and that they may have withdrawn consent to participate at any time. Before entry into the trial, consent was recorded by means of the subject’s dated signature. The subject received a copy of the signed and dated ICF, and the original signed ICF was retained in the study files. The protocol, informed consent form (ICF), and other relevant study documentation were submitted by each investigator to an appropriate independent ethics committee (IEC)/institutional review board (IRB) for review and approval before study initiation.
    Background therapy
    		 -
    Evidence for comparator
    		 -
    Actual start date of recruitment
    11 Aug 2016
    Long term follow-up planned
    No
    Independent data monitoring committee (IDMC) involvement?
    Yes
    Population of trial subjects
    Number of subjects enrolled per country
    Country: Number of subjects enrolled
    Australia: 3
    Country: Number of subjects enrolled
    Macedonia, the former Yugoslav Republic of: 9
    Country: Number of subjects enrolled
    Switzerland: 5
    Country: Number of subjects enrolled
    Spain: 6
    Country: Number of subjects enrolled
    United Kingdom: 11
    Country: Number of subjects enrolled
    Austria: 6
    Country: Number of subjects enrolled
    Denmark: 8
    Country: Number of subjects enrolled
    France: 5
    Country: Number of subjects enrolled
    Germany: 12
    Country: Number of subjects enrolled
    Hungary: 3
    Country: Number of subjects enrolled
    Italy: 7
    Worldwide total number of subjects
    75
    EEA total number of subjects
    58
    Number of subjects enrolled per age group
    In utero
    0
    Preterm newborn - gestational age < 37 wk
    0
    Newborns (0-27 days)
    0
    Infants and toddlers (28 days-23 months)
    0
    Children (2-11 years)
    0
    Adolescents (12-17 years)
    0
    Adults (18-64 years)
    75
    From 65 to 84 years
    0
    85 years and over
    0

    Subject disposition

    		 Close Top of page
    Recruitment
    Recruitment details
    		 -

    Pre-assignment
    Screening details
    		 HAE subjects with a documented recent history of frequent angioedema attacks were evaluated for participation in this study at a screening visit, which occurred within 21 days of Day 1.

    Period 1
    Period 1 title
    Parallel-group dose response study (overall period)
    Is this the baseline period?
    		 Yes
    Allocation method
    Randomised - controlled
    Blinding used
    		 Double blind
    Roles blinded
    		 Subject, Investigator, Monitor, Data analyst, Carer, Assessor
    Blinding implementation details
    In order to make informed decisions about the conduct of the study, sponsor employees were unblinded at the time that the administrative interim data packages were available for review for each interim analysis.

    Arms
    Are arms mutually exclusive
    Yes

    Arm title
    		 Placebo
    Arm description
    		 • Part 1: 36 Subjects were randomized in a 1:1 ratio to placebo:berotralstat • Part 2: 15 Subjects were randomized in a 1:3:3 ratio to placebo:berotralstat:berotralstat, with 2 different doses of berotralstat. • Part 3: 24 Subjects were randomized in a 1:3:3:3 ratio to placebo:berotralstat:berotralstat:berotralstat, with 3 different doses of berotralstat.
    Arm type
    		 Placebo

    Investigational medicinal product name
    Placebo
    Investigational medicinal product code
    Other name
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The reference in this study was placebo to match berotralstat capsules. Capsules were administered orally QD for 28 days.

    Arm title
    		 berotralstat
    Arm description
    		 • Part 1: 36 Subjects were randomized in a 1:1 ratio of placebo:berotralstat • Part 2: 15 Subjects were randomized in a 1:3:3 ratio of placebo:berotralstat (125mg):berotralstat (250mg). • Part 3: 24 Subjects were randomized in a 1:3:3:3 ratio of placebo:berotralstat (125mg):berotralstat (250mg):berotralstat (62.5mg).
    Arm type
    		 Experimental

    Investigational medicinal product name
    berotralstat
    Investigational medicinal product code
    Other name
    BCX7353
    Pharmaceutical forms
    Capsule
    Routes of administration
    Oral use
    Dosage and administration details
    The test drug in this study was berotralstat powder in capsules that were administered orally QD for 28 days.

