Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

Hereditary Angioedema (HAE) Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03240133
• Other study ID #: BCX7353-202
• Status: Completed
• Phase: Phase 2
• Start date: July 31, 2017
• Est. completion date: January 29, 2019

Study information

• Verified date: March 2021
• Source: BioCryst Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 58
• Est. completion date: January 29, 2019
• Est. primary completion date: January 29, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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.

Related Conditions & MeSH terms

• Angioedema
• Hereditary Angioedema (HAE)

Intervention

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

Locations

Country	Name	City	State
Austria	Study Center	Graz
Denmark	Study Center	Odense
France	Study Center	Grenoble
France	Study Center	Lille
Germany	Study Center	Berlin
Germany	Study Center	Frankfurt
Hungary	Study Center	Budapest
Israel	Study Center	Ashkelon
Israel	Study Center	Tel Aviv
Israel	Study Center	Tel HaShomer
Italy	Study Center	Milano
Italy	Study center	Padova
Italy	Study Center	Salerno
North Macedonia	Study Center	Skopje
Poland	Study Center	Kraków
Romania	Study Center	Târgu-Mures
Switzerland	Study Center	Zürich
United Kingdom	Study Center	Birmingham
United Kingdom	Study Center	Bristol
United Kingdom	Study Center	Cambridge
United Kingdom	Study Center	London
United Kingdom	Study Center	Manchester
United Kingdom	Study Center	Plymouth
United Kingdom	Study Center	Southampton

Sponsors (1)

Lead Sponsor	Collaborator
BioCryst Pharmaceuticals

Countries where clinical trial is conducted

Austria,  Denmark,  France,  Germany,  Hungary,  Israel,  Italy,  North Macedonia,  Poland,  Romania,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Subjects completed a 3-component VAS on a 100 mm scale for severity of abdominal pain, skin pain and skin swelling associated with the HAE attack, where zero indicated no pain or swelling and 100 mm indicated worst possible pain or swelling. Subjects completed the VAS immediately prior to study drug administration, then at 1, 2, 3, 4, approximately 8 & at 24 hours post-dose. The primary endpoint was the proportion of subject attacks with an improved or stable 3-symptom composite VAS score at 4 hours post dose. The 3-symptom composite was calculated as the average of the VAS scores for abdominal pain, skin pain, and skin swelling. A subject was considered improved or stable if the change from baseline (CFB; time of drug administration) in VAS was = 0.	Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose
Primary	Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours	The proportion of attacks for which subjects took SOC-Rx in the 24 hours following treatment with study drug. HAE Rescue Medications included C1-INH (Berinert, Cinryze, Ruconest) and Firazyr/Icatibant.	24 hours