Hereditary Amyloidosis
Hereditary Amyloidosis is a rare genetic condition where abnormal proteins build up in tissues and organs, leading to damage. It can affect the nerves, causing neuropathy, or the heart, leading to cardiac issues.
Long-Term Safety Study of NTLA-2001
We are evaluating the long-term safety and effects of NTLA-2001 in participants who have already received this treatment. This study aims to understand how it impacts their health over time.
What diseases are being studied?
Health conditions and diseases that the clinical trial is designed to study and treat.
Who can participate in the clinical trial?
These questions help us understand your situation so we can let the trial team know whether you might be a potential match. This is not a medical evaluation and is not part of the official screening, the study doctor will make the final decision..
- Are you willing to attend study visits and follow the study requirements?
- Are you a male who can agree to not donate sperm for 4 months after the last treatment?
- Are you a female who can agree to use a highly effective method of contraception for 7 months after the last treatment?
- Are you a female who is either postmenopausal or surgically sterile?
- Have you completed or discontinued from an Intellia-sponsored clinical study where you received NTLA-2001?
What experimental substances are being tested?
Experimental substances that are being investigated in this clinical trial. These are not yet approved for general use.
Ntla-2001
Single Guide Rna
What similar medicines might you know?
Don't worry if you don't know the medicines tested in the trial. There is a chance you have heard about the similar medicines. Check the list below to see if you are familiar with any of them.
Almus Gliclazide Teva Uk
Clazicon
Dacadis Mr
Where is the clinical trial being conducted?
Locations and medical facilities where this clinical trial is currently recruiting or conducting research. Select the locations where you would like to participate.
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Assistance Publique Hopitaux De Paris
Neurology
Croulebarbe, France
Region Vaesterbotten
Norrlands universitetssjukhus, Daniel Naezéns väg, 907 37 Umeå, Medicincentrum E:41
Umeå, Sweden
Sponsor: Intellia Therapeutics Inc.
Last updated: Nov 27, 2025
Disclaimer: Parts of this content have been automatically extracted from the EU Clinical Trials registry. While we strive for accuracy, please always contact the trial site or sponsor directly for correct and official information before making any decisions about participation. View on EU Clinical Trials.