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Small Molecule

BCX7353 capsules for Hereditary Angioedema (APeX-2 Trial)

Phase 3

Waitlist Available

Led By Bruce Zuraw, MD

Research Sponsored by BioCryst Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up part 2: 24 weeks (days 169 to 337). part 3: 48 weeks (days 338 to 674).

Awards & highlights

APeX-2 Trial Summary

This trial is testing a new drug to see if it can prevent acute angioedema attacks in people with Type I or Type II HAE.

Eligible Conditions

Hereditary Angioedema (HAE)
Hereditary Angioedema

APeX-2 Trial Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ part 2: 24 weeks (days 169 to 337). part 3: 48 weeks (days 338 to 674).

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~part 2: 24 weeks (days 169 to 337). part 3: 48 weeks (days 338 to 674).

This trial's timeline: 3 weeks for screening, Varies for treatment, and part 2: 24 weeks (days 169 to 337). part 3: 48 weeks (days 338 to 674). for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Part 1: The Rate of Investigator-confirmed HAE Attacks During Dosing in the Entire 24-week Treatment Period (Day 1 to Day 168)

Part 2 & 3: To Evaluate the Long-term Safety and Tolerability of Berotralstat 110 and 150 mg in Subjects With HAE

Secondary outcome measures

Part 1: Change From Baseline in Angioedema Quality of Life Questionnaire at Week 24 (Total Score)

Part 1: Proportion of Days With Angioedema Symptoms Through 24 Weeks

Part 1: Rate of Expert-confirmed Angioedema Events During Dosing in the Effective Treatment Period

+3 more

Side effects data

From 2022 Phase 3 trial • 121 Patients • NCT03485911

26%

Nasopharyngitis

17%

Nausea

14%

Dyspepsia

14%

Upper respiratory tract infection

12%

Urinary tract infection

12%

Diarrhoea

12%

Gastroesophageal reflux disease

Vomiting

Gastroenteritis viral

Abdominal pain

Oropharyngeal pain

Fatigue

Headache

Influenza

Cough

Toothache

Anxiety

Abdominal discomfort

Flatulence

Abdominal distension

Depression

Gastroenteritis

Sinusitis

Back pain

Contusion

Arthralgia

Pneumonia

Bipolar disorder

Gastritis alcoholic

Obesity

Limb injury

Oedema peripheral

Abdominal pain upper

Plasma cell myeloma

Pleural effusion

Pain in extremity

100%

80%

60%

40%

20%

Study treatment Arm

Placebo

Berotralstat 110mg Once Daily

Berotralstat 150mg Once Daily

APeX-2 Trial Design

3Treatment groups

Experimental Treatment

Placebo Group

Group I: BCX7353 150 mg once dailyExperimental Treatment1 Intervention

BCX7353 administered as oral capsules once daily

Group II: BCX7353 110 mg once dailyExperimental Treatment1 Intervention

BCX7353 administered as oral capsules once daily

Group III: PlaceboPlacebo Group1 Intervention

Matching placebo administered as oral capsules once daily

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

BCX7353 capsules

2019

Completed Phase 3

~140

Do I qualify?Apply below to find out

Find a Location

Who is running the clinical trial?

BioCryst PharmaceuticalsLead Sponsor

53 Previous Clinical Trials

4,366 Total Patients Enrolled

Bruce Zuraw, MDPrincipal InvestigatorUC San Diego School of Medicine, US HAE Angioedema Center

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Has BCX7353 been trialed before in a medical setting?

"BCX7353 capsules were first looked into back in 2018 at Study Center. As of now, there have been a total of 180 completed clinical trials. Right now, there are 3 studies that are still recruiting patients; a number of these studies taking place in Birmingham, Alabama."

Answered by AI

How many different places can patients receive this treatment?

"To participate in this trial, it is important to select a location nearest you from the 30 different sites. These include cities like Birmingham, Dallas and Scottsdale as well as other locations."

Answered by AI

Is this study actively looking for participants at this time?

"The latest information from clinicaltrials.gov reveals that this particular trial is not currently looking for any more patients. The study was posted on February 6th, 2018 and updated March 2nd, 2022. Even though this study isn't taking place at the moment, there are 25 other studies that are presently recruiting patients."

Answered by AI

Recent research and studies

The Journal of Allergy and Clinical Immunology: In PracticeJournal

Randomized Trial of the Efficacy and Safety of Berotralstat (BCX7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: Results of Apex-2 Through 48 Weeks (part 2)

Wedner, H. James, Emel Aygören-Pürsün, Jonathan Bernstein, Timothy Craig, Richard Gower, Joshua S. Jacobs, Douglas T. Johnston, et al.. 2021. “Randomized Trial of the Efficacy and Safety of Berotralstat (BCX7353) as an Oral Prophylactic Therapy for Hereditary Angioedema: Results of Apex-2 Through 48 Weeks (part 2)”. The Journal of Allergy and Clinical Immunology: In Practice. Elsevier BV. doi:10.1016/j.jaip.2021.03.057.

Journal of Allergy and Clinical ImmunologyJournal

Oral Once-daily Berotralstat for the Prevention of Hereditary Angioedema Attacks: A Randomized, Double-blind, Placebo-controlled Phase 3 Trial

Zuraw, Bruce, William R. Lumry, Douglas T. Johnston, Emel Aygören-Pürsün, Aleena Banerji, Jonathan A. Bernstein, Sandra C. Christiansen, et al.. 2021. “Oral Once-daily Berotralstat for the Prevention of Hereditary Angioedema Attacks: A Randomized, Double-blind, Placebo-controlled Phase 3 Trial”. Journal of Allergy and Clinical Immunology. Elsevier BV. doi:10.1016/j.jaci.2020.10.015.

clinicaltrials.govStudy

Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE

2018. "Efficacy and Safety Study of BCX7353 as an Oral Treatment for the Prevention of Attacks in HAE". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT03485911.