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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05396105




Registration number
NCT05396105
Ethics application status
Date submitted
28/04/2022
Date registered
31/05/2022

Titles & IDs
Public title
Extension Study of Oral PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Scientific title
A Phase II/III, Extension Study of Orally Administered PHA-022121 for Acute Treatment of Angioedema Attacks in Patients With Hereditary Angioedema
Secondary ID [1] 0 0
2023-505766-28-00
Secondary ID [2] 0 0
PHA022121-C303
Universal Trial Number (UTN)
Trial acronym
RAPIDe-2
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema 0 0
Hereditary Angioedema Type I 0 0
Hereditary Angioedema Type II 0 0
Hereditary Angioedema Types I and II 0 0
Hereditary Angioedema Attack 0 0
Hereditary Angioedema With C1 Esterase Inhibitor Deficiency 0 0
Hereditary Angioedema - Type 1 0 0
Hereditary Angioedema - Type 2 0 0
C1 Esterase Inhibitor [C1-INH] Deficiency 0 0
C1 Esterase Inhibitor Deficiency 0 0
C1 Esterase Inhibitor, Deficiency of 0 0
C1 Inhibitor Deficiency 0 0
Hereditary Angioedema Type III 0 0
Hereditary Angioedema (HAE) 0 0
Hereditary Angioedema Type I and II 0 0
Condition category
Condition code
Cardiovascular 0 0 0 0
Diseases of the vasculature and circulation including the lymphatic system
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Blood 0 0 0 0
Other blood disorders
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - deucrictibant selected dose

Experimental: Part B: Selected dose - Single dose of deucrictibant


Treatment: Drugs: deucrictibant selected dose
deucrictibant soft capsule for oral use
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
Timepoint [1] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [2] 0 0
Heart Rate
Timepoint [2] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [3] 0 0
Blood pressure
Timepoint [3] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [4] 0 0
Body temperature
Timepoint [4] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [5] 0 0
Clinical laboratory tests
Timepoint [5] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [6] 0 0
Electrocardiograms
Timepoint [6] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Primary outcome [7] 0 0
Physical Examination
Timepoint [7] 0 0
From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Secondary outcome [1] 0 0
Time to onset of symptom relief, defined as Patient Global Impression of Change (PGI-C) rating of at least "a little better" for 2 consecutive timepoints within 12 hours post-treatment
Timepoint [1] 0 0
Assessed from 1 hour to 12 hours post-treatment
Secondary outcome [2] 0 0
Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment
Timepoint [2] 0 0
Assessed from 1 hour to 12 hours post-treatment
Secondary outcome [3] 0 0
Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S), defined as achieving =1 point reduction in PGI-S from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
Timepoint [3] 0 0
Assessed from pre-treatment to 12 hours post-treatment
Secondary outcome [4] 0 0
Time to onset of symptom relief by VAS-3/VAS-5 (Part A) or AMRA (Part B), defined as a reduction of =30% from pretreatment in VAS/AMRA composite score, sustained for 2 consecutive timepoints)
Timepoint [4] 0 0
Assessed from pre-treatment to 48 hours post-treatment
Secondary outcome [5] 0 0
Time to symptom relief by VAS (Part A) or AMRA (Part B), based on achieving =50% reduction from pretreatment in VAS/AMRA composite score sustained for 2 consecutive timepoints.
Timepoint [5] 0 0
Assessed from pre-treatment to 48 hours post-treatment
Secondary outcome [6] 0 0
Proportion of deucrictibant-treated attacks requiring rescue medication within 24 hours post-treatment
Timepoint [6] 0 0
Assessed from pre-treatment to 24 hours post-treatment
Secondary outcome [7] 0 0
Proportion of deucrictibant-treated attacks with almost complete or complete symptom relief by VAS-3/ VAS-5/ AMRA through 24 hours post-treatment
Timepoint [7] 0 0
Assessed from pre-treatment to 24 hours post-treatment

Eligibility
Key inclusion criteria
Key

1. Provision of written informed consent. If the participant is a minor (i.e., <18 years of age or as determined by local law), consent will be obtained from the participant's parent/legally designated representative/guardian and written assent will be obtained from the participant, per country regulations.
2. For participants from Study C201, received at least one dose of study drug (including the non-attack visit) in Study C201. For participants from Study C306, participant was randomized (and for adolescent participants 12 to <18 years received a dose of study drug in a non-attack state at Visit 1) and completed Study C306, with 2 attacks treated, or after closure of that study by the Sponsor.

Enrollment of adolescents (=12 to <18 years or age of adulthood as defined locally) from these studies is with consideration of local age requirements.
3. Female participants of childbearing potential (or who become of childbearing potential during the study) must agree to the protocol-specified pregnancy testing and to be abstinent from heterosexual intercourse or to use an acceptable contraception method as defined in the protocol and as available locally from enrollment until 30 days after the last study drug administration.
4. In the opinion of the Investigator, the participant (and parent/caregiver for adolescent participants) is willing and able to comply with the protocol.

