Dose-ranging Study of Oral PHA-022121 for Acute Treatment... | NCT04618211

NCT04618211

ID	Type	Description	Link
2020-003445-11	EudraCT Number

Status Module

Description Module

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Low dose/placebo	Other	Single low dose of deucrictibant or placebo	Drug: Deucrictibant Drug: Placebo
Medium dose/placebo	Other	Single medium dose of deucrictibant or placebo	Drug: Deucrictibant Drug: Placebo
High dose/placebo	Other	Single high dose of deucrictibant or placebo	Drug: Deucrictibant Drug: Placebo

Name	Type	Description	Arm Group Labels	Other Names
Deucrictibant	Drug	deucrictibant soft capsules for oral use	High dose/placebo Low dose/placebo Medium dose/placebo

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Change of the 3-symptom Composite Visual Analogue Scale (VAS-3) Score From Pre-treatment to 4 Hours Post-treatment	The primary endpoint of the study was the change of the VAS-3 (3-symptom composite visual analogue scale) score from pre-treatment to 4 hours post-treatment. The VAS-3 was calculated as the mean of the VAS scores of the 3 major HAE symptoms: skin swelling, skin pain, and abdominal pain. The VAS scores of the 3 major HAE symptoms (skin swelling, skin pain, and abdominal pain) could range between 0 (No swelling/No pain) and 100 (Extreme swelling/Excruciating pain)	Assessed from pre-treatment to 4 hours post-treatment

Secondary Outcomes

Measure	Description	Time Frame
Time to Onset of Symptom Relief by ≥30% Reduction in Visual Analogue Scale (VAS-3) Composite Score From the Pre-treatment Score	VAS-3 scores range between 0 and 100.	Assessed from pre-treatment to 48-hours post-treatment
Time to Onset of Almost Complete or Complete Symptom Relief by Visual Analogue Scale (VAS-3)

Eligibility Module

Name	Affiliation	Role
Marcus Maurer, Prof MD	Charite University, Berlin, Germany	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Study site		Birmingham	Alabama	35209	United States
Study site

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
41865747	Derived	Maurer M, Stobiecki M, Valerieva A, Hakl R, Staevska MT, Bouillet L, Du-Thanh A, Kessel A, Kiani-Alikhan S, Magerl M, Reshef A, Baeza ML, Fain O, Farkas H, Greve J, Guilarte M, Jacobs JS, Li HH, Lleonart R, Manning ME, Sussman GL, Anderson J, Chapdelaine H, Cohn DM, Hagin D, Kralickova P, Ritchie B, Spadaro G, Staubach P, Tarzi MD, Yang WH, Jouvin MH, Crabbe R, van Leeuwen S, Chen H, Zhu L, Knolle J, Lesage A, Lu P, Riedl MA, Aygoren-Pursun E. Oral deucrictibant for on-demand treatment of hereditary angioedema attacks (RAPIDe-1): a randomised, double-blind, placebo-controlled, phase 2 trial. Lancet Haematol. 2026 Apr;13(4):e200-e214. doi: 10.1016/S2352-3026(25)00341-2. Epub 2026 Mar 19.

Results Section

Participant Flow Module

Arm/Group Information

ID	Title	Description
FG000	PHA-022121 Low Dose - 10 mg	Part I: Single low dose of PHA-022121 (10 mg), non-attack state. Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design, administered in the following sequence: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Participants in Part I assigned to the Low Dose cohort remain in the Low Dose cohort in Part II.

Baseline Characteristics Module

Outcome Measures Module

Adverse Events Module

More Info Module

Type	Includes Protocol	Includes SAP	Includes ICF	Document Label	Document Date	Document Uploaded Date	Document File Name
Prot	Yes	No	No	Study Protocol	Apr 25, 2022	Jun 13, 2025

Baseline Measures

Title

Description

Population Description

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Denominator Units Selected

Denominators

Classes

Age, Continuous

Mean

Standard Deviation

years

Title

Denominators

Categories

Title	Measurements
	BG00042.9± 14.15 BG00145.7± 13.89 BG00241.4± 15.38 BG003

Age, Customized

Count of Participants

Participants

Title

Denominators

Categories

Adults (18-64 years)

Title	Measurements
	BG00022 BG00124 BG002

Sex: Female, Male

Count of Participants

Participants

Title

Denominators

Categories

Title	Measurements
Female	BG00017 BG00117 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

