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 : February 9, 2021
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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 |
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 |
- 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.
- 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.
- 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.
- 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.
- 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.
- 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.
- Occurrence of treatment-emergent adverse events (TEAEs) [ Time Frame: From post-dose non-attack visit through study completion, approximately 26 weeks ]
- Occurrence of treatment-related adverse events (AEs) [ Time Frame: From post-dose non-attack visit through study completion, approximately 26 weeks ]
- 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 |
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
- 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

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
Contact: Clinical Team Pharvaris | +31 (71) 203-6410 | clinical@pharvaris.com |
Canada, Alberta | |
Study site | Not yet recruiting |
Edmonton, Alberta, Canada | |
Canada, Ontario | |
Study site | Recruiting |
Ottawa, Ontario, Canada | |
Study site | Recruiting |
Toronto, Ontario, Canada | |
Canada, Quebec | |
Study site | Not yet recruiting |
Montréal, Quebec, Canada | |
Study site | Not yet recruiting |
Québec City, Quebec, Canada |
Principal Investigator: | Marcus Maurer, Prof MD | Charite University, Berlin, Germany |
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: | February 9, 2021 |
Last Verified: | February 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | No |
Studies a U.S. FDA-regulated Device Product: | No |
HAE HAE Type I HAE Type II Oral Treatment |
Bradykinin B2 Receptor Antagonists PHVS416 PHA121 |
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 |