Hereditary Angioedema (HAE)
Conditions
Keywords
HAE, Angioedema
Brief summary
This is a Phase 3 multicenter, randomized, double-blind, placebo-controlled clinical trial evaluating the safety and efficacy of subcutaneous administration of navenibart in adult and adolescent participants with type 1 or type 2 hereditary angioedema (HAE). The goal of this clinical trial is to evaluate the efficacy and safety of navenibart compared to placebo in preventing HAE attacks in participants with HAE.
Interventions
DRUGnavenibart
Navenibart will be administered as a subcutaneous injection.
DRUGPlacebo
Placebo will be administered as a subcutaneous injection.
Sponsors
Astria Therapeutics, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Intervention model description
Adult participants will be randomly assigned to one of 4 arms. Adolescent participants will be assigned to a single experimental group.
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Documented diagnosis of HAE (Type 1 or 2). The following must be met: 1. Documented clinical history consistent with HAE 2. Lab findings consistent with HAE Type 1 or 2 * Experienced at least 2 HAE attacks during the Run-In period, as confirmed by an investigator based on meeting the protocol-specified definition of an HAE attack.
Exclusion criteria
* Any concomitant diagnosis of another form of chronic angioedema, such as acquired C1 inhibitor deficiency, HAE with normal C1-INH (also known as HAE type 3), idiopathic angioedema, or angioedema associated with urticaria. * Use of therapies prescribed for the prevention of HAE attacks may not be used during the trial or within the below time frames prior to the Run-In Period (adult participants may be on these medications at the time of the Screening Visit, but will need to washout prior to entering the Run-In Period). 1. Tranexamic acid, oral danazol, oral stanazolol, and oral oxandrolone within 3 days prior to Run-In 2. Plasma-derived C1INH for LTP within 14 days prior to Run-In 3. Berotralstat within 21 days prior to Run-In 4. Lanadelumab within 70 days prior to Run-In 5. Garadacimab within 90 days prior to Run-In
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period. | Day 1 through Day 181 |
Secondary
| Measure | Time frame |
|---|---|
| Number of moderate or severe investigator-confirmed HAE attacks during the 6-month Treatment Period. | Day 1 through Day 181 |
| Number of investigator-confirmed HAE attacks that require on-demand treatment during the 6-month Treatment Period. | Day 1 through Day 181 |
| Percent reduction in monthly investigator-confirmed HAE attacks in the 6-month Treatment Period versus the Run-In Period. | Baseline through Day 181 |
| Time to first investigator-confirmed HAE attack after first and second dose. | Day 1 through Day 181 |
| The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction from the Run-In Period in investigator-confirmed HAE attack rate (for adult participants: compared to placebo during the 6-month Treatment Period). | Baseline through Day 181 |
| Number of participants with no investigator-confirmed HAE attacks during the 6-month Treatment Period. | Day 1 through Day 181 |
| Change from baseline (Day 1) in the Angioedema Quality of Life questionnaire total score. | Day 1 through Day 181 |
Countries
Australia, Austria, Brazil, Bulgaria, Canada, Czechia, France, Germany, Hong Kong, Hungary, Israel, Italy, Japan, Netherlands, New Zealand, North Macedonia, Poland, Portugal, Puerto Rico, South Africa, South Korea, Spain, United Kingdom, United States
Outcome results
None listed