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Trial record 1 of 1 for:    NCT03240133
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Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

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ClinicalTrials.gov Identifier: NCT03240133
Recruitment Status : Completed
First Posted : August 4, 2017
Results First Posted : April 1, 2021
Last Update Posted : April 1, 2021
Sponsor:
Information provided by (Responsible Party):
BioCryst Pharmaceuticals

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.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema (HAE) Drug: BCX7353 Drug: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 58 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : July 31, 2017
Actual Primary Completion Date : January 29, 2019
Actual Study Completion Date : January 29, 2019

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Part1: BCX7353 750 mg Drug: BCX7353
oral liquid formulation

Experimental: Part 2: BCX7353 500 mg Drug: BCX7353
oral liquid formulation

Experimental: Part 3: BCX7353 250 mg Drug: BCX7353
oral liquid formulation

Placebo Comparator: Parts 1, 2 and 3: placebo Drug: Placebo
oral liquid formulation to match BCX7353




Primary Outcome Measures :
  1. Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score [ Time Frame: Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose ]
    Subjects completed a 3-component VAS on a 100 mm scale for severity of abdominal pain, skin pain and skin swelling associated with the HAE attack, where zero indicated no pain or swelling and 100 mm indicated worst possible pain or swelling. Subjects completed the VAS immediately prior to study drug administration, then at 1, 2, 3, 4, approximately 8 & at 24 hours post-dose. The primary endpoint was the proportion of subject attacks with an improved or stable 3-symptom composite VAS score at 4 hours post dose. The 3-symptom composite was calculated as the average of the VAS scores for abdominal pain, skin pain, and skin swelling. A subject was considered improved or stable if the change from baseline (CFB; time of drug administration) in VAS was ≤ 0.

  2. Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours [ Time Frame: 24 hours ]
    The proportion of attacks for which subjects took SOC-Rx in the 24 hours following treatment with study drug. HAE Rescue Medications included C1-INH (Berinert, Cinryze, Ruconest) and Firazyr/Icatibant.



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Ages Eligible for Study:   18 Years to 70 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03240133


Locations
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Austria
Study Center
Graz, Austria
Denmark
Study Center
Odense, Denmark
France
Study Center
Grenoble, France
Study Center
Lille, France
Germany
Study Center
Berlin, Germany
Study Center
Frankfurt, Germany
Hungary
Study Center
Budapest, Hungary
Israel
Study Center
Ashkelon, Israel
Study Center
Tel Aviv, Israel
Study Center
Tel HaShomer, Israel
Italy
Study Center
Milano, Italy
Study center
Padova, Italy
Study Center
Salerno, Italy
North Macedonia
Study Center
Skopje, North Macedonia
Poland
Study Center
Kraków, Poland
Romania
Study Center
Târgu-Mureş, Romania
Switzerland
Study Center
Zürich, Switzerland
United Kingdom
Study Center
Birmingham, United Kingdom
Study Center
Bristol, United Kingdom
Study Center
Cambridge, United Kingdom
Study Center
London, United Kingdom
Study Center
Manchester, United Kingdom
Study Center
Plymouth, United Kingdom
Study Center
Southampton, United Kingdom
Sponsors and Collaborators
BioCryst Pharmaceuticals
Investigators
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Principal Investigator: Hilary Longhurst, MBBS, PhD Barts & The London NHS Trust
  Study Documents (Full-Text)

Documents provided by BioCryst Pharmaceuticals:
Statistical Analysis Plan  [PDF] January 29, 2019
Study Protocol  [PDF] March 16, 2018

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Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03240133    
Other Study ID Numbers: BCX7353-202
First Posted: August 4, 2017    Key Record Dates
Results First Posted: April 1, 2021
Last Update Posted: April 1, 2021
Last Verified: March 2021

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by BioCryst Pharmaceuticals:
HAE
hereditary angioedema
kallikrein inhibitor
plasma kallikrein
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn