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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06679881
Registration number
NCT06679881
Ethics application status
Date submitted
24/10/2024
Date registered
8/11/2024
Date last updated
8/09/2025
Titles & IDs
Public title
Long-Term, Open-label Study of Oral Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With HAE
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Scientific title
A Long-term, Open-label Study to Evaluate the Safety and Efficacy of Orally Administered Deucrictibant Extended-Release Tablet for Prophylaxis Against Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
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Secondary ID [1]
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PHA022121-C307
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Universal Trial Number (UTN)
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Trial acronym
CHAPTER-4
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Hereditary Angioedema (HAE)
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Blood
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Other blood disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Deucrictibant
Experimental: Deucrictibant - Deucrictibant
Treatment: Drugs: Deucrictibant
Deucrictibant extended-release tablet for once daily oral use
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
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Assessment method [1]
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Timepoint [1]
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Up to 138 weeks
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Primary outcome [2]
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Change in heart rate
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Assessment method [2]
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Timepoint [2]
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Up to 138 weeks
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Primary outcome [3]
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Change in blood pressure
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Assessment method [3]
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Timepoint [3]
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Up to 138 weeks
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Primary outcome [4]
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Change in body temperature
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Assessment method [4]
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0
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Timepoint [4]
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Up to 138 weeks
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Primary outcome [5]
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Change in clinical laboratory tests from baseline
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Assessment method [5]
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Hematology, blood chemistry, and urinalysis. Descriptive in nature, no formal statistical hypothesis testing will be performed.
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Timepoint [5]
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Up to 138 weeks
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Primary outcome [6]
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Change in electrocardiograms (ECGs) from baseline
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Assessment method [6]
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Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
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Timepoint [6]
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Up to 138 weeks
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Secondary outcome [1]
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Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period
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Assessment method [1]
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Timepoint [1]
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130 weeks
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Secondary outcome [2]
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Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period
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Assessment method [2]
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Timepoint [2]
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130 weeks
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Secondary outcome [3]
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Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period
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Assessment method [3]
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Timepoint [3]
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130 weeks
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Secondary outcome [4]
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Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period
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Assessment method [4]
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Timepoint [4]
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130 weeks
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Secondary outcome [5]
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Proportion of time without angioedema symptoms during the Treatment Period
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Assessment method [5]
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Timepoint [5]
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130 weeks
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Secondary outcome [6]
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Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire
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Assessment method [6]
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The AE-QoL is a short 17-item questionnaire designed to retrospectively assess HRQoL, with a recall period of 4 weeks. Its results can be displayed as a total score or as 4 domain scores. The scores range from 0 to 100, after linear transformation of raw values, with higher scores indicating higher HRQoL impairment.
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Timepoint [6]
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130 weeks
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Secondary outcome [7]
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Patient reported outcome: Patient Global Assessment of Change (PGA-Change)
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Assessment method [7]
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PGA-Change assesses on a 5-point scale how the participant's QoL has been impacted by HAE since start taking the study drug.
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Timepoint [7]
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130 weeks
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Secondary outcome [8]
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Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)
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Assessment method [8]
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AECT-4wk measures disease control retrospectively, it comprises 4 questions over a 4-week recall period. Scores for the responses in the AECT range from 0 to 16, with higher scores indicating better disease control (= 9 poorly controlled; = 10 well controlled).
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Timepoint [8]
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130 weeks
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Secondary outcome [9]
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Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)
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Assessment method [9]
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WPAI-SHP is a questionnaire assessing how a health condition impacts a person's ability to work and do regular activities and it includes 4 domains. Scores indicate the percentage of time the patient missed work or was less productive owing to HAE-related complications.
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Timepoint [9]
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Up to 134 weeks
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Secondary outcome [10]
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Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
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Assessment method [10]
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TSQM-9 is a 9-item questionnaire evaluating patient treatment satisfaction and it includes 3 domains. Scoring is by domain and each domain score is computed by summing the individual TSQM items in each domain and then transforming the composite score into a value ranging from 0 to 100, with higher scores indicating higher satisfaction.
