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Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03240133
Recruitment Status : Completed
First Posted : August 4, 2017
Last Update Posted : July 10, 2019
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

Tracking Information
First Submitted Date  ICMJE August 2, 2017
First Posted Date  ICMJE August 4, 2017
Last Update Posted Date July 10, 2019
Actual Study Start Date  ICMJE July 31, 2017
Actual Primary Completion Date January 29, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 3, 2017)
Proportion of subjects with improved or stable composite visual analog scale (VAS) score [ Time Frame: 4 hours postdose ]
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: June 7, 2018)
  • Proportion of subject attacks with a patient global assessment of improved or stable symptoms [ Time Frame: 4 hours post-dose ]
  • Proportion of subject attacks with no symptoms or mild symptoms of an HAE attack [ Time Frame: 4 hours post-dose ]
  • Proportion of subject attacks requiring standard of care attack treatment [ Time Frame: 24 hours post-dose ]
  • Time to use of standard of care acute attack treatment [ Time Frame: 24 hours post-dose ]
  • Time to stable or improved symptoms [ Time Frame: 24 hours post-dose ]
  • Time to symptom relief [ Time Frame: first documented 50% reduction in VAS score ]
  • Time to almost complete symptom relief [ Time Frame: first documented VAS score <10mm ]
  • Time to initial symptom relief [ Time Frame: time of study ingestion to worst of symptoms is over ]
  • Time to complete symptom relief [ Time Frame: time of study ingestion to attack has ended ]
  • Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 16 days post third dose ]
Original Secondary Outcome Measures  ICMJE
 (submitted: August 3, 2017)
  • Proportion of subject attacks with a patient global assessment of improved or stable symptoms [ Time Frame: 4 hours post-dose ]
  • Proportion of subject attacks with no symptoms or mild symptoms of an HAE attack [ Time Frame: 4 hours post-dose ]
  • Proportion of subject attacks requiring standard of care attack treatment [ Time Frame: 24 hours post-dose ]
  • Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 16 days post third dose ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
Official Title  ICMJE A Randomized, Double-blind, Placebo-controlled, Dose-ranging, Study to Evaluate the Efficacy, Safety and Tolerability of Single Doses of BCX7353 as an Acute Attack Treatment in Subjects With Hereditary Angioedema
Brief Summary This 3-part study will evaluate the efficacy and safety of an oral kallikrein inhibitor, BCX7353, in the treatment angioedema attacks in subjects with Type I or II hereditary angioedema (HAE). In each study part, subjects will treat 3 attacks with BCX7353 (2 attacks) or placebo (1 attack), in a randomly allocated order. In Part 1, the dose of 750mg will be assessed relative to placebo in up to 36 patients. If this is shown to be effective, then a further 12 patients will be enrolled at a 500mg dose (Part 1), followed by a further 12 (if efficacy still shown) at a dose of 250mg (Part 3) to determine the minimum effective dose of BCX7353 compared to placebo for treating HAE attacks. Efficacy will be determined by subject diary entries completed at pre-defined times post-dose.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Hereditary Angioedema (HAE)
Intervention  ICMJE
  • Drug: BCX7353
    oral liquid formulation
  • Drug: Placebo
    oral liquid formulation to match BCX7353
Study Arms  ICMJE
  • Experimental: Part1: BCX7353 750 mg
    Intervention: Drug: BCX7353
  • Experimental: Part 2: BCX7353 500 mg
    Intervention: Drug: BCX7353
  • Experimental: Part 3: BCX7353 250 mg
    Intervention: Drug: BCX7353
  • Placebo Comparator: Parts 1, 2 and 3: placebo
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: August 3, 2017)
60
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE January 29, 2019
Actual Primary Completion Date January 29, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Able to provide written, informed consent.
  2. A clinical diagnosis of hereditary angioedema Type 1 or Type 2 as documented at any time in the medical records or at the screening visit.
  3. Access to and ability to use standard of care acute attack treatment for attacks of HAE.
  4. Sexually active women of child-bearing potential and sexually active men must utilize effective contraception.

Exclusion Criteria:

  1. Women who are pregnant or breast-feeding.
  2. Any clinical condition or medical history that would interfere with the subject's safety or ability to participate in the study.
  3. Use of C1INH, androgens or tranexamic acid for prophylaxis of HAE attacks.
  4. History of or current alcohol or drug abuse.
  5. Infection with hepatitis B, hepatitis C or HIV.
  6. Participation in any other investigational drug study currently or within the last 30 days.
  7. Positive drugs of abuse screen (unless as used as medical treatment, e.g., with a prescription).
  8. An immediate family relationship to either Sponsor employees, the Investigator or employees of the study site.
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 70 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Austria,   Denmark,   France,   Germany,   Hungary,   Israel,   Italy,   North Macedonia,   Poland,   Romania,   Switzerland,   United Kingdom
Removed Location Countries Macedonia, The Former Yugoslav Republic of
 
Administrative Information
NCT Number  ICMJE NCT03240133
Other Study ID Numbers  ICMJE BCX7353-202
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party BioCryst Pharmaceuticals
Study Sponsor  ICMJE BioCryst Pharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Hilary Longhurst, MBBS, PhD Barts & The London NHS Trust
PRS Account BioCryst Pharmaceuticals
Verification Date July 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP