NCT05047185

Brief Summary

This study evaluates the safety and efficacy of PHA-022121 administered orally for prophylaxis against angioedema attacks in patients with hereditary angioedema (HAE). The study consists of 2 parts, with patients completing participation in Part 1 prior to initiation of treatment in Part 2. Part 1 of the study has 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. Patients will continue to the single open-label arm in Part 2 of the study after completion of Part 1. The screening period is up to 8 weeks and the treatment periods are 12 weeks (Part 1) and 30 months (Part 2) in duration.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
34

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Apr 2022

Typical duration for phase_2

Geographic Reach
9 countries

19 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

September 7, 2021

Completed
10 days until next milestone

First Posted

Study publicly available on registry

September 17, 2021

Completed
7 months until next milestone

Study Start

First participant enrolled

April 19, 2022

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 30, 2025

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 30, 2025

Completed
Last Updated

July 30, 2025

Status Verified

July 1, 2025

Enrollment Period

3.2 years

First QC Date

September 7, 2021

Last Update Submit

July 29, 2025

Conditions

Hereditary AngioedemaHereditary Angioedema Type IHereditary Angioedema Type IIHereditary Angioedema Types I and IIHereditary Angioedema AttackHereditary Angioedema With C1 Esterase Inhibitor DeficiencyHereditary Angioedema - Type 1Hereditary Angioedema - Type 2C1 Esterase Inhibitor DeficiencyC1 Inhibitor Deficiency

Keywords

HAEHAE Type IHAE Type IIOral TreatmentBradykinin B2 Receptor AntagonistPHVS416PHA121ProphylaxisDeucrictibant

Outcome Measures

Primary Outcomes (1)

  • Number of investigator-confirmed HAE attacks

    Day 0 to Day 84

Secondary Outcomes (12)

  • Number of investigator-confirmed moderate or severe HAE attacks during the treatment period

    Day 0 to Day 84

  • Number of investigator-confirmed HAE attacks requiring acute treatment during the treatment period

    Day 0 to Day 84

  • Number of patients achieving reduction in attack rate during the treatment period relative to baseline

    Day 0 to Day 84

  • Number of patients that are attack-free during the treatment period

    Day 0 to Day 84

  • Number and proportion of days with angioedema symptoms during the treatment period

    Day 0 to Day 84

  • +7 more secondary outcomes

Study Arms (4)

Part 1: Low dose

EXPERIMENTAL

BID low dose of deucrictibant

Drug: Deucrictibant low dose

Part 1: High dose

EXPERIMENTAL

BID high dose of deucrictibant

Drug: Deucrictibant high dose

Part 1: Placebo

PLACEBO COMPARATOR

BID placebo

Drug: Placebo

Part 2: Open-label

EXPERIMENTAL

BID high dose of deucrictibant

Drug: Deucrictibant high dose

Interventions

Deucrictibant low doseDRUG

Deucrictibant softgel capsules for oral use (PHVS416)

Part 1: Low dose
Deucrictibant high doseDRUG

Deucrictibant softgel capsules for oral use (PHVS416)

Part 1: High dosePart 2: Open-label
PlaceboDRUG

Matching placebo capsules for oral use

Part 1: Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • Signed and dated informed consent form
  • Diagnosis of HAE type I or II
  • Documented history of at least 3 HAE attacks within the last 3 consecutive months prior to screening, or a minimum of 2 HAE attacks during the screening period
  • Reliable access and experience to use standard of care acute attack medications

You may not qualify if:

  • 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 enrolment
  • Abnormal hepatic function
  • Abnormal renal function
  • History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
  • Participation in any other investigational drug study within defined period

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (19)

Study site

Birmingham, Alabama, 35209, United States

Location

Study site

Paradise Valley, Arizona, 85253, United States

Location

Study site

St Louis, Missouri, 63141, United States

Location

Study Site

Vienna, A-1090, Austria

Location

Study site

Sofia, 1680, Bulgaria

Location

Study site

Ottawa, Ontario, K1H 1E4, Canada

Location

Study site

Montreal, Quebec, H2W 1R7, Canada

Location

Study site

Berlin, 10117, Germany

Location

Study site

Frankfurt, 60323, Germany

Location

Study site

Dublin, D08NHY1, Ireland

Location

Study site

Padua, PD, 35128, Italy

Location

Study site

Milan, 20157, Italy

Location

Study site

Palermo, 0146, Italy

Location

Study site

Krakow, Poland

Location

Study site

Brighton, England, BN2 1ES, United Kingdom

Location

Study site

Bristol, England, BS10 5NB, United Kingdom

Location

Study site

Cambridge, England, CB2 0QQ, United Kingdom

Location

Study site

London, England, E1 1FR, United Kingdom

Location

Study site

Southampton, England, SO16 6YD, United Kingdom

Location

MeSH Terms

Conditions

Angioedemas, HereditaryHereditary Angioedema Types I and II

Condition Hierarchy (Ancestors)

AngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Study Officials

  • Marc Riedl, MD

    UC San Diego, La Jolla, California, United States

    PRINCIPAL INVESTIGATOR

  • Emel Aygören-Pürsün, MD

    University Hospital Frankfurt - Goethe University, Frankfurt, Germany

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
QUADRUPLE
Who Masked
PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
PREVENTION
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

September 7, 2021

First Posted

September 17, 2021

Study Start

April 19, 2022

Primary Completion

June 30, 2025

Study Completion

June 30, 2025

Last Updated

July 30, 2025

Record last verified: 2025-07

Data Sharing

IPD Sharing
Will not share

Locations

BU(1)US(3)AU(1)DE(2)PL(1)GB(5)IT(3)CA(2)IE(1)