    Number of subjects in period 1
    		Placebo berotralstat
    Started
    22
    53
    Completed
    22
    52
    Not completed
    0
    1
         Adverse event, non-fatal
    -
    1

    Baseline characteristics

    		 Close Top of page
    Baseline characteristics reporting groups
    Reporting group title
    Parallel-group dose response study
    Reporting group description
    		 -

    Reporting group values
    		 Parallel-group dose response study Total
    Number of subjects
    		 75 75
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    		 75 75
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    		 44.5 ( 12.5 ) -
    Gender categorical
    Units: Subjects
        Female
    		 46 46
        Male
    		 29 29
    Race
    Units: Subjects
        American Indian or Alaska Native
    		 0 0
        Asian
    		 2 2
        Black or African American
    		 0 0
        Native Hawaiian or other Pacific Islander
    		 0 0
        White
    		 68 68
        Other
    		 5 5
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    		 6 6
        Not Hispanic or Latino
    		 61 61
        Not Reported
    		 5 5
        Unknown
    		 3 3
    Weight
    Units: kilogram(s)
        arithmetic mean (standard deviation)
    		 78.13 ( 17.98 ) -
    Height
    Units: centimetres
        arithmetic mean (standard deviation)
    		 168.61 ( 9.72 ) -
    Body Mass Index
    Units: kilograms/metre
        arithmetic mean (standard deviation)
    		 27.44 ( 5.58 ) -
    Subject analysis sets

    Subject analysis set title
    berotralstat 350 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (350 mg) administerd orally QD for 28 days.

    Subject analysis set title
    berotralstat 250 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (250 mg) administerd QD for 28 days.

    Subject analysis set title
    berotralstat 125 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (125 mg) administerd QD for 28 days.

    Subject analysis set title
    berotralstat 62.5 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (62.5 mg) administerd QD for 28 days.

    Subject analysis sets values
    		 berotralstat 350 mg berotralstat 250 mg berotralstat 125 mg berotralstat 62.5 mg
    Number of subjects
    18
    14
    14
    7
    Age categorical
    Units: Subjects
        Adults (18-64 years)
    18
    14
    14
    7
    Age continuous
    Units: years
        arithmetic mean (standard deviation)
    43.8 ( 11.6 )
    40.9 ( 13.4 )
    48.1 ( 12.6 )
    38.9 ( 16.6 )
    Gender categorical
    Units: Subjects
        Female
    11
    6
    10
    6
        Male
    7
    8
    4
    1
    Race
    Units: Subjects
        American Indian or Alaska Native
    0
    0
    0
    0
        Asian
    1
    1
    0
    0
        Black or African American
    0
    0
    0
    0
        Native Hawaiian or other Pacific Islander
    0
    0
    0
    0
        White
    15
    12
    14
    7
        Other
    2
    1
    0
    0
    Ethnicity
    Units: Subjects
        Hispanic or Latino
    1
    1
    0
    1
        Not Hispanic or Latino
    14
    10
    14
    5
        Not Reported
    0
    3
    0
    1
        Unknown
    3
    0
    0
    0
    Weight
    Units: kilogram(s)
        arithmetic mean (standard deviation)
    80.13 ( 15.83 )
    76.88 ( 13.57 )
    84.28 ( 24.44 )
    65.66 ( 14.86 )
    Height
    Units: centimetres
        arithmetic mean (standard deviation)
    166.60 ( 6.2 )
    171.91 ( 9.68 )
    165.81 ( 11.03 )
    165.00 ( 5.13 )
    Body Mass Index
    Units: kilograms/metre
        arithmetic mean (standard deviation)
    28.75 ( 4.78 )
    25.88 ( 2.97 )
    30.43 ( 6.99 )
    24.08 ( 5.07 )

    End points

    		 Close Top of page
    End points reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 • Part 1: 36 Subjects were randomized in a 1:1 ratio to placebo:berotralstat • Part 2: 15 Subjects were randomized in a 1:3:3 ratio to placebo:berotralstat:berotralstat, with 2 different doses of berotralstat. • Part 3: 24 Subjects were randomized in a 1:3:3:3 ratio to placebo:berotralstat:berotralstat:berotralstat, with 3 different doses of berotralstat.

    Reporting group title
    berotralstat
    Reporting group description
    		 • Part 1: 36 Subjects were randomized in a 1:1 ratio of placebo:berotralstat • Part 2: 15 Subjects were randomized in a 1:3:3 ratio of placebo:berotralstat (125mg):berotralstat (250mg). • Part 3: 24 Subjects were randomized in a 1:3:3:3 ratio of placebo:berotralstat (125mg):berotralstat (250mg):berotralstat (62.5mg).

    Subject analysis set title
    berotralstat 350 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (350 mg) administerd orally QD for 28 days.

    Subject analysis set title
    berotralstat 250 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (250 mg) administerd QD for 28 days.

    Subject analysis set title
    berotralstat 125 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (125 mg) administerd QD for 28 days.

    Subject analysis set title
    berotralstat 62.5 mg
    Subject analysis set type
    		 Per protocol
    Subject analysis set description
    Capsules of berotralstat (62.5 mg) administerd QD for 28 days.

