Trial Review

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06343779




Registration number
NCT06343779
Ethics application status
Date submitted
18/03/2024
Date registered
3/04/2024
Date last updated
20/11/2024

Titles & IDs
Public title
Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
Scientific title
A Phase 3, Randomized, Double-blind, Placebo-controlled, Cross-over Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Attacks in Adolescents and Adults With Hereditary Angioedema
Secondary ID [1] 0 0
PHA022121-C306
Universal Trial Number (UTN)
Trial acronym
RAPIDe-3
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
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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Deucrictibant, Placebo

Experimental: Arm 1 - Deucrictibant administered for first HAE attack, placebo administered for second HAE attack.

Experimental: Arm 2 - Placebo administered for first HAE attack, deucrictibant administered for second HAE attack.


Treatment: Drugs: Deucrictibant, Placebo
Deucrictibant Soft Capsules for Oral Use
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary 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
Pre-treatment to 12 hours post-treatment.
Secondary outcome [1] 0 0
Proportion of study drug-treated attacks achieving PGI-C rating of at least "a little better" at 4 hours post-treatment.
Timepoint [1] 0 0
Pre-treatment to 4 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
Pre-treatment to 12 hours post-treatment.
Secondary outcome [3] 0 0
Time to substantial symptom relief by Patient Global Impression of Severity (PGI-S).
Timepoint [3] 0 0
Pre-treatment to 12 hours post-treatment.
Secondary outcome [4] 0 0
Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment.
Timepoint [4] 0 0
Pre-treatment to 48 hours post-treatment.
Secondary outcome [5] 0 0
Time to End of Progression (EoP) in attack symptoms within 12 hours.
Timepoint [5] 0 0
Pre-treatment to 12 hours post-treatment.
Secondary outcome [6] 0 0
Proportion of study drug-treated attacks requiring rescue medication within 24 hours post-treatment.
Timepoint [6] 0 0
Pre-treatment to 24 hours post-treatment.
Secondary outcome [7] 0 0
Proportion of attacks achieving symptom resolution.
Timepoint [7] 0 0
Pre-treatment to 24 hours post-treatment.
Secondary outcome [8] 0 0
Time to substantial symptom relief by Angioedema Symptom Rating Scale (AMRA).
Timepoint [8] 0 0
Pre-treatment to 12 hours post-treatment.
Secondary outcome [9] 0 0
Time to almost complete or complete symptom relief by AMRA.
Timepoint [9] 0 0
Pre-treatment to 24 hours post-treatment.
Secondary outcome [10] 0 0
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA.
Timepoint [10] 0 0
Pre-treatment to 24 hours post-treatment.
Secondary outcome [11] 0 0
Time to EoP in attack symptoms within 12 hours.
Timepoint [11] 0 0
Pre-treatment to 12 hours post-treatment.

Eligibility
Key inclusion criteria
1. Provision of written informed consent/assent.
2. Male or female, aged =12 to =75 years at the time of providing written informed consent/assent.
3. Diagnosis of HAE-1/2.
4. History of at least 2 HAE attacks in the last 3 months before screening.
5. Experience with using standard-of-care treatment to effectively manage on-demand treatment for HAE attacks.
6. Participants on long-term prophylactic therapy with plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat, or lanadelumab) must be on a stable dose and regimen and intend to remain on the same dose for 6 months before screening and the duration of the study. OR, Participant has stopped using plasma-derived C1-INH (danazol, anti-fibrinolytics, berotralstat) at least 2 weeks or lanadelumab at least 10 weeks before screening.
7. Capable of recording, without assistance, electronic HAE diary and ePRO data using an electronic device.
8. For adolescent participants aged =12 and <18 years of age: body weight =40 kg.
9. Female participants of childbearing potential must agree to the protocol specified pregnancy testing and contraception methods.
Minimum age
12 Years
Maximum age
75 Years
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 breastfeeding.
2. Any diagnosis of angioedema other than HAE-1/2.
3. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
4. Use of attenuated androgens for short-term prophylaxis within 2 weeks before screening.
5. Abnormal hepatic function.
6. Abnormal renal function (eGFR <60 ml/min/1.73 m2).
7. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse.
8. Has received prior on-demand HAE treatment with deucrictibant.
9. Currently participating in any other investigational drug study or receiving other investigational treatment within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
10. Prior gene therapy for any indication at any time.
11. Use of concomitant medications with systemic absorption that are strong inhibitors of CYP3A4 or strong inducers of CYP3A4 within the last 30 days, or within 5 half-lives (whichever is longer) of the time of randomization.
12. Known hypersensitivity to study drug or any of the excipients of study drug.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 3
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
26/02/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2026
Actual
Sample size
Target
120
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW
Recruitment hospital [1] 0 0
Study Site - Campbelltown
Recruitment hospital [2] 0 0
Study Site - Box Hill
Recruitment postcode(s) [1] 0 0
2560 - Campbelltown
Recruitment postcode(s) [2] 0 0
3128 - Box Hill
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
Bulgaria
State/province [11] 0 0
Sofia
Country [12] 0 0
Canada
State/province [12] 0 0
Alberta
Country [13] 0 0
Germany
State/province [13] 0 0
Berlin
Country [14] 0 0
Germany
State/province [14] 0 0
Frankfurt am Main
Country [15] 0 0
Germany
State/province [15] 0 0
Lubeck
Country [16] 0 0
Hong Kong
State/province [16] 0 0
Hong Kong
Country [17] 0 0
Hungary
State/province [17] 0 0
Budapest
Country [18] 0 0
Italy
State/province [18] 0 0
Catania
Country [19] 0 0
Italy
State/province [19] 0 0
Milano
Country [20] 0 0
Italy
State/province [20] 0 0
Milan
Country [21] 0 0
Italy
State/province [21] 0 0
Padova
Country [22] 0 0
Italy
State/province [22] 0 0
Palermo
Country [23] 0 0
Japan
State/province [23] 0 0
Chiba-city
Country [24] 0 0
Japan
State/province [24] 0 0
Hiroshima
Country [25] 0 0
Japan
State/province [25] 0 0
Kanagawa
Country [26] 0 0
Japan
State/province [26] 0 0
Osaka
Country [27] 0 0
Japan
State/province [27] 0 0
Tokyo
Country [28] 0 0
Korea, Republic of
State/province [28] 0 0
Daegu
Country [29] 0 0
Korea, Republic of
State/province [29] 0 0
Seoul
Country [30] 0 0
North Macedonia
State/province [30] 0 0
Skopje
Country [31] 0 0
Poland
State/province [31] 0 0
Kraków
Country [32] 0 0
Puerto Rico
State/province [32] 0 0
San Juan
Country [33] 0 0
Sweden
State/province [33] 0 0
Lund
Country [34] 0 0
Turkey
State/province [34] 0 0
Ankara
Country [35] 0 0
Turkey
State/province [35] 0 0
Istanbul
Country [36] 0 0
United Kingdom
State/province [36] 0 0
Camberley
Country [37] 0 0
United Kingdom
State/province [37] 0 0
London
Country [38] 0 0
United Kingdom
State/province [38] 0 0
Plymouth

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, Pharvaris
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
Pharvaris Clinical Team
Address 0 0
Country 0 0
Phone 0 0
+31 (71) 203-6410
Fax 0 0
Email 0 0
clinicaltrials@pharvaris.com
Contact person for scientific queries

No information has been provided regarding IPD availability


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/NCT06343779