Hereditary Angioedema (HAE)
Conditions
Keywords
HAE, Angioedema, Long-Term, Safety
Brief summary
This is a Phase 3 multicenter trial in 2 parts to evaluate the long-term safety and efficacy of navenibart in adult and adolescent participants with hereditary angioedema (HAE) who participated in STAR-0215-301 (NCT06842823; ALPHA-ORBIT). Part 1 provides all participants with navenibart in a dose-controlled fashion; Part 2 introduces a personalized dosing option (PDO) for participants based on individual needs.
Interventions
DRUGnavenibart
Navenibart will be administered as a subcutaneous injection.
Sponsors
Astria Therapeutics, Inc.
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
PREVENTION
Masking
NONE
Masking description
All participants will knowingly receive navenibart, however participants, care providers, investigators, and outcome assessors will be blinded to their treatment arm for a duration specific to the individual.
Intervention model description
In Part 1, adult participants who completed STAR-0215-301 and received navenibart will maintain their fixed dosing regimen from STAR-0215-301, whereas those who were randomized to placebo in STAR-0215-301 will receive navenibart 600 mg Q3M. In Part 2, adult participants may move among dosing regimens via the personalized dosing option (PDO) based on their individual needs. Adult participants who did not complete STAR-0215-301 may be eligible to enroll in Part 2 of STAR-0215-302 if they meet certain criteria. Adolescent participants will continue to receive their fixed dosing regimen from STAR-0215-301 until they turn 18 years of age.
Eligibility
Sex/Gender
ALL
Age
12 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1\. Participants from STAR-0215-301 who met one of the following conditions: 1. Completed STAR-0215-301 through the Day 181 visit 2. Withdrew from STAR-0215-301 but met the following criteria: i. Received 2 doses of IP ii. completed ≥ 2 months of trial follow-up after the second dose of IP iii. Met other eligibility criteria as assessed by Investigator
Exclusion criteria
1. Participation in an investigational clinical trial other than STAR-0215- 301 in the 30 days or any exposure to an investigational drug (other than navenibart in STAR-0215-301) within 5 half-lives before informed consent/assent 2. Any exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen containing medications with systemic absorption (such as hormonal contraceptives or hormone replacement therapy) within 30 days before Screening. 3. Known sensitivity to the ingredients in the formulation of IP
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | Day 1 through Month 48 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Number of time-normalized investigator-confirmed HAE attacks | Day 1 through Month 48 | — |
| Number of moderate or severe investigator-confirmed HAE attacks | Day 1 through Month 48 | — |
| Number of investigator-confirmed HAE attacks that require on-demand treatment | Day 1 through Month 48 | — |
| Percent reduction in monthly investigator-confirmed HAE attacks | Day 1 through Month 48 | — |
| Time to first investigator-confirmed HAE attack after first dose | Day 1 through Month 48 | — |
| The number of participants responding to treatment, defined as a ≥ 50%, ≥ 70%, or ≥ 90% reduction in investigator-confirmed HAE attack rate | Day 1 through Month 48 | — |
| Number of participants with no investigator-confirmed HAE attacks | Day 1 through Month 48 | — |
| Angioedema Quality of Life questionnaire total score | Day 1 through Month 48 | The Angioedema Quality of Life Questionnaire (AE-QoL) consists of 17 questions divided into four domains: functioning (4 questions), fatigue/mood (5), fears/shame (6), and food/nutrition (2). Each question is answered on a 5-point scale from "never" to "very often." Scores are calculated for each domain and then normalized to a scale from 0 to 100, where 0 represents the best possible quality of life and 100 the worst. |
Countries
Brazil, Bulgaria, Canada, Czechia, France, Germany, Hong Kong, Israel, Italy, Japan, Netherlands, North Macedonia, Poland, Puerto Rico, South Africa, Spain, United Kingdom, United States
Outcome results
None listed