White

Title	Measurements
	BG00022 BG00124 BG002

Race/Ethnicity, Customized

Count of Participants

Participants

Title

Denominators

Categories

Hispanic or Latino

Title	Measurements
	BG0003 BG0012 BG002

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Change of the 3-symptom Composite Visual Analogue Scale (VAS-3) Score From Pre-treatment to 4 Hours Post-treatment

The primary endpoint of the study was the change of the VAS-3 (3-symptom composite visual analogue scale) score from pre-treatment to 4 hours post-treatment. The VAS-3 was calculated as the mean of the VAS scores of the 3 major HAE symptoms: skin swelling, skin pain, and abdominal pain. The VAS scores of the 3 major HAE symptoms (skin swelling, skin pain, and abdominal pain) could range between 0 (No swelling/No pain) and 100 (Extreme swelling/Excruciating pain)

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Least Squares Mean

Standard Error

Score on a scale

Assessed from pre-treatment to 4 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.
OG003	Placebo	Part II: 2 single high doses of PHA-022121 (10, 20, or 30 mg) and one single dose of placebo in a 3-way crossover design: PHA-022121 dose / PHA-022121 dose / Placebo PHA-022121 dose / Placebo / PHA-022121 dose Placebo / PHA-022121 dose / PHA-022121 dose Part II efficacy data utilized for the analysis.

Units	Counts
Participants	OG00021 OG00116 OG00220 OG003

Title

Denominators

Categories

Title	Measurements
	OG000-14.83± 1.833 OG001-13.10± 2.111 OG002-14.37± 1.975 OG003

Group IDs	Group Description	Statistical Method	Statistical Comment	P-Value	P-Value Comment	Parameter Type	Parameter Value	Dispersion Type	Dispersion Value	Confidence Interval Sides	Confidence Interval %	CI Lower Limit	CI Upper Limit	CI Lower Limit Comment	CI Upper Limit Comment	Estimate Comment	Tested Non-Inferiority	Non-Inferiority Type	Non-Inferiority Comment	Other Analysis Description
OG000 OG003		Mixed model repeated measures		< 0.0001	Nominal p-value	Least squares mean difference	-16.75	Standard Error of the Mean	2.423	2-Sided	95	-21.52	-11.97					Superiority
OG001 OG003		Mixed model repeated measures

Secondary

Time to Onset of Symptom Relief by ≥30% Reduction in Visual Analogue Scale (VAS-3) Composite Score From the Pre-treatment Score

VAS-3 scores range between 0 and 100.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

95% Confidence Interval

Hours

Assessed from pre-treatment to 48-hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose -10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Time to Onset of Almost Complete or Complete Symptom Relief by Visual Analogue Scale (VAS-3)

VAS scores range between 0 and 100. Almost complete symptom relief is defined as all 3 individual VAS scores of the VAS-3 having a value < 10. Complete symptom relief is defined as all 3 individual VAS scores are of the VAS-3 having a value of 0.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

95% Confidence Interval

Hours

Assessed from pre-treatment to 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Time to a ≥50% Reduction in VAS-3 Composite Score From the Pre-treatment Score

Time to a ≥50% reduction in VAS-3 composite score from the pre-treatment score.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

95% Confidence Interval

Hours

Assessed from pre-treatment to 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Change in the Mean Symptom Complex Severity (MSCS) Score From Pre-treatment to 4 Hours Post-treatment

MSCS scores range between 0 and 3. A higher score means a worse outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Least Squares Mean

95% Confidence Interval

Score on a scale

Pre-treatment and 4 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Treatment Outcome Score (TOS) at 4 Hours Post-treatment

TOS range between -100 and 100. A positive score indicates improvement, a score of 0 indicates no change, and a negative score indicates worsening compared to pre-treatment.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Least Squares Mean

95% Confidence Interval

Score on a scale

4 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Time to Onset of Primary Symptom Relief Assessed by a 30% Reduction in the VAS for the Primary Symptom

Time to onset of primary symptom relief assessed by a 30% reduction in the VAS for the primary symptom within 48 hours post-treatment

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

95% Confidence Interval

Hours

Within 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 12 Hours.

