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Gene Editing Therapy

NTLA-2001 for ATTR-CM

Phase 3
Recruiting
Research Sponsored by Intellia Therapeutics
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Medical history of heart failure (HF)
Symptoms of HF are optimally managed and clinically stable within 28 days prior to administration of study intervention
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years
Awards & highlights
Pivotal Trial

Summary

This trial is testing if a single dose of NTLA-2001 is effective and safe in people with ATTR-CM, a heart condition.

Who is the study for?
This trial is for people with a heart condition called ATTR-CM, which involves abnormal protein deposits in the heart. Participants must have a confirmed diagnosis, stable heart failure symptoms, and specific levels of NT-proBNP (a heart failure marker) in their blood.
What is being tested?
The study is testing NTLA-2001's effectiveness and safety against a placebo. It's given as a single dose to see if it can help patients with ATTR-CM. The trial randomly assigns participants to receive either NTLA-2001 or placebo.
What are the potential side effects?
While the side effects of NTLA-2001 are not detailed here, common risks may include injection site reactions, potential allergic responses, and possible unforeseen impacts on organs where amyloid deposits might be affected.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have a history of heart failure.
Select...
My heart failure symptoms are stable and well-managed for the last 28 days.
Select...
I have been diagnosed with ATTR amyloidosis affecting my heart.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups
Experimental Treatment
Placebo Group
Group I: NTLA-2001Experimental Treatment1 Intervention
Single intravenous (IV) infusion of NTLA-2001
Group II: PlaceboPlacebo Group1 Intervention
Single IV infusion of normal saline

Find a Location

Who is running the clinical trial?

Intellia TherapeuticsLead Sponsor
9 Previous Clinical Trials
557 Total Patients Enrolled
Regeneron PharmaceuticalsIndustry Sponsor
671 Previous Clinical Trials
385,737 Total Patients Enrolled
~510 spots leftby Dec 2027
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