Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03240133
• Date of registration: 02/08/2017
• Prospective Registration: No
• Primary sponsor: BioCryst Pharmaceuticals
• Public title: Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
• Scientific 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
• Date of first enrolment: July 31, 2017
• Target sample size: 60
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03240133
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Double (Participant, Investigator).
• Phase: Phase 2

Countries of recruitment

Austria, Denmark, France, Germany, Hungary, Israel, Italy, Macedonia, The Former Yugoslav Republic of, North Macedonia, Poland, Romania, Switzerland, United Kingdom

Contacts

• Name: Hilary Longhurst, MBBS, PhD
• Affiliation: Barts & The London NHS Trust

Key inclusion & exclusion 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.



Age minimum: 18 Years
Age maximum: 70 Years
Gender: All

Health Condition(s) or Problem(s) studied

Hereditary Angioedema (HAE)

Intervention(s)

Drug: BCX7353

Drug: Placebo

Primary Outcome(s)

Proportion of subjects with improved or stable composite visual analog scale (VAS) score [Time Frame: 4 hours postdose]

Secondary Outcome(s)

Time to complete symptom relief [Time Frame: time of study ingestion to attack has ended]

Time to symptom relief [Time Frame: first documented 50% reduction in VAS score]

Proportion of subject attacks with no symptoms or mild symptoms of an HAE attack [Time Frame: 4 hours post-dose]

Proportion of subject attacks with a patient global assessment of improved or stable symptoms [Time Frame: 4 hours post-dose]

Time to stable or improved symptoms [Time Frame: 24 hours post-dose]

Time to use of standard of care acute attack treatment [Time Frame: 24 hours post-dose]

Time to almost complete symptom relief [Time Frame: first documented VAS score <10mm]

Incidence and severity of adverse events and laboratory abnormalities [Time Frame: 16 days post third dose]

Proportion of subject attacks requiring standard of care attack treatment [Time Frame: 24 hours post-dose]

Time to initial symptom relief [Time Frame: time of study ingestion to worst of symptoms is over]

Secondary ID(s)

BCX7353-202

Source(s) of Monetary Support

Please refer to primary and secondary sponsors