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 participants from Study PHA022121-C201 (NCT04618211), Study PHA022121-C306 (NCT06343779) and deucrictibant treatment naïve HAE-nC1INH adult participants 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 is open-label treatment and will include participants rolling over from Part A. Participants from Study PHA022121-C201 who did not participate in Part A, participants from Study PHA022121-C306, and deucrictibant treatment naïve HAE-nC1INH adult participants who elect to participate in this extension study and meet the eligibility requirements.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
150
3 capsules of deucrictibant or matching placebo will be administered orally for each HAE attack
deucrictibant soft capsules will be administered orally for each HAE attack
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
Proportion of attacks achieving symptom resolution, defined as achieving PGI-S rating of "none" at 24 hours post-treatment.
Time frame: At 24 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
Time to onset of symptom relief, assessed by a ≥30% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from the pre-treatment score
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
Time to substantial symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as a ≥50% reduction in VAS-3/ VAS-5 (Part A) or AMRA (Part B) composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment
VAS/AMRA scores range between 0 and 100. A larger reduction means a better outcome.
Time frame: Assessed from pre-treatment to 12 hours post-treatment
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by VAS-3/ VAS-5 (Part A) or AMRA (Part B), defined as all item scores in VAS-3/ VAS-5/ AMRA having a value ≤10 at 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: At 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