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Phase 2 N=74 Randomized Quadruple-blind Treatment

Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema

Hereditary Angioedema · Hereditary Angioedema Type I · Hereditary Angioedema Type II · Hereditary Angioedema Types I and II · Hereditary Angioedema Attack

Enrolled (actual)
74
Serious AEs
0.0%
Results posted
Dec 2025
Primary outcome: Primary: Change of the 3-symptom Composite Visual Analogue Scale (VAS-3) Score From Pre-treatment to 4 Hours Post-treatment — -14.83; -13.10; -14.37; 1.92 Score on a scale — p=< 0.0001

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Deucrictibant (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Pharvaris Netherlands B.V.
Primary completion
Sep 2022

Outcome Measures

OutcomeResultp-value
PRIMARY
Change of the 3-symptom Composite Visual Analogue Scale (VAS-3) Score From Pre-treatment to 4 Hours Post-treatment
-14.83; -13.10; -14.37; 1.92 < 0.0001 sig
SECONDARY
Time to Onset of Symptom Relief by ≥30% Reduction in Visual Analogue Scale (VAS-3) Composite Score From the Pre-treatment Score
2.1; 2.7; 2.5; 8.0 < 0.0001 sig
SECONDARY
Time to Onset of Almost Complete or Complete Symptom Relief by Visual Analogue Scale (VAS-3)
5.8; 20.0; 20.0; 42.0 < 0.0001 sig
SECONDARY
Time to a ≥50% Reduction in VAS-3 Composite Score From the Pre-treatment Score
3.3; 4.0; 4.0; 22.8 < 0.0001 sig
SECONDARY
Change in the Mean Symptom Complex Severity (MSCS) Score From Pre-treatment to 4 Hours Post-treatment
-1.08; -0.91; -0.68; -0.29 < 0.0001 sig
SECONDARY
Treatment Outcome Score (TOS) at 4 Hours Post-treatment
60.52; 59.08; 67.44; -3.62 < 0.0001 sig
SECONDARY
Time to Onset of Primary Symptom Relief Assessed by a 30% Reduction in the VAS for the Primary Symptom
2.0; 3.0; 2.9; 23.3
SECONDARY
Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 12 Hours.
7; 3; 2; 31
SECONDARY
Time to First HAE Rescue Medication Use for Study Drug-treated Attacks Within 48 Hours Post-treatment
NA; NA; NA; 6.0
SECONDARY
Time to Change in the VAS Score for Skin Pain Score - 30% Reduction
2.9; 3.4; 2.7; 22.8
SECONDARY
Change in Mean Symptom Complex Severity Score From Pre-treatment to 24 Hours Post-treatment
-1.65; -1.50; -1.68; -1.20
SECONDARY
TOS at 24 Hours Post-treatment
94.44; 93.60; 94.44; 76.92
SECONDARY
Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Effectiveness Domain Score
79.17; 70.45; 72.92; 65.38
SECONDARY
Time to Onset of Primary Symptom Relief by 50% Reduction in VAS Score
2.9; 4.5; 4.0; 22.8
SECONDARY
Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 24 Hours.
37; 28; 31; 51; 9; 3
SECONDARY
Proportion of Study Drug Treated Attacks Requiring HAE Rescue Medication Within 48 Hours
37; 28; 31; 51; 9; 4
SECONDARY
Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Convenience Domain Score
80.56; 83.08; 77.55; 76.07
SECONDARY
Treatment Satisfaction Questionnaire for Medication Scores at 48 Hours Post-treatment - Satisfaction Domain Score
82.50; 80.68; 76.74; 64.10
SECONDARY
Time to Change in the VAS Score for Skin Swelling Score - 30% Reduction
2.5; 3.5; 3.8; 23.3
SECONDARY
Time to Change in the VAS Score for Abdominal Pain Score - 30% Reduction
1.9; 1.4; 2.9; 20.0

Summary

This study evaluates the efficacy of orally administered deucrictibant for the acute treatment of attacks in patients with hereditary angioedema (HAE). Eligible subjects are randomized to one of three single doses of deucrictibant and placebo. The study will compare symptom relief (skin pain, skin swelling, abdominal pain) during HAE attacks and safety of each dose of deucrictibant with placebo.

Eligibility Criteria

Key Inclusion Criteria

  • Signed and dated informed consent form
  • Diagnosis of HAE type I or II
  • Documented history of HAE attacks: at least three in the last 4 months, or at least two in the last 2 months prior to screening
  • Reliable access and experience to use standard of care acute attack medications

Key Exclusion Criteria

  • Pregnancy or breast-feeding
  • Clinically significant abnormal electrocardiogram
  • Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study
  • Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrollment
  • Positive serology for HIV or active infection with hepatitis B virus or hepatitis C virus
  • Abnormal hepatic function
  • Abnormal renal function
  • History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
  • History of documented severe hypersensitivity to any medicinal product
  • Participation in any other investigational drug study within defined period
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT04618211). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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