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Dose-ranging Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema (RAPIDe-1)

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ClinicalTrials.gov Identifier: NCT04618211
Recruitment Status : Recruiting
First Posted : November 5, 2020
Last Update Posted : July 14, 2021
Sponsor:
Information provided by (Responsible Party):
Pharvaris Netherlands B.V.

Brief Summary:
This study evaluates the efficacy of orally administered PHA-022121 for the acute treatment of attacks in patients with hereditary angioedema (HAE). Eligible subjects are randomized to one of three single doses of PHA-022121 and placebo. The study will compare symptom relief (skin pain, skin swelling, abdominal pain) during HAE attacks and safety of each dose of PHA-022121 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

Detailed Description:
In Part I of the study, patients in non-attack state receive the assigned active single dose of PHA-022121 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 PHA-022121 or placebo (cross-over).

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 54 participants
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official 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
Actual Study Start Date : February 3, 2021
Estimated Primary Completion Date : September 2022
Estimated Study Completion Date : September 2022


Arm Intervention/treatment
Low dose/placebo
Single low dose of PHA-022121 or placebo
Drug: PHA-022121
PHA-022121 soft capsules for oral use (PHVS416)

Drug: Placebo
Matching placebo capsules for oral use

Medium dose/placebo
Single medium dose of PHA-022121 or placebo
Drug: PHA-022121
PHA-022121 soft capsules for oral use (PHVS416)

Drug: Placebo
Matching placebo capsules for oral use

High dose/placebo
Single high dose of PHA-022121 or placebo
Drug: PHA-022121
PHA-022121 soft capsules for oral use (PHVS416)

Drug: Placebo
Matching placebo capsules for oral use




Primary Outcome Measures :
  1. Change of the 3-symptom composite visual analogue scale (VAS-3) score from pre-treatment to 4 hours post-treatment [ Time Frame: Pre-treatment and 4 hours post-treatment ]
    VAS-3 scores range between 0 and 100. A larger reduction means a better outcome.


Secondary Outcome Measures :
  1. Time to onset of symptom relief by visual analogue scale (VAS-3) score [ Time Frame: Assessed from pre-treatment to 48 hours post-treatment ]
    VAS-3 scores range between 0 and 100. Symptom relief is defined as a 50% or higher reduction of the VAS-3 score.

  2. Time to onset of primary symptom relief by visual analogue scale (VAS) [ Time Frame: Assessed from pre-treatment to 48 hours post-treatment ]
    VAS scores range between 0 and 100. Primary symptom relief is defined as any VAS score reduction of the primary symptom above the threshold.

  3. Mean symptom complex severity (MSCS) score [ Time Frame: 4 hours post-treatment ]
    MSCS scores range between 0 and 3. A higher score means a worse outcome.

  4. Treatment outcome score (TOS) [ Time Frame: Pre-treatment and 4 hours post-treatment ]
    TOS scores 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.

  5. Treatment satisfaction questionnaire for medication (TSQM) scores [ Time Frame: 48 hours post-treatment ]
    TSQM scores range from 0 to 100. A higher score means a better outcome.

  6. Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From post-dose non-attack visit through study completion, approximately 26 weeks ]
  7. Occurrence of treatment-related adverse events (AEs) [ Time Frame: From post-dose non-attack visit through study completion, approximately 26 weeks ]
  8. Occurrence of treatment-emergent serious adverse events (TESAEs) [ Time Frame: From post-dose non-attack visit through study completion, approximately 26 weeks ]


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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

Key Inclusion Criteria:

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

Key Exclusion Criteria:

  1. Pregnancy or breast-feeding
  2. Clinically significant abnormal electrocardiogram
  3. Any other systemic disease or significant disease or disorder that would interfere with the patient's safety or ability to participate in the study
  4. Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrollment
  5. Positive serology for HIV or active infection with hepatitis B virus or hepatitis C virus
  6. Abnormal hepatic function
  7. Abnormal renal function
  8. History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
  9. History of documented severe hypersensitivity to any medicinal product
  10. 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): NCT04618211


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

Locations
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Bulgaria
Study site Recruiting
Sofia, Bulgaria
Canada, Alberta
Study site Recruiting
Edmonton, Alberta, Canada
Canada, Ontario
Study site Recruiting
Ottawa, Ontario, Canada
Study site Recruiting
Toronto, Ontario, Canada
Canada, Quebec
Study site Recruiting
Montréal, Quebec, Canada
Study site Recruiting
Québec City, Quebec, Canada
Czechia
Study site Recruiting
Brno, Czechia
Study site Recruiting
Hradec Králové, Czechia
France
Study site Recruiting
Grenoble, France
Study site Recruiting
Montpellier, France
Study site Recruiting
Paris, France
Germany
Study site Recruiting
Dresden, Germany
Study site Recruiting
Frankfurt, Germany
Study site Recruiting
Mainz, Germany
Study site Recruiting
Ulm, Germany
Hungary
Study site Recruiting
Budapest, Hungary
Israel
Study site Recruiting
Ashkelon, Israel
Italy
Study site Recruiting
Naples, Italy
Netherlands
Study site Recruiting
Amsterdam, Netherlands
Spain
Study site Recruiting
Barcelona, Spain
Study site Recruiting
Madrid, Spain
United Kingdom
Study site Recruiting
Brighton, United Kingdom
Sponsors and Collaborators
Pharvaris Netherlands B.V.
Investigators
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Principal Investigator: Marcus Maurer, Prof MD Charite University, Berlin, Germany
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Responsible Party: Pharvaris Netherlands B.V.
ClinicalTrials.gov Identifier: NCT04618211    
Other Study ID Numbers: PHA022121-C201
2020-003445-11 ( EudraCT Number )
First Posted: November 5, 2020    Key Record Dates
Last Update Posted: July 14, 2021
Last Verified: July 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
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 Antagonists
PHVS416
PHA121
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
Genetic Diseases, Inborn