Clinical trial NCT06842823: A Study of Navenibart in Participants With Hereditary Angioedema
Official title: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Navenibart in Participants With Hereditary Angioedema (HAE)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Hereditary Angioedema (HAE)
Keywords: HAE
Angioedema
Lead sponsor: Astria Therapeutics, Inc.
Sponsor class: INDUSTRY
First posted: 2025-02-24
Enrollment: 145
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Adult participants will be randomly assigned to one of 4 arms. Adolescent participants will be assigned to a single experimental group.
Primary purpose: TREATMENT

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.


Eligibility criteria: 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. Lanadelumab within 90 days prior to Run-In
  2. Berotralstat within 21 days prior to Run-In
  3. Plasma-derived C1INH for LTP within 14 days prior to Run-In
  4. Tranexamic acid, oral danazol, oral stanazolol, and oral oxandrolone within 3 days prior to Run-In
  5. All other prophylactic therapies, including investigational drugs, require consultation with the Medical Monitor

Primary outcomes:
measure: Number of time-normalized investigator-confirmed HAE attacks during the 6-month Treatment Period.
timeFrame: Day 1 through Day 181
Secondary outcomes:
measure: Number of moderate or severe investigator-confirmed HAE attacks during the 6-month Treatment Period.
timeFrame: Day 1 through Day 181
measure: Number of investigator-confirmed HAE attacks that require on-demand treatment during the 6-month Treatment Period.
timeFrame: Day 1 through Day 181
measure: Percent reduction in monthly investigator-confirmed HAE attacks in the 6-month Treatment Period versus the Run-In Period.
timeFrame: Baseline through Day 181
measure: Time to first investigator-confirmed HAE attack after first and second dose.
timeFrame: Day 1 through Day 181
measure: 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).
timeFrame: Baseline through Day 181
measure: Number of participants with no investigator-confirmed HAE attacks during the 6-month Treatment Period.
timeFrame: Day 1 through Day 181
measure: Change from baseline (Day 1) in the Angioedema Quality of Life questionnaire total score.
timeFrame: Day 1 through Day 181
Other outcomes:
measure: Incidence of treatment-emergent adverse events.
timeFrame: Day 1 through Day 361
Locations:
city: Scottsdale
country: United States
facility: Site 2
state: Arizona
city: Little Rock
country: United States
facility: Site 3
state: Arkansas
city: Santa Monica
country: United States
facility: Site 4
state: California
city: Walnut Creek
country: United States
facility: Site 1
state: California
city: Centennial
country: United States
facility: Site 8
state: Colorado
city: Wheaton
country: United States
facility: Site 6
state: Maryland
city: Cincinnati
country: United States
facility: Site 7
state: Ohio
city: Ottawa
country: Canada
facility: Site 5
state: Ontario
Study officials:
References: