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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 : Recruiting
First Posted : August 4, 2017
Last Update Posted : June 11, 2018
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

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 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
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018

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: 4 hours postdose ]

Secondary Outcome Measures :
  1. Proportion of subject attacks with a patient global assessment of improved or stable symptoms [ Time Frame: 4 hours post-dose ]
  2. Proportion of subject attacks with no symptoms or mild symptoms of an HAE attack [ Time Frame: 4 hours post-dose ]
  3. Proportion of subject attacks requiring standard of care attack treatment [ Time Frame: 24 hours post-dose ]
  4. Time to use of standard of care acute attack treatment [ Time Frame: 24 hours post-dose ]
  5. Time to stable or improved symptoms [ Time Frame: 24 hours post-dose ]
  6. Time to symptom relief [ Time Frame: first documented 50% reduction in VAS score ]
  7. Time to almost complete symptom relief [ Time Frame: first documented VAS score <10mm ]
  8. Time to initial symptom relief [ Time Frame: time of study ingestion to worst of symptoms is over ]
  9. Time to complete symptom relief [ Time Frame: time of study ingestion to attack has ended ]
  10. Incidence and severity of adverse events and laboratory abnormalities [ Time Frame: 16 days post third dose ]


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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


Contacts
Contact: Clinical Trials 919-859-1302 clinicaltrials@biocryst.com

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

Responsible Party: BioCryst Pharmaceuticals
ClinicalTrials.gov Identifier: NCT03240133     History of Changes
Other Study ID Numbers: BCX7353-202
First Posted: August 4, 2017    Key Record Dates
Last Update Posted: June 11, 2018
Last Verified: June 2018

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:
Angioedema
Angioedemas, Hereditary
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Genetic Diseases, Inborn
Kallikreins
Coagulants
Fertility Agents, Male
Fertility Agents
Reproductive Control Agents
Physiological Effects of Drugs