    Primary: Difference in HAE Attack Rate

    		 Close Top of page
    End point title
    		 Difference in HAE Attack Rate
    End point description
    Efficacy was evaluated by the number of acute angioedema attacks. To ensure that consistent, objective assessments were used in accepting subject-reported attack data, a panel of expert physicians in the treatment of HAE patients adjudicated all subject-reported attacks prior to their inclusion in primary efficacy analyses.
    End point type
    Primary
    End point timeframe
    Investigators collected data from patient diaries from the first day of dosing through Day 29 (the effective dosing period).
    End point values
    		 Placebo berotralstat berotralstat 350 mg berotralstat 250 mg berotralstat 125 mg berotralstat 62.5 mg
    Number of subjects analysed
    22
    53
    18
    14
    14
    7
    Units: attacks/week
    least squares mean (standard error)
        HAE Attack Rate: Entire Dosing Period
    0.952 ( 0.104 )
    0.494 ( 0.067 )
    0.519 ( 0.115 )
    0.527 ( 0.130 )
    0.249 ( 0.130 )
    0.852 ( 0.185 )
    Statistical analysis title
    		 Difference vs Placebo: All berotralstat
    Statistical analysis description
    The primary analysis of treatment-effect was performed using an ANCOVA with the adjusted qualifying attack rate as the covariate. Statistically significant and clinically meaningful reductions in the rate of attacks were observed in subjects treated with berotralstat compared to placebo at doses ≥ 125 mg QD.
    Comparison groups
    Placebo v berotralstat
    Number of subjects included in analysis
    75
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 ANCOVA
    Parameter type
    		 Difference Least Mean Square
    Point estimate
    -0.458
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.703
         upper limit
    		 -0.214
    Statistical analysis title
    		 Difference vs Placebo: berotralstat 350 mg
    Statistical analysis description
    The primary analysis of treatment-effect was performed using an ANCOVA with the adjusted qualifying attack rate as the covariate. Statistically significant and clinically meaningful reductions in the rate of attacks were observed in subjects treated with 350 mg berotralstat compared to placebo.
    Comparison groups
    Placebo v berotralstat 350 mg
    Number of subjects included in analysis
    40
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.006
    Method
    		 ANCOVA
    Parameter type
    		 Difference Least Mean Square
    Point estimate
    -0.433
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.74
         upper limit
    		 -0.127
    Statistical analysis title
    		 Difference vs Placebo: berotralstat 250 mg
    Statistical analysis description
    The primary analysis of treatment-effect was performed using an ANCOVA with the adjusted qualifying attack rate as the covariate. Statistically significant and clinically meaningful reductions in the rate of attacks were observed in subjects treated with 250 mg berotralstat compared to placebo.
    Comparison groups
    Placebo v berotralstat 250 mg
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.012
    Method
    		 ANCOVA
    Parameter type
    		 Difference Least Mean Square
    Point estimate
    -0.425
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.755
         upper limit
    		 -0.095
    Statistical analysis title
    		 Difference vs Placebo: berotralstat 125 mg
    Statistical analysis description
    The primary analysis of treatment-effect was performed using an ANCOVA with the adjusted qualifying attack rate as the covariate. Statistically significant and clinically meaningful reductions in the rate of attacks were observed in subjects treated with 125 mg berotralstat compared to placebo.
    Comparison groups
    Placebo v berotralstat 125 mg
    Number of subjects included in analysis
    36
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 < 0.001
    Method
    		 ANCOVA
    Parameter type
    		 Difference Least Mean Square
    Point estimate
    -0.703
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -1.033
         upper limit
    		 -0.373
    Statistical analysis title
    		 Difference vs Placebo: berotralstat 62.5 mg
    Statistical analysis description
    The primary analysis of treatment-effect was performed using an ANCOVA with the adjusted qualifying attack rate as the covariate. Statistically significant reductions in the rate of attacks were not observed in subjects treated with 62.5 mg berotralstat compared to placebo.
    Comparison groups
    Placebo v berotralstat 62.5 mg
    Number of subjects included in analysis
    29
    Analysis specification
    Pre-specified
    Analysis type
    		 superiority
    P-value
    		 = 0.639
    Method
    		 ANCOVA
    Parameter type
    		 Difference Least Mean Square
    Point estimate
    -0.1
    Confidence interval
         level
    		 95%
         sides
    2-sided
         lower limit
    		 -0.52
         upper limit
    		 0.32

    Adverse events

    		 Close Top of page
    Adverse events information
    Timeframe for reporting adverse events
    Adverse event data were collected from Day 1 of the treatment period of the study through the last dose plus 30 days.
    Assessment type
    		 Systematic
    Dictionary used for adverse event reporting
    Dictionary name
    		 MedDRA
    Dictionary version
    18.1
    Reporting groups
    Reporting group title
    Placebo
    Reporting group description
    		 The reference in this study was placebo capsules to match berotralstat capsules.

