A Long Term Safety Study of BCX7353 in Hereditary Angioedema (APeX-S)

• ClinicalTrials.gov Identifier: NCT03472040
• Recruitment Status: Active, not recruiting
• First Posted: March 21, 2018
• Last Update Posted: December 17, 2020
• Sponsor: BioCryst Pharmaceuticals
• Information provided by (Responsible Party): BioCryst Pharmaceuticals

Tracking Information

• First Submitted Date: February 27, 2018
• First Posted Date: March 21, 2018
• Last Update Posted Date: December 17, 2020
• Actual Study Start Date: February 16, 2018
• Estimated Primary Completion Date: February 2024 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: March 5, 2019)

The long term safety of oral BCX7353 capsules [ Time Frame: 96 weeks ]

The number and percentage of subjects with treatment-emergent adverse events

• Original Primary Outcome Measures (submitted: March 14, 2018): The long term safety of oral BCX7353 capsules based on analysis of treatment emergent adverse events and laboratory abnormalities [ Time Frame: 48 weeks ]

Current Secondary Outcome Measures (submitted: March 5, 2019)

• The rate of acute attacks of angioedema during treatment [ Time Frame: 96 weeks ]
• The durability of response to treatment [ Time Frame: 96 weeks ]
  To evaluate if the rate of attacks remains consistent (durable) over time
• Patient reported quality of life (QoL) during treatment [ Time Frame: 96 weeks ]
  To measure changes in quality of life over time from baseline, assessed by use of an HAE-specific Angioedema QoL (AE-QoL) questionnaire
• Patient's satisfaction with medication during long term administration of BCX7353 [ Time Frame: 96 weeks ]
  To measure satisfaction with study medication using the Treatment Satisfaction Questionnaire for Medication (TSQM)

Original Secondary Outcome Measures (submitted: March 14, 2018)

• The rate of acute attacks of angioedema during treatment [ Time Frame: 48 weeks ]
• The durability of response to treatment [ Time Frame: 48 weeks ]
  To evaluate if the rate of attacks remains consistent (durable) over time
• Patient reported quality of life (QoL) during treatment [ Time Frame: 48 weeks ]
  To measure changes in quality of life over time from baseline, assessed by use of an HAE-specific Angioedema QoL (AE-QoL) questionnaire
• Patient's satisfaction with medication during long term administration of BCX7353 [ Time Frame: 48 weeks ]
  To measure satisfaction with study medication using the Treatment Satisfaction Questionnaire for Medication (TSQM)

• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: A Long Term Safety Study of BCX7353 in Hereditary Angioedema
• Official Title: An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
• Brief Summary: This is an open-label study to evaluate the long term safety and effectiveness of oral treatment with BCX7353 in preventing acute angioedema attacks in patients with Type I and Type II HAE.
• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2; Phase 3
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Prevention

Condition

• Hereditary Angioedema
• HAE
• Prophylaxis

Intervention

Drug: BCX7353

BCX7353 mg oral capsules administered once daily

Study Arms

Experimental: BCX7353 150 mg once daily

Intervention: Drug: BCX7353

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Active, not recruiting
• Actual Enrollment (submitted: December 15, 2020): 386
• Original Estimated Enrollment (submitted: March 14, 2018): 160
• Estimated Study Completion Date: February 2024
• Estimated Primary Completion Date: February 2024 (Final data collection date for primary outcome measure)

Eligibility Criteria

Key Inclusion Criteria:

• Subjects with HAE Type I or II who either have participated in a previous BCX7353 study or, in selected countries, in the opinion of the Investigator are expected to derive benefit from an oral treatment for the prevention of angioedema attacks.
• Access to appropriate medication for treatment of acute attacks
• Acceptable effective contraception
• Written informed consent

Key Exclusion Criteria:

• Pregnancy or breast-feeding
• Any clinically significant medical condition or medical history that, in the opinion of the Investigator or Sponsor, would interfere with the subject's safety or ability to participate in the study
• Any laboratory parameter abnormality that, in the opinion of the Investigator, is clinically significant and relevant for this study
• Discontinuation of study drug due to a hypersensitivity reaction BCX7353 in a prior study
• Severe hypersensitivity to multiple medicinal products or severe hypersensitivity/ anaphylaxis with unclear etiology
• Unacceptable noncompliance in a previous BCX7353 study (if applicable) as assessed by the Sponsor or Investigator
• Investigational drug exposure, other than BCX7353, within 30 days prior to the screening visit (or baseline if no screening visit)

Sex/Gender

• Sexes Eligible for Study: All
• Gender Based Eligibility: Yes

• Ages: 12 Years and older (Child, Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: Australia, Austria, Denmark, France, Germany, Hong Kong, Hungary, Israel, Italy, Korea, Republic of, New Zealand, North Macedonia, Poland, Serbia, Slovakia, South Africa, Spain, Switzerland, United Kingdom, United States
• Removed Location Countries: Macedonia, The Former Yugoslav Republic of

Administrative Information

• NCT Number: NCT03472040
• Other Study ID Numbers: BCX7353-204
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No
• Product Manufactured in and Exported from the U.S.: Yes

IPD Sharing Statement

• Plan to Share IPD: No

• Responsible Party: BioCryst Pharmaceuticals
• Study Sponsor: BioCryst Pharmaceuticals
• Collaborators: Not Provided

Investigators

• Principal Investigator: Henriette Farkas, MD; Semmelweis University, Budapest, Hungary

• PRS Account: BioCryst Pharmaceuticals
• Verification Date: December 2020