Key
Minimum age
12 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
1. Any female who is pregnant, plans to become pregnant, or is breast-feeding.
2. Any other systemic disease (e.g., cardiovascular, gastrointestinal, renal, respiratory, neurological) or significant disease or disorder that, in the opinion of the Investigator, would interfere with the participant's safety or ability to participate in the study.
3. Use of lanadelumab for long-term HAE prophylactic therapy within 12 weeks prior to enrollment in Part A.
4. For Part A: Use of C1-esterase inhibitor, oral kallikrein inhibitors, attenuated androgens, anti-fibrinolytics, or monoclonal HAE therapy within a defined period prior to enrolment.

For Part B: If a participant is receiving long-term prophylactic therapy with one of the following medications indicated for HAE: plasma-derived C1-INH, danazol at less than or equal to 200 mg/day, anti-fibrinolytics, berotralstat, or lanadelumab, they must be on a stable dose and regimen for at least 3 months before screening and intends to remain on the same dose for the duration of the study.
5. History of alcohol or drug abuse within defined period, or current evidence of substance dependence or abuse
6. Participation in any other investigational drug study within defined period
7. Discontinued from parent study after enrollment for any study drug-related safety reason or non-compliance including significant protocol deviation.
8. Use of concomitant medications that are strong CYP3A4 inhibitors (e.g., clarithromycin, erythromycin, itraconazole, ketoconazole, ritonavir) or strong CYP3A4 inducers (e.g., carbamazepine and phenytoin).

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
28/12/2022
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/06/2027
Actual
Sample size
Target
140
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Study site - Campbelltown
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
Arkansas
Country [4] 0 0
United States of America
State/province [4] 0 0
California
Country [5] 0 0
United States of America
State/province [5] 0 0
Colorado
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Michigan
Country [8] 0 0
United States of America
State/province [8] 0 0
Missouri
Country [9] 0 0
United States of America
State/province [9] 0 0
Pennsylvania
Country [10] 0 0
United States of America
State/province [10] 0 0
Texas
Country [11] 0 0
Argentina
State/province [11] 0 0
Buenos Aires
Country [12] 0 0
Austria
State/province [12] 0 0
Graz
Country [13] 0 0
Austria
State/province [13] 0 0
Wien
Country [14] 0 0
Bulgaria
State/province [14] 0 0
Sofia
Country [15] 0 0
Canada
State/province [15] 0 0
Alberta
Country [16] 0 0
Canada
State/province [16] 0 0
Quebec
Country [17] 0 0
Czechia
State/province [17] 0 0
Brno
Country [18] 0 0
France
State/province [18] 0 0
Grenoble
Country [19] 0 0
France
State/province [19] 0 0
Paris
Country [20] 0 0
Germany
State/province [20] 0 0
Berlin
Country [21] 0 0
Germany
State/province [21] 0 0
Frankfurt am Main
Country [22] 0 0
Germany
State/province [22] 0 0
Lubeck
Country [23] 0 0
Hong Kong
State/province [23] 0 0
Hong Kong
Country [24] 0 0
Hungary
State/province [24] 0 0
Budapest
Country [25] 0 0
Israel
State/province [25] 0 0
Ashkelon
Country [26] 0 0
Italy
State/province [26] 0 0
Catania
Country [27] 0 0
Italy
State/province [27] 0 0
Milano
Country [28] 0 0
Italy
State/province [28] 0 0
Napoli
Country [29] 0 0
Italy
State/province [29] 0 0
Padova
Country [30] 0 0
Italy
State/province [30] 0 0
Palermo
Country [31] 0 0
Italy
State/province [31] 0 0
Roma
Country [32] 0 0
Japan
State/province [32] 0 0
Hiroshima
Country [33] 0 0
Japan
State/province [33] 0 0
Kanagawa
Country [34] 0 0
Japan
State/province [34] 0 0
Osaka
Country [35] 0 0
Japan
State/province [35] 0 0
Tokyo
Country [36] 0 0
Korea, Republic of
State/province [36] 0 0
Daegu
Country [37] 0 0
Korea, Republic of
State/province [37] 0 0
Seoul
Country [38] 0 0
Netherlands
State/province [38] 0 0
Amsterdam
Country [39] 0 0
Poland
State/province [39] 0 0
Kraków
Country [40] 0 0
Puerto Rico
State/province [40] 0 0
San Juan
Country [41] 0 0
South Africa
State/province [41] 0 0
Cape Town
Country [42] 0 0
Spain
State/province [42] 0 0
Barcelona
Country [43] 0 0
Spain
State/province [43] 0 0
Madrid
Country [44] 0 0
Sweden
State/province [44] 0 0
Lund
Country [45] 0 0
Turkey
State/province [45] 0 0
Ankara
Country [46] 0 0
Turkey
State/province [46] 0 0
Istanbul

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Pharvaris Netherlands B.V.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Study Director
Address 0 0
Pharvaris Netherlands B.V.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05396105