🏛️ Verified NIH Record • Updated 2025-12-05
A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) (NCT05392114)
An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: Ionis Pharmaceuticals, Inc.
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the long-term safety and efficacy of donidalorsen in people with HAE and the effects of donidalorsen on the number of HAE attacks and their impact on quality of life (QoL).
Who can join: Review the eligibility criteria below.
Location: Ionis Investigative Site — Paradise Valley, Arizona
Age range: 12 Years
🧪 Interventions in This Study
📋 "Do I Qualify?" — 1-Minute Self Screener
Instant ChecklistFull eligibility criteria are available on the official NIH protocol. View the criteria ↗
📍 All Participating Trial Locations (50 Sites)
Showing the first 25 of 50 sites.
- Ionis Investigative Site — Paradise Valley, Arizona
- Ionis Investigative Site — La Jolla, California
- Ionis Investigative Site — Santa Monica, California
- Ionis Investigative Site — Walnut Creek, California
- Ionis Investigative Site — Tampa, Florida
- Ionis Investigative Site — Kansas City, Kansas
- Ionis Investigative Site — Boston, Massachusetts
- Ionis Investigative Site — Ann Arbor, Michigan
- Ionis Investigative Site — Detroit, Michigan
- Ionis Investigative Site — St Louis, Missouri
- Ionis Investigative Site — Mooresville, North Carolina
- Ionis Investigative Site — Cincinnati, Ohio
- Ionis Investigative Site — Columbus, Ohio
- Ionis Investigative Site — Hershey, Pennsylvania
- Ionis Investigative Site — Dallas, Texas
- Ionis Investigative Site — Murray, Utah
- Ionis Investigative Site — Edegem,
- Ionis Investigative Site — Sofia,
- Ionis Investigative Site — Sofia,
- Ionis Investigative Site — Ottawa, Ontario
- Ionis Investigative Site — Edmonton,
- Ionis Investigative Site — La Tronche,
- Ionis Investigative Site — Marseille,
- Ionis Investigative Site — Paris,
- Ionis Investigative Site — Berlin,
💰 Cost, Insurance & Patient Rights
- 100% Free Items: The investigational treatment and protocol-required procedures are supplied at zero cost by the sponsor.
- Routine Patient Care: Under federal law (ACA § 2709) and many state statutes, routine care costs are covered during participation.
- Voluntary Participation: You may withdraw at any time without affecting your standard medical care.
More studies in Hereditary Angioedema
- A Gene Therapy Study of BMN 331 in Subjects With Hereditary Angioedema — Birmingham, Alabama
- HAELO: A Phase 3 Study to Evaluate NTLA-2002 in Participants With Hereditary Angioedema (HAE) — Scottsdale, Arizona
- A Long-term Study of STAR-0215 in Participants With Hereditary Angioedema — Birmingham, Alabama
- Berotralstat Treatment in Children With Hereditary Angioedema — Vienna, Ontario
- Safety and Effectiveness of Sebetralstat (KVD900) for Short-Term Prophylaxis Before Procedures in People With Hereditary Angioedema (KONTROL) — Colorado Springs, Colorado
Explore by condition: All Hereditary Angioedema clinical trials
💡 How to Participate
- Check the checklist above to see if you may qualify.
- Review the official NIH record below to find the coordinating site.
- Ask about the visit schedule, what is covered at no cost, and any travel support.
- Confirm with your own doctor before making a decision.