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
The study consists of a Screening Period during which eligibility is confirmed (only for participants not rolling over within 28 days from a previous deucrictibant prophylactic study), a Treatment Period in which participants will receive open-label deucrictibant extended-release tablet once daily for approximately 240 weeks, followed by an End of Study visit after maximum 4 weeks. Participants will undergo regular safety (e.g. lab draws) and efficacy assessments, will complete an electronic diary, and also complete questionnaires at predefined timepoints during the study.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
170
Deucrictibant extended-release tablet for once daily oral use
Number of participants with treatment-emergent adverse events (TEAEs), including serious adverse events (SAEs), adverse events of special interest (AESIs), and TEAEs leading to study drug discontinuation
Time frame: Up to Week 240
Change in heart rate
Time frame: Up to Week 240
Change in blood pressure
Time frame: Up to Week 240
Change in body temperature
Time frame: Up to Week 240
Number of participants with clinically significant changes in Hematology
Blood samples will be collected for the analysis of hematology parameters.
Time frame: Up to Week 240
Number of participants with clinically significant changes in Clinical Chemistry
Blood samples will be collected for the analysis of clinical chemistry parameters.
Time frame: Up to Week 240
Number of participants with clinically significant changes in Urinalysis
Urine samples will be collected for the analysis of urinalysis parameters.
Time frame: Up to Week 240
Change from Baseline electrocardiograms (ECGs)
Digital triplicate 12-lead ECG. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed HAE attacks during the Treatment Period
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Study Site
Birmingham, Alabama, United States
Study Site
Scottsdale, Arizona, United States
Study Site
Little Rock, Arkansas, United States
Study Site
San Diego, California, United States
Study Site
Santa Monica, California, United States
Study Site
Walnut Creek, California, United States
Study Site
Colorado Springs, Colorado, United States
Study Site
Wheaton, Illinois, United States
Study Site
St Louis, Missouri, United States
Study Site
Las Vegas, Nevada, United States
...and 57 more locations
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed HAE attacks treated with on-demand medication during the Treatment Period
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed moderate or severe HAE attacks during Treatment Period
Time frame: Up to Week 240
Time-normalized number of Investigator-confirmed severe HAE attacks during the Treatment Period
Time frame: Up to Week 240
Proportion of time without angioedema symptoms during the Treatment Period
Time frame: Up to Week 240
Patient reported outcome: Angioedema Quality of Life (AE-QoL) questionnaire
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.
Time frame: Up to Week 132
Patient reported outcome: Patient Global Assessment of Change (PGA-Change)
PGA-Change assesses on a 5-point scale how the participant's QoL has been impacted by HAE since start taking the study drug.
Time frame: Up to Week 132
Patient reported outcome: Angioedema Control Test 4-week version (AECT-4wk)
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).
Time frame: Up to Week 132
Patient reported outcome: Work Productivity and Activity Impairment Questionnaire - Specific Health Problem (WPAI-SHP)
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.
Time frame: Up to Week 132
Patient reported outcome: Abbreviated Treatment Satisfaction Questionnaire for Medication (TSQM-9)
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.
Time frame: Up to Week 132
Pharmacokinetics [PK]: Pre-dose plasma concentration of deucrictibant and deucrictibant metabolites (Ctrough)
Time frame: Up to Week 96
Pharmacokinetics [PK]: Urine concentration of deucrictibant and deucrictibant metabolites
Time frame: Up to Week 48