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Study of Oral Deucrictibant Soft Capsule for On-Demand Treatment of Angioedema Attacks in Adolescents and Adults With Hereditary Angioedema
This is a Phase 3, multicenter, randomized, double-blind, placebo-controlled, 2-period, 2-treatment cross-over study to evaluate the efficacy and safety of orally administered deucrictibant compared to placebo for the on-demand treatment of HAE attacks, including non-severe laryngeal attacks, in participants ≥12 to ≤75 years of age with HAE type 1 or type 2 (HAE-1/2), a proportion of whom are using long-term prophylactic medication for HAE.
Study details:
The study consists of a Screening Phase during which eligibility is confirmed, a Treatment Phase in which participants will be randomized and receive double blinded study drug to treat 2 qualifying HAE attacks (i. e. , 2 Treatment Periods within the Treatment Phase), and an End-of-Study Follow-up Phase after the second attack treated with study drug.
In addition, for adolescent participants (age ≥12 to \<18 years), PK samples are collected after administration of deucrictibant at Day 1 in a non-attack state.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 12 and older
Healthy volunteers accepted : No
Gender eligible for study: All
Things to know
Study dates
Study start: 2024-02-26
Primary completion: 2026-03-01
Study completion finish: 2026-03-01
Study type
TREATMENT
Phase
PHASE3
Trial ID
NCT06343779
Intervention or treatment
DRUG: Deucrictibant, Placebo
Conditions
- • Hereditary Angioedema
- • Hereditary Angioedema Type I
- • Hereditary Angioedema Type II
- • Hereditary Angioedema Types I and II
- • Hereditary Angioedema Attack
- • Hereditary Angioedema With C1 Esterase Inhibitor Deficiency
- • Hereditary Angioedema - Type 1
- • Hereditary Angioedema - Type 2
- • C1 Esterase Inhibitor [C1-INH] Deficiency
- • C1 Esterase Inhibitor Deficiency
- • C1 Esterase Inhibitor, Deficiency of
- • C1 Inhibitor Deficiency
Find a site
Closest Location:
Study Site
Research sites nearby
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Study Site
Campbelltown, New South Wales, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: Arm 1
| DRUG: Deucrictibant, Placebo
|
EXPERIMENTAL: Arm 2
| DRUG: Deucrictibant, Placebo
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
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. | The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. | Pre-treatment to 12 hours post-treatment. |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Proportion of study drug-treated attacks achieving PGI-C rating of at least "a little better" at 4 hours post-treatment. | The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. | Pre-treatment to 4 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. | The PGI-C (7-point scale) is used to evaluate the change in the HAE attack symptoms as compared to pre-treatment. | Pre-treatment 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 (5-point scale) from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment. | Pre-treatment to 12 hours post-treatment. |
Time to complete symptom resolution, defined as achieving PGI-S rating of "none" within 48 hours post-treatment. | The PGI-S (5-point scale) is used to evaluate the severity of HAE attack symptoms. | Pre-treatment to 48 hours post-treatment. |
Time to End of Progression (EoP) in attack symptoms within 12 hours. | EoP time defined as the earliest post-treatment timepoint after which all subsequent PGI-C ratings are stable or improved. | Pre-treatment to 12 hours post-treatment. |
Proportion of study drug-treated attacks requiring rescue medication within 24 hours post-treatment. | Rescue medication is defined as the participant's usual acute on-demand HAE treatment taken if symptoms persist or progress after study drug administration. | Pre-treatment to 24 hours post-treatment. |
Proportion of attacks achieving symptom resolution. | Defined as achieving PGI-S rating of "none" with one dose of study drug at 24 hours post-treatment. | Pre-treatment to 24 hours post-treatment. |
Time to substantial symptom relief by Angioedema Symptom Rating Scale (AMRA). | Defined as a ≥50% reduction in AMRA composite score from pre-treatment for 2 consecutive timepoints within 12 hours post-treatment. | Pre-treatment to 12 hours post-treatment. |
Time to almost complete or complete symptom relief by AMRA. | Defined as all item scores in AMRA having a value ≤10 for 2 consecutive timepoints within 24 hours post-treatment. | Pre-treatment to 24 hours post-treatment. |
Proportion of study drug-treated attacks reaching almost complete or complete symptom relief by AMRA. | Defined as all item scores in AMRA having a value ≤10 at 24 hours post-treatment. | Pre-treatment to 24 hours post-treatment. |
Time to EoP in attack symptoms within 12 hours. | Defined as the earliest post-treatment timepoint after which every individual AMRA item is stable or improved at all subsequent timepoints. | Pre-treatment to 12 hours post-treatment. |
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