Proportion of blinded study drug treated attacks requiring HAE rescue medication within 12 hours post-treatment.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Number

Attacks requiring HAE rescue medication

Assessed at 12 hours post study drug treatment

Number of Treated Attacks

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Time to First HAE Rescue Medication Use for Study Drug-treated Attacks Within 48 Hours Post-treatment

The proportion of treated attacks with first use of HAE rescue medication within 48 hours post-treatment with PHA-022121.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point. Medians are as follows: Low Dose 10 mg - NA (

Posted

Median

95% Confidence Interval

Hours

Assessed at 48 hours post study drug treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Time to Change in the VAS Score for Skin Pain Score - 30% Reduction

Time to change in the VAS score for Skin Pain Score - 30% reduction within 48-hours post-treatment. VAS-3 scores range between 0 and 100. A larger reduction means a better outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

Inter-Quartile Range

Hours

Within 48 hours post treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Change in Mean Symptom Complex Severity Score From Pre-treatment to 24 Hours Post-treatment

Change in the Mean Symptom Complex Severity (MSCS) score from pre-treatment to 24 hours post-treatment. A lower MSCS score indicates a better outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Least Squares Mean

Standard Error

Score on a scale

24 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

TOS at 24 Hours Post-treatment

Treatment outcome score at 24 hours post-treatment. A higher score indicates improvement.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Mean

Standard Deviation

Score on a scale

Assessed within 24 hours post treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Part II efficacy data utilized for the analysis.

Secondary

Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Effectiveness Domain Score

MMRM analysis of Treatment Satisfaction Questionnaire for Medication (TSQM) at 48 hours post-treatment. The 11-item TSQM (version II) evaluated participant treatment satisfaction with the medication for the following scales: effectiveness, side effects, convenience, and overall satisfaction Scale scores were transformed into scores ranging from 0 to 100 and could be used to calculate a total composite score, a higher score indicating greater satisfaction.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Mean

Standard Deviation

Score on a scale

48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.

Secondary

Time to Onset of Primary Symptom Relief by 50% Reduction in VAS Score

Time to Onset of Primary Symptom Relief by 50% Reduction in VAS Score within 48 hours post-treatment. VAS-3 scores range between 0 and 100. A larger reduction means a better outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

95% Confidence Interval

Hours

Within 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 24 Hours.

Qualifying attacks treated with study drug may use approved rescue medication if no symptom relief within 4 hours has been experienced.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Number

Attacks requiring HAE rescue medication

Assessed at 24 hours post-study drug treatment

Number of Treated Attacks

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 48 Hours

Qualifying attacks treated with study drug may use approved rescue medication if no symptom relief within 4 hours has been experienced.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Number

Attacks requiring HAE rescue medication

Assessed at 48 hours post-study drug treatment

Number of Treated Attacks

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Convenience Domain Score

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Mean

Standard Deviation

Score on a scale

48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.

Secondary

Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Satisfaction Domain Score

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Mean

Standard Deviation

Score on a scale

48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.

Secondary

Time to Change in the VAS Score for Skin Swelling Score - 30% Reduction

Time to change in the VAS score for Skin Swelling Score - 30% reduction within 48 hours post-treatment. VAS-3 scores range between 0 and 100. A larger reduction means a better outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

Inter-Quartile Range

Hours

Within 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Secondary

Time to Change in the VAS Score for Abdominal Pain Score - 30% Reduction

Time to change in the VAS score for Abdominal Pain Score - 30% reduction within 48 hours post-treatment. VAS-3 scores range between 0 and 100. A larger reduction means a better outcome.

Part I efficacy data not collected, consistent with and pre-specified in the study protocol. Includes all participants with evaluable data who completed the relevant assessment time point.

Posted

Median

Inter-Quartile Range

Hours

Within 48 hours post-treatment

ID	Title	Description
OG000	PHA-022121 Low Dose - 10 mg	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Part II efficacy data utilized for the analysis.
OG001	PHA-022121 Medium Dose - 20 mg	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Part II efficacy data utilized for the analysis.
OG002	PHA-022121 High Dose - 30 mg

