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Dose-ranging Study of Oral PHA-022121 for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Type I or Type II (HAE CHAPTER-1)

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ClinicalTrials.gov Identifier: NCT05047185
Recruitment Status : Recruiting
First Posted : September 17, 2021
Last Update Posted : February 23, 2023
Sponsor:
Information provided by (Responsible Party):
Pharvaris Netherlands B.V.

Study Description
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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 50 weeks (Part 2) in duration.

Condition or disease Intervention/treatment Phase
Hereditary Angioedema Hereditary Angioedema Type I Hereditary Angioedema Type II Hereditary Angioedema Types I and II Hereditary Angioedema Attack Hereditary Angioedema With C1 Esterase Inhibitor Deficiency Hereditary Angioedema - Type 1 Hereditary Angioedema - Type 2 C1 Esterase Inhibitor Deficiency C1 Inhibitor Deficiency Drug: PHA-022121 low dose Drug: PHA-022121 high dose Drug: Placebo Phase 2

Study Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 30 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Prevention
Official Title: A Phase II, Double-blind, Placebo-controlled, Randomized, Dose-ranging, Parallel Group Study to Evaluate the Safety and Efficacy of PHA-022121 Administered Orally for Prophylaxis Against Angioedema Attacks in Patients With Hereditary Angioedema Due to C1-Inhibitor Deficiency (Type I or Type II)
Actual Study Start Date : April 19, 2022
Estimated Primary Completion Date : December 2023
Estimated Study Completion Date : December 2024

Resource links provided by the National Library of Medicine


Arms and Interventions
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Arm Intervention/treatment
Experimental: Part 1: Low dose
BID low dose of PHA-022121
 Drug: PHA-022121 low dose
PHA-022121 softgel capsules for oral use (PHVS416)
Experimental: Part 1: High dose
BID high dose of PHA-022121
 Drug: PHA-022121 high dose
PHA-022121 softgel capsules for oral use (PHVS416)
Placebo Comparator: Part 1: Placebo
BID placebo
 Drug: Placebo
Matching placebo capsules for oral use
Experimental: Part 2: Open-label
BID high dose of PHA-022121
 Drug: PHA-022121 high dose
PHA-022121 softgel capsules for oral use (PHVS416)


Outcome Measures
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Primary Outcome Measures :
  1. Number of investigator-confirmed HAE attacks [ Time Frame: Day 0 to Day 84 ]

Secondary Outcome Measures :
  1. Number of investigator-confirmed moderate or severe HAE attacks during the treatment period [ Time Frame: Day 0 to Day 84 ]
  2. Number of investigator-confirmed HAE attacks requiring acute treatment during the treatment period [ Time Frame: Day 0 to Day 84 ]
  3. Number of patients achieving reduction in attack rate during the treatment period relative to baseline [ Time Frame: Day 0 to Day 84 ]
  4. Number of patients that are attack-free during the treatment period [ Time Frame: Day 0 to Day 84 ]
  5. Number and proportion of days with angioedema symptoms during the treatment period [ Time Frame: Day 0 to Day 84 ]
  6. Time to first investigator-confirmed HAE attack in the treatment period [ Time Frame: Day 0 to Day 84 ]
  7. Number of investigator-confirmed HAE attacks resulting in a visit to the emergency department or an admission to hospital [ Time Frame: Day 0 to Day 84 ]
  8. Number of investigator-confirmed angioedema attacks during the treatment period in Part 2. [ Time Frame: Day 84 to Day 252 ]
  9. Number of investigator-confirmed moderate or severe angioedema attacks during the treatment period in Part 2. [ Time Frame: Day 84 to Day 252 ]
  10. Number of investigator-confirmed angioedema attacks requiring acute treatment during the treatment period in Part 2. [ Time Frame: Day 84 to Day 252 ]
  11. Incidence of HAE attacks during the treatment period in Part 2 (attack rate trend over time). [ Time Frame: Day 84 to Day 252 ]
  12. Number and proportion of days with angioedema symptoms during the treatment period in Part 2. [ Time Frame: Day 84 to Day 252 ]

Eligibility Criteria
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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • 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

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 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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05047185


Contacts
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Contact: Pharvaris Clinical Team +31 (71) 203-6410 clinicaltrials@pharvaris.com

Locations
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United States, Alabama
Study site Active, not recruiting
Birmingham, Alabama, United States, 35209
United States, Arizona
Study site Active, not recruiting
Paradise Valley, Arizona, United States, 85253
United States, California
Study site Active, not recruiting
San Diego, California, United States, 92122
Study site Active, not recruiting
Santa Monica, California, United States, 90404
United States, Colorado
Study site Active, not recruiting
Colorado Springs, Colorado, United States, 80907
United States, Florida
Study site Active, not recruiting
Tampa, Florida, United States, 33613
United States, Michigan
Study site Active, not recruiting
Ann Arbor, Michigan, United States, 48106
United States, Missouri
Study site Active, not recruiting
Saint Louis, Missouri, United States, 63141
United States, New Jersey
Study site Active, not recruiting
Fair Lawn, New Jersey, United States, 07410
United States, Pennsylvania
Study site Active, not recruiting
Hershey, Pennsylvania, United States, 17033
Bulgaria
Study site Recruiting
Sofia, Bulgaria, 1431
Study site Recruiting
Sofia, Bulgaria, 1680
Canada, Ontario
Study site Recruiting
Ottawa, Ontario, Canada, K1H 1E4
Study site Recruiting
Toronto, Ontario, Canada, M3B 3S6
Canada, Quebec
Study site Recruiting
Montréal, Quebec, Canada, H2W 1R7
Germany
Study site Recruiting
Berlin, Germany, 10117
Study site Recruiting
Dresden, Germany, 01307
Study site Recruiting
Frankfurt, Germany, 60323
Israel
Study site Recruiting
Haifa, Israel, 31096
Study site Recruiting
Haifa, Israel
Study site Recruiting
Tel Aviv-Yafo, Israel
Italy
Study site Recruiting
Padua, PD, Italy, 35128
Poland
Study site Recruiting
Kraków, Poland
Spain
Study site Recruiting
Barcelona, Spain, 08907
United Kingdom
Study site Recruiting
Brighton, England, United Kingdom, BN2 1ES
Study site Recruiting
Bristol, England, United Kingdom, BS10 5NB
Study site Recruiting
London, United Kingdom, E1 1FR
Study site Recruiting
Manchester, United Kingdom, M13 9WL
Sponsors and Collaborators
Pharvaris Netherlands B.V.
Investigators
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Principal Investigator: Marc Riedl, MD UC San Diego, La Jolla, California, United States
Principal Investigator: Emel Aygören-Pürsün, MD University Hospital Frankfurt - Goethe University, Frankfurt, Germany
More Information
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Responsible Party: Pharvaris Netherlands B.V.
ClinicalTrials.gov Identifier: NCT05047185    
Other Study ID Numbers: PHA022121-C301
2021-000227-13 ( EudraCT Number )
First Posted: September 17, 2021    Key Record Dates
Last Update Posted: February 23, 2023
Last Verified: February 2023
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Pharvaris Netherlands B.V.:
HAE
HAE Type I
HAE Type II
Oral Treatment
 Bradykinin B2 Receptor Antagonist
PHVS416
PHA121
Prophylaxis
Additional relevant MeSH terms:
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Angioedema
Angioedemas, Hereditary
Hereditary Angioedema Types I and II
Hereditary Complement Deficiency Diseases
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
 Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes