Actively Recruiting
Long-Term Follow-Up of Subjects Treated With NTLA-2001 for Transthyretin-Related Amyloidosis
Led by Intellia Therapeutics · Updated on 2025-08-26
72
Participants Needed
4
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to observe the long-term effects of the investigational therapy NTLA-2001 in individuals who previously received this treatment in an earlier clinical trial. The study focuses on patients with transthyretin-related conditions, including familial amyloid polyneuropathy, familial amyloid cardiomyopathy, and wild-type transthyretin cardiac amyloidosis. Participants who received at least a partial dose of NTLA-2001 in a prior Intellia Therapeutics clinical study are followed observationally in this study. There are no new treatments or interventions administered during this follow-up. The study duration may extend up to 15 years to monitor outcomes. During the follow-up, researchers will track the occurrence of serious adverse events related to the treatment and specific adverse events of special interest. They will also measure biomarkers related to transthyretin amyloidosis, such as serum TTR and serum prealbumin, to better understand long-term changes. Participant involvement includes periodic evaluations and data collection over the study period, with no active treatment provided.
CONDITIONS
Brief Title
Long-Term Follow-Up (LTFU) of Subjects Dosed With NTLA-2001
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Completed or discontinued from an Intellia-sponsored clinical study with a full or partial dose of NTLA-2001
- Provided informed consent for this long-term follow-up study
You will not qualify if you...
- None specified in the study criteria
AI-Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 15 years
Participants who previously received NTLA-2001 are observed over time to monitor safety and biomarkers related to transthyretin-related amyloidosis.
Trial Site Locations
Total: 4 locations
1
Clinical Trial Site
Paris, France
Actively Recruiting
2
Clinical Trial Site
Auckland, New Zealand
Actively Recruiting
3
Clinical Trial Site
Umeå, Sweden
Actively Recruiting
4
Clinical Trial Site
London, United Kingdom
Actively Recruiting
Research Team
T
Trial Manager at Intellia
How is the study designed?
Study Type
OBSERVATIONAL
Masking
N/A
Allocation
N/A
Model
N/A
Primary Purpose
N/A
Number of Arms
0
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