Arm/Groups

ID	Title	Description	Deaths (At Risk)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Part I PHA-022121 Low Dose - 10 mg	Part I: Single low dose of PHA-022121 (10 mg), non-attack state.	23	23	2	23
EG001	Part I PHA-022121 Medium Dose - 20 mg	Part I: Single medium dose of PHA-022121 (20 mg), non-attack state.	24	24	2	24
EG002	Part I PHA-022121 High Dose - 30 mg	Part I: Single high dose of PHA-022121 (30 mg), non-attack state.	25	25	4	25
EG003	Part II PHA-022121 Low Dose - 10 mg Study Drug Treated Attacks	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Number at risk reflects number of study drug treated attacks.	38	38	3	38
EG004	Part II PHA-022121 Medium Dose - 20 mg Study Drug Treated Attacks	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Number at risk reflects number of study drug treated attacks.	29	29	1	29
EG005	Part II PHA-022121 High Dose - 30 mg Study Drug Treated Attacks	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Number at risk reflects number of study drug treated attacks.	36	36	2	36
EG006	Part II PHA-022121 Low Dose - 10 mg Participants	Part II: 2 single low doses of PHA-022121 (10 mg) and one single dose of placebo in a 3-way crossover design: Low dose / Low dose / Placebo Low dose / Placebo / Low dose Placebo / Low dose / Low dose Number at risk reflects number of participants at risk.	21	21	3	21
EG007	Part II PHA-022121 Medium Dose - 20 mg Participants	Part II: 2 single medium doses of PHA-022121 (20 mg) and one single dose of placebo in a 3-way crossover design: Medium dose / Medium dose / Placebo Medium dose / Placebo / Medium dose Placebo / Medium dose / Medium dose Number at risk reflects number of participants at risk.	16	16	1	16
EG008	Part II PHA-022121 High Dose - 30 mg Participants	Part II: 2 single high doses of PHA-022121 (30 mg) and one single dose of placebo in a 3-way crossover design: High dose / High dose / Placebo High dose / Placebo / High dose Placebo / High dose / High dose Number at risk reflects number of participants at risk.	22	22	2	22
EG009	Part II Placebo	Part II: 2 single high doses of PHA-022121 (10, 20, or 30 mg) and one single dose of placebo in a 3-way crossover design: PHA-022121 dose / PHA-022121 dose / Placebo PHA-022121 dose / Placebo / PHA-022121 dose Placebo / PHA-022121 dose / PHA-022121 dose	53	53	4	53

Other Adverse Events

Term	Organ System	Source Vocabulary	Assessment Type	Statistical Information
Blood bilirubin increased	Investigations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG0030 events0 affected38 at risk EG0040 events0 affected29 at risk EG0051 events1 affected36 at risk EG0060 events0 affected21 at risk EG0070 events0 affected16 at risk EG0081 events1 affected22 at risk EG0090 events0 affected53 at risk
Headache	Nervous system disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0011 events1 affected24 at risk EG0022 events2 affected25 at risk EG003
Fatigue	General disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Nausea	Gastrointestinal disorders	MedDRA (25.0)	Non-systematic Assessment	EG0001 events1 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Vomiting	Gastrointestinal disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Lower urinary tract symptoms	Renal and urinary disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Gastrointestinal infection	Infections and infestations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Nasopharyngitis	Infections and infestations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0022 events2 affected25 at risk EG003
Dental caries	Gastrointestinal disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0011 events1 affected24 at risk EG0020 events0 affected25 at risk EG003
Viral Infection	Infections and infestations	MedDRA (25.0)	Non-systematic Assessment	EG0001 events1 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Sinusitis	Infections and infestations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Blister	Skin and subcutaneous tissue disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Mean cell hemoglobin decreased	Investigations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Mean cell volume decreased	Investigations	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0020 events0 affected25 at risk EG003
Arthralgia	Musculoskeletal and connective tissue disorders	MedDRA (25.0)	Non-systematic Assessment	EG0000 events0 affected23 at risk EG0010 events0 affected24 at risk EG0021 events1 affected25 at risk EG003

ID	Term
D054179	Angioedemas, Hereditary
D056829	Hereditary Angioedema Types I and II

ID	Term
D000799	Angioedema
D014652	Vascular Diseases
D002318	Cardiovascular Diseases
D000081208	Hereditary Complement Deficiency Diseases
D000081207	Primary Immunodeficiency Diseases
D030342	Genetic Diseases, Inborn
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D014581	Urticaria
D017445	Skin Diseases, Vascular
D012871	Skin Diseases
D017437	Skin and Connective Tissue Diseases
D006969	Hypersensitivity, Immediate
D006967	Hypersensitivity
D007154	Immune System Diseases
D007153	Immunologic Deficiency Syndromes

ID	Title	Description
BG000	PHA-022121 Low Dose - 10 mg	Part I: Single low dose of PHA-022121 (10 mg), non-attack state.
BG001	PHA-022121 Medium Dose - 20 mg	Part I: Single medium dose of PHA-022121 (20 mg), non-attack state.
BG002	PHA-022121 High Dose - 30 mg	Part I: Single high dose of PHA-022121 (30 mg), non-attack state.
BG003	Total	Total of all reporting groups