Trial · NCT03240133

Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema

NCT03240133, Phase 2, Completed, BioCryst Pharmaceuticals; results first posted 2021-04-01

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema. Trials, record NCT03240133. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03240133

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Study of BCX7353 as a Treatment for Attacks of Hereditary Angioedema
NCT number: NCT03240133
Status: Completed
Phases: Phase 2
Enrollment: 58 participants, actual
Lead sponsor: BioCryst Pharmaceuticals
Conditions: Hereditary Angioedema (HAE)
Interventions: BCX7353, Placebo
First posted: 2017-08-04
Last updated: 2021-04-01
Results first posted: 2021-04-01
Limitations and caveats: In this interim clinical study report (CSR), the VAS change from pre-dose at 4 hours and the proportion of attacks requiring SOC-Rx through the 24 hour post dose were discussed. Assessment of other efficacy endpoints were to be discussed in a final CSR, but this is no longer planned due to a decision to not proceed further with trials of acute treatment of HAE attacks with berotralstat.

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose	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.
Primary	Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours	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.

Source ClinicalTrials.gov

Outcomes2

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Mean composite VAS for HAE attack symptoms severity prior to IMP treatment and 4 hours post-dose	The number of participants analyzed in each analysis set corresponds to number of HAE attacks treated with placebo or berotralstat.	VAS score - millimeters	MEAN
PRIMARY	Percentage of Attacks Treated With Standard of Care Acute Attack Medication (SOC-Rx) Through 24 Hours	24 hours		Percentage attacks treated with SOC-Rx	NUMBER

Source ClinicalTrials.gov

Results by group18

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 1: Berotralstat 750 mg - Predose	13.96	Standard Deviation 9.84	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 1: Placebo - Pre-dose	15.04	Standard Deviation 11.90	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 1: Berotralstat 750 mg - 4hr Post-dose	10.54	Standard Deviation 11.39	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 1: Placebo - 4hr Post-dose	18.42	Standard Deviation 14.19	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 2: Berotralstat 500 mg - Predose	17.69	Standard Deviation 15.25	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 2: Placebo - Pre-dose	13.48	Standard Deviation 16.11	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 2: Berotralstat 500 mg - 4hr Post-dose	11.31	Standard Deviation 15.75	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 2: Placebo - 4hr Post-dose	9.26	Standard Deviation 11.53	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 3: Berotralstat 250 mg - Predose	14.57	Standard Deviation 11.78	VAS score - millimeters
Proportion of Subjects With Improved or Stable Composite Visual Analog Scale (VAS) Score	Part 3: Placebo - Pre-dose	11.33	Standard Deviation 10.17	VAS score - millimeters

1 to 10 of 18Source ClinicalTrials.gov

See all 18 results by group

Participant flow18

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks	33
Overall Study	STARTED	Part 2: Berotralstat (500 mg) & Placebo Treated HAE Attacks	14
Overall Study	STARTED	Part 3: Berotralstat (250 mg) & Placebo Treated HAE Attacks	11
Overall Study	Completed Period 1	Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks	33
Overall Study	Completed Period 1	Part 2: Berotralstat (500 mg) & Placebo Treated HAE Attacks	14
Overall Study	Completed Period 1	Part 3: Berotralstat (250 mg) & Placebo Treated HAE Attacks	11
Overall Study	Completed Period 2	Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks	32
Overall Study	Completed Period 2	Part 2: Berotralstat (500 mg) & Placebo Treated HAE Attacks	11
Overall Study	Completed Period 2	Part 3: Berotralstat (250 mg) & Placebo Treated HAE Attacks	11
Overall Study	Completed Period 3	Part 1: Berotralstat (750 mg) & Placebo Treated HAE Attacks	30

1 to 10 of 18Source ClinicalTrials.gov

See all 18 participant flow

Adverse events128

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk	Events
other	Arthropod bite	Injury, poisoning and procedural complications	Part1: Berotralstat 750 mg	0	33	0
other	Arthropod bite	Injury, poisoning and procedural complications	Part 2: Berotralstat 500 mg	0	14	0
other	Arthropod bite	Injury, poisoning and procedural complications	Part 3: Berotralstat 250 mg	1	11	1
other	Arthropod bite	Injury, poisoning and procedural complications	Parts 1, 2 and 3: Placebo	1	53	1
other	Fall	Injury, poisoning and procedural complications	Part1: Berotralstat 750 mg	0	33	0
other	Fall	Injury, poisoning and procedural complications	Part 2: Berotralstat 500 mg	0	14	0
other	Fall	Injury, poisoning and procedural complications	Part 3: Berotralstat 250 mg	1	11	1
other	Fall	Injury, poisoning and procedural complications	Parts 1, 2 and 3: Placebo	0	53	0
other	Limb injury	Injury, poisoning and procedural complications	Part1: Berotralstat 750 mg	1	33	1
other	Limb injury	Injury, poisoning and procedural complications	Part 2: Berotralstat 500 mg	1	14	1

1 to 10 of 128Source ClinicalTrials.gov

See all 128 adverse events

Adverse event totals12

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Part1: Berotralstat 750 mg	0	33
Total, all-cause mortality	Part 2: Berotralstat 500 mg	0	14
Total, all-cause mortality	Part 3: Berotralstat 250 mg	0	11
Total, all-cause mortality	Parts 1, 2 and 3: Placebo	0	53
Total, other adverse events	Part1: Berotralstat 750 mg	12	33
Total, other adverse events	Part 2: Berotralstat 500 mg	8	14
Total, other adverse events	Part 3: Berotralstat 250 mg	7	11
Total, other adverse events	Parts 1, 2 and 3: Placebo	17	53
Total, serious adverse events	Part1: Berotralstat 750 mg	0	33
Total, serious adverse events	Part 2: Berotralstat 500 mg	1	14

1 to 10 of 12Source ClinicalTrials.gov

See all 12 adverse event totals

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
36408587	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03240133

GET https://conductscience.org/api/trials/v1/NCT03240133/events?page=2

API reference For AI assistants