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Timepoint [10]
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Up to 134 weeks
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Secondary outcome [11]
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Pharmacokinetics [PK]: Deucrictibant plasma concentration pre-dose (Ctrough)
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Assessment method [11]
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Timepoint [11]
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130 weeks
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Eligibility
Key inclusion criteria
1. Provision of written informed consent/assent.
2. Male or female, aged =12 years at the time of providing written informed consent/assent.
3. Diagnosis of hereditary angioedema (HAE)
4. For participants that did not participate in a previous deucrictibant prophylactic study: history of at least 1 attack in the last 3 consecutive months prior to Screening
5. Reliable access and ability to use standard of care on-demand treatments to effectively manage acute HAE attacks.
6. Willing and able to adhere to all protocol requirements, including eDiary and ePRO data recording.
7. Female participants of childbearing must agree to the protocol specified pregnancy testing and contraception methods.
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Minimum age
12
Years
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Maximum age
No limit
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. Any diagnosis of angioedema other than HAE
2. Participation in a clinical study with any other investigational drug within the last 30 days or within 5 half-lives of the investigational drug at ICF signature (whichever is longer)
3. Prior gene therapy for any indication at any time
4. Participants who discontinued from previous studies with deucrictibant prophylactic and/or on-demand treatment due to safety reasons or compliance issues that, in the opinion of the Investigator, would interfere with the participant's safety or compliance to participate in the study
5. Exposure to ACE inhibitors or any estrogen-containing medications with systemic absorption within 4 weeks of Screening
6. Use of prophylactic treatment for HAE within 2 weeks of Screening for C1INH, oral kallikrein inhibitors, or anti-fibrinolytics; within 4 weeks of Screening for attenuated androgens; within 5 half-lives of Screening for monoclonal antibodies, or within 7 days of Screening for short-term prophylaxis
7. Any females who are pregnant, plan to become pregnant, or are currently breast-feeding
8. Abnormal hepatic function
9. Moderate or severe renal impairment
10. Any clinically significant comorbidity or systemic dysfunction that would interfere with the participant's safety or ability to participate in the study.
11. History of alcohol or drug abuse within the previous year, or current evidence of substance dependence or abuse
12. Use of medications that are moderate and strong inhibitors or strong inducers of CYP3A4 within the last 30 days or within 5 half-lives (whichever is longer) of the time of randomization
13. Known hypersensitivity to deucrictibant or any of the excipients of the study drug
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Not applicable
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
1/02/2025
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/12/2028
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Actual
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Sample size
Target
130
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
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Recruitment hospital [1]
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Study Site - Melbourne
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Recruitment hospital [2]
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Study Site - Perth
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Recruitment postcode(s) [1]
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3052 - Melbourne
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Recruitment postcode(s) [2]
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- Melbourne
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Recruitment postcode(s) [3]
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- Perth
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Missouri
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Country [3]
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Austria
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State/province [3]
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Vienna
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Country [4]
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Bulgaria
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State/province [4]
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Sofia
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Country [5]
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Canada
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State/province [5]
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Montreal
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Country [6]
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Germany
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State/province [6]
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Berlin
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Country [7]
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Germany
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State/province [7]
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Frankfurt
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Country [8]
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Hong Kong
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State/province [8]
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Hong Kong
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Country [9]
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Ireland
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State/province [9]
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Dublin
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Country [10]
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Italy
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State/province [10]
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Padua
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Country [11]
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Poland
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State/province [11]
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Krakow
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Country [12]
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South Africa
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State/province [12]
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Cape Town
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Country [13]
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South Korea
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State/province [13]
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Daegu
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Country [14]
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South Korea
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State/province [14]
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Seoul
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Country [15]
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Spain
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State/province [15]
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Barcelona
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Country [16]
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United Kingdom
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State/province [16]
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Brighton
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Country [17]
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United Kingdom
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State/province [17]
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Bristol
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Country [18]
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United Kingdom
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State/province [18]
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Cambridge
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Country [19]
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United Kingdom
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State/province [19]
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London
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Country [20]
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United Kingdom
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State/province [20]
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Plymouth
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Pharvaris Netherlands B.V.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a Phase 3, multicenter, long-term, open-label study to evaluate the safety and efficacy of once-daily orally administered deucrictibant extended-release tablet for prophylaxis to prevent angioedema attacks in participants aged =12 years with Hereditary Angioedema
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Trial website
https://clinicaltrials.gov/study/NCT06679881
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Study Director, Pharvaris
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Address
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Pharvaris Netherlands B.V.
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Pharvaris Clinical Team
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Address
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Country
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Phone
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0031 712-036-410
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Fax
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Email
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[email protected]
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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/NCT06679881
Download to PDF