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NCT Number: NCT04618211

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

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.

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Key information

Conditions

Hereditary Angioedema Angioedema Angioedemas, Hereditary C1 Esterase Inhibitor Deficiency C1 Inhibitor Deficiency Cardiovascular Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hereditary Angioedema - Type 1 Hereditary Angioedema - Type 2 Hereditary Angioedema Attack Hereditary Angioedema Type I Hereditary Angioedema Type II Hereditary Angioedema Types I and II Hereditary Angioedema With C1 Esterase Inhibitor Deficiency Hereditary Complement Deficiency Diseases Hypersensitivity Hypersensitivity, Immediate Immune System Diseases Immunologic Deficiency Syndromes Primary Immunodeficiency Diseases Skin Diseases Skin Diseases, Vascular Skin and Connective Tissue Diseases Urticaria Vascular Diseases

Age range

18 year–75 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2

Primary location

Study site, Sofia, Bulgaria

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About this study

In Part I of the study, patients in non-attack state receive the assigned active single dose of deucrictibant at the study center to assess pharmacokinetics (the way the body absorbs, distributes, and gets rid of the drug) and safety. In Part II of the study, patients self-administer blinded study drug at home to treat three HAE attacks with deucrictibant or placebo (cross-over).

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

deucrictibant

Drug

deucrictibant soft capsules for oral use

Other names: PHA-022121, PHA121, PHVS416

Placebo

Drug

Matching placebo capsules for oral use

Primary outcomes

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

    Time frame: Assessed 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)

Secondary outcomes

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

    Time frame: Assessed from pre-treatment to 48-hours post-treatment

    VAS-3 scores range between 0 and 100.

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

    Time frame: Assessed from pre-treatment to 48 hours post-treatment

    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.

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

    Time frame: Assessed from pre-treatment to 48 hours post-treatment

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

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

    Time frame: Pre-treatment and 4 hours post-treatment

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

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

    Time frame: 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.

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

    Time frame: Within 48 hours post-treatment

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

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

    Time frame: Assessed at 12 hours post study drug treatment

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

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

    Time frame: Assessed at 48 hours post study drug treatment

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

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

    Time frame: Within 48 hours post treatment

    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.

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

    Time frame: 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.

  11. TOS at 24 Hours Post-treatment

    Time frame: Assessed within 24 hours post treatment

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

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

    Time frame: 48 hours post-treatment

    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.

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

    Time frame: Within 48 hours post-treatment

    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.

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

    Time frame: Assessed at 24 hours post-study drug treatment

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

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

    Time frame: Assessed at 48 hours post-study drug treatment

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

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

    Time frame: 48 hours post-treatment

    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.

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

    Time frame: 48 hours post-treatment

    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.

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

    Time frame: Within 48 hours post-treatment

    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.

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

    Time frame: Within 48 hours post-treatment

    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.

Sponsors and collaborators

Lead sponsor

Pharvaris Netherlands B.V.

Industry

Registry information

Official study title

A Phase II, Double-blind, Placebo-controlled, Randomized, Cross-over, Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-inhibitor Deficiency Type I and II

Acronym: RAPIDe-1

Important dates

Study start
2021
Primary completion
2022
Study completion
2023
First posted
Nov 5, 2020
Registry last updated
Dec 17, 2025

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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