🏛️ Verified NIH Record • Updated 2026-02-06
Berotralstat Treatment in Children With Hereditary Angioedema (NCT05453968)
A Phase 3 Study to Evaluate the Safety and Pharmacokinetics of Berotralstat Prophylaxis in Children With Hereditary Angioedema Who Are 2 to < 12 Years of Age
🔵 Active, Not Recruiting Phase3 💊 Investigational Drug / Biologic Sponsor: BioCryst Pharmaceuticals
🩺 Plain-English Summary (8th-Grade Level)
The purpose of this study is to evaluate the pharmacokinetics (PK), safety and effectiveness of berotralstat to determine the appropriate weight-based dose for pediatric participants 2 to \< 12 years of age for prophylactic treatment to prevent attacks of hereditary angioedema (HAE).
Who can join: Review the eligibility criteria below.
Location: Investigative Site #1 — Vienna, Ontario
Age range: 2 Years to 11 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 (15 Sites)
- Investigative Site #1 — Vienna, Ontario
- Investigative Site #1 — Ottawa, Ontario
- Investigative Site #3 — Grenoble,
- Investigative Site #2 — Marseille,
- Investigative Site #1 — Paris,
- Investigative Site #1 — Berlin,
- Investigative Site #2 — Frankfurt,
- Investigative Site #2 — Haifa,
- Investigative Site #1 — Tel Aviv,
- Investigative Site #1 — Padova,
- Investigative Site #1 — Krakow,
- Investigative Site #1 — Sângeorgiu de Mureş,
- Investigative Site #1 — Madrid,
- Investigative Site #2 — Málaga,
- Investigative Site #1 — Bristol,
💰 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
- A Study to Assess the Long-Term Safety and Efficacy of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE) — Paradise Valley, Arizona
- 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
- 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.