A Long Term Safety Study of BCX7353 in Hereditary Angioedema

• Register: ClinicalTrials.gov
• Last refreshed on: 23 January 2023
• Main ID: NCT03472040
• Date of registration: 27/02/2018
• Prospective Registration: No
• Primary sponsor: BioCryst Pharmaceuticals
• Public title: A Long Term Safety Study of BCX7353 in Hereditary Angioedema APeX-S
• Scientific title: An Open-label Study to Evaluate the Long-term Safety of Daily Oral BCX7353 in Subjects With Type I and II Hereditary Angioedema
• Date of first enrolment: February 16, 2018
• Target sample size: 386
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03472040
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Prevention. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

Australia, Austria, Denmark, France, Germany, Hong Kong, Hungary, Israel, Italy, Korea, Republic of, Macedonia, The Former Yugoslav Republic of, New Zealand, North Macedonia, Poland, Serbia, Slovakia, South Africa, Spain, Switzerland, United Kingdom, United States

Contacts

• Name: Henriette Farkas, MD
• Affiliation: Semmelweis University, Budapest, Hungary

Key inclusion & exclusion 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)



Age minimum: 12 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Hereditary Angioedema

Prophylaxis

HAE

Intervention(s)

Drug: BCX7353

Primary Outcome(s)

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

Secondary Outcome(s)

Patient's satisfaction with medication during long term administration of BCX7353 [Time Frame: 96 weeks]

The durability of response to treatment [Time Frame: 96 weeks]

The rate of acute attacks of angioedema during treatment [Time Frame: 96 weeks]

Patient reported quality of life (QoL) during treatment [Time Frame: 96 weeks]

Secondary ID(s)

BCX7353-204

Source(s) of Monetary Support

Please refer to primary and secondary sponsors