    Reporting group title
    berotralstat
    Reporting group description
    		 Part 1: Subjects were randomized in a 1:1 ratio to placebo:berotralstat. A total of 36 subjects were enrolled and treated in Part 1: 18 berotralstat (350 mg) and 18 placebo subjects. Part 2: Subjects were randomized in a 1:3:3 ratio. A total of 15 subjects were enrolled and treated in Part 2: 6 berotralstat (250mg), 7 berotralstat (125mg) and 2 placebo subjects. Part 3: Subjects were randomized in a 1:3:3:3 ratio to placebo:berotralstat (125mg):berotralstat (250mg):berotralstat (62.5mg). A total of 24 subjects were enrolled and treated in Part 3: 8 berotralstat (250mg), 7 berotralstat (125mg), 7 berotralstat (62.5mg) and 2 placebo subjects.

    Serious adverse events
    		 Placebo berotralstat
    Total subjects affected by serious adverse events
         subjects affected / exposed
    0 / 22 (0.00%)
    1 / 53 (1.89%)
         number of deaths (all causes)
    0
    0
         number of deaths resulting from adverse events
    0
    0
    Gastrointestinal disorders
    Gastrointestinal infection
         subjects affected / exposed
    0 / 22 (0.00%)
    1 / 53 (1.89%)
         occurrences causally related to treatment / all
    0 / 0
    0 / 1
         deaths causally related to treatment / all
    0 / 0
    0 / 0
    Frequency threshold for reporting non-serious adverse events: 2%
    Non-serious adverse events
    		 Placebo berotralstat
    Total subjects affected by non serious adverse events
         subjects affected / exposed
    15 / 22 (68.18%)
    36 / 53 (67.92%)
    Investigations
    Alanine aminotransferase increased
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    Injury, poisoning and procedural complications
    Contusion
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1
    Nervous system disorders
    Headache
         subjects affected / exposed
    4 / 22 (18.18%)
    6 / 53 (11.32%)
         occurrences all number
    4
    6
    Migraine
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    General disorders and administration site conditions
    Fatigue
         subjects affected / exposed
    1 / 22 (4.55%)
    3 / 53 (5.66%)
         occurrences all number
    1
    3
    Influenza like illness
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1
    Gastrointestinal disorders
    Abdominal pain
         subjects affected / exposed
    0 / 22 (0.00%)
    6 / 53 (11.32%)
         occurrences all number
    0
    6
    Abdominal pain upper
         subjects affected / exposed
    0 / 22 (0.00%)
    3 / 53 (5.66%)
         occurrences all number
    0
    3
    Diarrhoea
         subjects affected / exposed
    2 / 22 (9.09%)
    6 / 53 (11.32%)
         occurrences all number
    2
    6
    Flatulence
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    Nausea
         subjects affected / exposed
    0 / 22 (0.00%)
    6 / 53 (11.32%)
         occurrences all number
    0
    6
    Vomiting
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    Constipation
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1
    Gastrooesophageal reflux disease
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1
    Skin and subcutaneous tissue disorders
    Rash
         subjects affected / exposed
    2 / 22 (9.09%)
    0 / 53 (0.00%)
         occurrences all number
    2
    0
    Musculoskeletal and connective tissue disorders
    Arthralgia
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1
    Infections and infestations
    Nasopharyngitis
         subjects affected / exposed
    6 / 22 (27.27%)
    8 / 53 (15.09%)
         occurrences all number
    6
    8
    Pharyngitis
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    Gastrointestinal infection
         subjects affected / exposed
    0 / 22 (0.00%)
    2 / 53 (3.77%)
         occurrences all number
    0
    2
    Upper respiratory tract infection
         subjects affected / exposed
    1 / 22 (4.55%)
    1 / 53 (1.89%)
         occurrences all number
    1
    1

    More information

    		 Close Top of page

    Substantial protocol amendments (globally)
    Were there any global substantial amendments to the protocol? Yes
    Date
    Amendment
    23 Sep 2016
    Protocol Amendment 1.0 * The follow-up visit was changed from Day 42 to Day 44. *In the inclusion criteria, clinical diagnosis for Type 1 HAE was modified. *The contraceptive requirements were updated. *Summary findings for Study BCX7353 101 were updated based on final data for the study.
    30 Dec 2016
    Protocol Amendment 2.0 Additional safety laboratory analyses were added.
    21 Mar 2017
    Protocol Amendment 3.0 *The power assessment based on sample size and study attack rate was corrected. *Reference to the collection of a blood sample for cleaved HK as a possible exploratory PD analysis was removed..

    Interruptions (globally)
    Were there any global interruptions to the trial? No

    Limitations and caveats
    Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data.
    None reported

    Online references
    http://www.ncbi.nlm.nih.gov/pubmed/30044938
    For support, Contact us.
    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

    European Medicines Agency © 1995-Fri Apr 26 06:56:51 CEST 2024 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA