This study evaluates the safety and efficacy of long-term on-demand treatment with orally administered deucrictibant for acute hereditary angioedema (HAE) attacks, including laryngeal attacks. The study will enroll patients from Study PHA022121-C201 (NCT04618211) and Study PHA022121-C306 (NCT06343779) who elect to participate in this extension study and meet the eligibility requirements.
Part A of the study will enroll adult participants from Study PHA022121-C201. The double-blind treatment assignment from Study PHA022121-C201 will be maintained. Part B will include participants rolling over from Part A and additionally enroll participants from Study PHA022121-C201 who did not participate in Part A, and participants from Study PHA022121-C306 who elect to participate in this extension study and meet the eligibility requirements.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
140
deucrictibant soft capsule for oral use
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
Treatment-emergent Adverse Events (TEAEs), treatment-related TEAEs, treatment-emergent serious adverse events (TESAEs), treatment-related TESAEs, and TEAEs leading to deucrictibant discontinuation
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Heart Rate
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Blood pressure
Systolic and diastolic blood pressure will be measured. Descriptive in nature, no formal statistical hypothesis testing will be performed.
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Body temperature
Descriptive in nature, no formal statistical hypothesis testing will be performed.
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Clinical laboratory tests
hematology, blood chemistry, urinalysis
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Electrocardiograms
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
Physical Examination
Time frame: From enrollment through study completion, up to 54 months (dependent on time of enrollment).
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
PGI-C evaluates the change in the attack symptoms over time with a 7-point response scale.
Time frame: Assessed from 1 hour to 12 hours post-treatment
Time to substantial symptom relief, defined as achieving PGI-C rating of at least "better" for 2 consecutive timepoints within 12 hours post-treatment
PGI-C evaluates the change in the attack symptoms over time with a 7-point response scale.
Time frame: Assessed from 1 hour to 12 hours post-treatment
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
PGI-S evaluates the severity of attack symptoms with a 5-point response scale.
Time frame: Assessed from pre-treatment to 12 hours post-treatment
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)
VAS scores range between 0 and 100. A larger reduction means a better outcome.
Time frame: Assessed from pre-treatment to 48 hours post-treatment
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.
VAS/AMRA scores range between 0 and 100. A larger reduction means a better outcome.
Time frame: Assessed from pre-treatment to 48 hours post-treatment
Proportion of deucrictibant-treated attacks requiring rescue medication within 24 hours post-treatment
Time frame: Assessed from pre-treatment to 24 hours post-treatment
Proportion of deucrictibant-treated attacks with almost complete or complete symptom relief by VAS-3/ VAS-5/ AMRA through 24 hours post-treatment
Almost complete or complete symptom relief is defined as all individual item scores in VAS/AMRA having a value ≤10 sustained for 2 consecutive timepoints.
Time frame: Assessed from pre-treatment to 24 hours post-treatment
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Walnut Creek, California, United States
Study site
Colorado Springs, Colorado, United States
Study site
Chevy Chase, Maryland, United States
Study site
Boston, Massachusetts, United States
Study site
Detroit, Michigan, United States
...and 53 more locations