Clinical trial NCT03240133: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Hereditary Angioedema (HAE)
Keywords: HAE
hereditary angioedema
kallikrein inhibitor
plasma kallikrein
Lead sponsor: BioCryst Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2017-08-04
Enrollment: 58
Sex: ALL
Minimum age (years): 18
Maximum age (years): 70
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

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.


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.

Primary outcomes:
measure: Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score
timeFrame: Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose
measure: Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours
timeFrame: 24 hours
Secondary outcomes:
Other outcomes:
Locations:
city: Graz
country: Austria
facility: Study Center
city: Odense
country: Denmark
facility: Study Center
city: Grenoble
country: France
facility: Study Center
city: Lille
country: France
facility: Study Center
city: Berlin
country: Germany
facility: Study Center
city: Frankfurt
country: Germany
facility: Study Center
city: Budapest
country: Hungary
facility: Study Center
city: Ashkelon
country: Israel
facility: Study Center
city: Tel Aviv
country: Israel
facility: Study Center
city: Tel HaShomer
country: Israel
facility: Study Center
city: Milano
country: Italy
facility: Study Center
city: Padova
country: Italy
facility: Study center
city: Salerno
country: Italy
facility: Study Center
city: Skopje
country: North Macedonia
facility: Study Center
city: Kraków
country: Poland
facility: Study Center
city: Târgu-Mureş
country: Romania
facility: Study Center
city: Zürich
country: Switzerland
facility: Study Center
city: Birmingham
country: United Kingdom
facility: Study Center
city: Bristol
country: United Kingdom
facility: Study Center
city: Cambridge
country: United Kingdom
facility: Study Center
city: London
country: United Kingdom
facility: Study Center
city: Manchester
country: United Kingdom
facility: Study Center
city: Plymouth
country: United Kingdom
facility: Study Center
city: Southampton
country: United Kingdom
facility: Study Center
Study officials:
affiliation: Barts & The London NHS Trust
name: Hilary Longhurst, MBBS, PhD
role: PRINCIPAL_INVESTIGATOR
References: