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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Adult Dosing Regimen 1 | Experimental | Participants will receive 600 mg of navenibart every 3 months. |
|
| Adult Dosing Regimen 2 | Experimental | Participants will receive 300 mg of navenibart every 3 months. |
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| Adult Dosing Regimen 3 | Experimental | Participants will receive 600 mg of navenibart every 6 months. |
|
| Adolescent Dosing Regimen 1 | Experimental | Participants will receive 300 mg of navenibart every 3 months. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| navenibart | Drug | Navenibart will be administered as a subcutaneous injection. |
|
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment-emergent adverse events (TEAEs) | Day 1 through Month 48 |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | |
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Inclusion Criteria:
1. Participants from STAR-0215-301 who met one of the following conditions:
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:
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| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Site 2 | Scottsdale | Arizona | 85251 | United States | ||
| Site 3 |
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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.
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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.
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|
| 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 | 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. | Day 1 through Month 48 |
| Little Rock |
| Arkansas |
| 72205 |
| United States |
| Site 7 | San Diego | California | 92122 | United States |
| Site 4 | Santa Monica | California | 90404 | United States |
| Site 1 | Walnut Creek | California | 94598 | United States |
| Site 5 | Centennial | Colorado | 80112 | United States |
| Site 19 | Colorado Springs | Colorado | 80907 | United States |
| Site 21 | Wheaton | Maryland | 20902 | United States |
| Site 6 | Cincinnati | Ohio | 45236 | United States |
| Site 9 | Hershey | Pennsylvania | 17033 | United States |
| Site 22 | Santo André | 09060870 | Brazil |
| Site 20 | Sofia | 1680 | Bulgaria |
| Site 23 | Edmonton | AB T6G 2B7 | Canada |
| Site 25 | Frankfurt | D-60590 | Germany |
| Site 10 | Hong Kong | Hong Kong |
| Site 24 | Petah Tikva | 4920235 | Israel |
| Site 27 | Fukuoka | 830-0011 | Japan |
| Site 17 | Amsterdam | Netherlands |
| Site 18 | Skopje | 1000 | North Macedonia |
| Site 16 | Rzeszów | 35-051 | Poland |
| Site 11 | Cape Town | South Africa |
| Site 26 | Valencia | 46026 | Spain |
| Site 12 | Bristol | BS10 5N | United Kingdom |
| Site 8 | Cambridge | CB2 0QQ | United Kingdom |
| Site 14 | Frimley | GU16 7UJ | United Kingdom |
| Site 15 | Hampstead | NW3 2QG | United Kingdom |
| Site 13 | London | E1 2ES | United Kingdom |
| Site 14 | Plymouth | PL6 8HD | United Kingdom |
| ID | Term |
|---|---|
| D054179 | Angioedemas, Hereditary |
| D000799 | Angioedema |
| ID | Term |
|---|---|
| D014652 | Vascular Diseases |
| D002318 | Cardiovascular Diseases |
| D000081208 | Hereditary Complement Deficiency Diseases |
| D000081207 | Primary Immunodeficiency Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D014581 | Urticaria |
| D017445 | Skin Diseases, Vascular |
| D012871 | Skin Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D006969 | Hypersensitivity, Immediate |
| D006967 | Hypersensitivity |
| D007154 | Immune System Diseases |
| D007153 | Immunologic Deficiency Syndromes |
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