Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

• ClinicalTrials.gov Identifier: NCT03240133
• Recruitment Status: Completed
• First Posted: August 4, 2017
• Last Update Posted: July 10, 2019
• Sponsor: BioCryst Pharmaceuticals
• Information provided by (Responsible Party): BioCryst Pharmaceuticals

Tracking Information

• First Submitted Date: August 2, 2017
• First Posted Date: August 4, 2017
• Last Update Posted Date: July 10, 2019
• Actual Study Start Date: July 31, 2017
• Actual Primary Completion Date: January 29, 2019 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (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: Same as current

Current Secondary Outcome Measures (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 (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: Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
• 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
• 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: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: Randomized; Intervention Model: Crossover Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
• Condition: Hereditary Angioedema (HAE)

Intervention

• Drug: BCX7353
  oral liquid formulation
• Drug: Placebo
  oral liquid formulation to match BCX7353

Study Arms

• 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

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 3, 2017): 60
• Original Estimated Enrollment: Same as current
• Actual Study Completion Date: January 29, 2019
• Actual Primary Completion Date: January 29, 2019 (Final data collection date for primary outcome measure)

Eligibility 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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years to 70 Years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: 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: NCT03240133
• Other Study ID Numbers: 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: Not Provided
• Responsible Party: BioCryst Pharmaceuticals
• Study Sponsor: BioCryst Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Principal Investigator: Hilary Longhurst, MBBS, PhD; Barts & The London NHS Trust

• PRS Account: BioCryst Pharmaceuticals
• Verification Date: July 2019