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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 : December 6, 2021
Sponsor:
Information provided by (Responsible Party):
Pharvaris Netherlands B.V.

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 will have 3 parallel arms and approximately 30 patients will be equally randomized to one of two dose regimens of PHA-022121 or matching placebo. The screening period is up to 8 weeks and the treatment period is 12 weeks in duration. The study will assess the efficacy of PHA-022121 as prophylaxis against HAE attacks, and safety of each dose of PHA-022121 compared with placebo.

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 Drug: Placebo Phase 2

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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)
Estimated Study Start Date : December 2021
Estimated Primary Completion Date : November 2022
Estimated Study Completion Date : December 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Low dose
BID low dose of PHA-022121
Drug: PHA-022121
PHA-022121 softgel capsules for oral use (PHVS416)

High dose
BID high dose of PHA-022121
Drug: PHA-022121
PHA-022121 PHA-022121 softgel capsules for oral use (PHVS416)

Placebo
BID placebo
Drug: Placebo
Matching placebo capsules for oral use




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 ]


Information from the National Library of Medicine

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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

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 Recruiting
Birmingham, Alabama, United States, 35209
United States, California
Study site Recruiting
Santa Monica, California, United States, 90404
United States, Missouri
Study site Recruiting
Saint Louis, Missouri, United States, 63141
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
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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: December 6, 2021
Last Verified: December 2021
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
Vascular Diseases
Cardiovascular Diseases
Urticaria
Skin Diseases, Vascular
Skin Diseases
Hypersensitivity, Immediate
Hypersensitivity
Immune System Diseases
Hereditary Complement Deficiency Diseases
Primary Immunodeficiency Diseases
Genetic Diseases, Inborn
Immunologic Deficiency Syndromes