Clinical research
Being tested in people in registered clinical trials. Being in trials is not evidence that a treatment works or is safe.
What this trial is testing
The earlier trial of the hereditary angioedema treatment, testing whether a single infusion could reduce swelling attacks. Its results were published in a major medical journal and supported the move into the Phase 3 study that followed.
A Phase 1/2 study of NTLA-2002 in adults with hereditary angioedema, evaluating safety, pharmacodynamics and attack rate following a single dose. Results were published in the New England Journal of Medicine and supported progression to the Phase 3 HAELO study.
Sources
- New England Journal of Medicine · 2024
CRISPR-based therapy for hereditary angioedema ↗
What a trial result does and does not mean
Early-phase results come from small numbers of participants and short follow-up. They can change with more people and more time, and a trial that is recruiting, active or even completed has not established that a treatment works. Enrolment criteria are strict; eligibility is decided by the trial site, never by a website.