Plain-English translation of NCT05697861 on ClinicalTrials.gov ↗ · Source last updated · Translation generated · How we translate trials
This is a follow-up study for people who have already received NTLA-2001, an experimental gene-editing treatment for rare diseases affecting the nerves or heart. Researchers want to check in with you over time to see how the medication continues to work and whether any long-term effects develop. You would not receive any new doses of the treatment in this study.
Gene-editing treatments are very new, and doctors need to understand how patients fare months and years after receiving them. This study helps researchers track whether this medication keeps working, whether any delayed side effects occur, and how it affects your quality of life in the long run.
You likely qualify if…
You likely don't qualify if…
You would be asked to return for periodic visits so doctors can monitor your health and assess how you are doing after your previous treatment. This is an observational study, meaning researchers are watching and gathering information rather than giving you new medications or procedures. The study team will review your medical records and may conduct routine health assessments to track long-term outcomes.
AI-generated summary from trial data · Jun 13, 2026 · Not medical advice
France
New Zealand
Sweden
United Kingdom
Sponsor
Intellia Therapeutics
Enrollment target
~72 participants
Started
July 2023
Primary completion
March 2038
Age range
18 Years and older
Last updated on clinicaltrials.gov in August 2025.
Reach out to the team running this trial. Response times vary — some teams are faster than others.
Central contact
Trial Manager at Intellia
Intellia Therapeutics
Tell us you're interested and we'll help connect you with the research team. We'll walk you through what to expect first — no email needed to get started.