← Back to Search

Gene Editing Therapy

NTLA-2001 for Transthyretin Amyloidosis

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 maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months

Awards & highlights

Study 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.Check my eligibility

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.See study design

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 weeks3 visits

Treatment ~ Varies

Follow Up ~ maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months

This trial's timeline: 3 weeks for screening, Varies for treatment, and maximum study duration is dependent on event rates and is estimated to be at least 18 months and up to 48 months for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Composite outcome of cardiovascular (CV) mortality and CV events

Secondary outcome measures

Change from baseline to month 18 in KCCQ-OS score

Change in baseline to month 18 in serum TTR

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

0 / 3Eligibility criteria met

Find a Location

Who is running the clinical trial?

Intellia TherapeuticsLead Sponsor

6 Previous Clinical Trials

417 Total Patients Enrolled

Regeneron PharmaceuticalsIndustry Sponsor

621 Previous Clinical Trials

379,840 Total Patients Enrolled

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Are prospective patients still eligible to participate in this ongoing medical study?

"Indeed, according to the information available on clinicaltrials.gov, this trial is actively seeking participants. The original posting date for the trial was December 13th, 2023 and it was last modified on January 12th, 2024."

Answered by AI

What is the total number of participants being selected for enrollment in this clinical trial?

"Indeed, the information available on clinicaltrials.gov confirms that this specific clinical trial is actively seeking eligible participants. The study was initially posted on December 13th, 2023 and last updated on January 12th, 2024. A total of 765 individuals will be enrolled in this trial across two designated locations."

Answered by AI

Which specific criteria determine the inclusion of individuals into this research study?

"To be eligible for this trial, individuals must have a diagnosis of transthyretin amyloidosis and fall within the age range of 18 to 90. The study aims to recruit a total of 765 participants."

Answered by AI

Is there an age limit that excludes individuals below 45 years from participating in this research?

"This research is seeking to include individuals who are above 18 years of age but below the age of 90."

Answered by AI

Has the FDA granted approval for the use of NTLA-2001 in medical treatment?

"Based on the phase of this trial, which is Phase 3, our team at Power rates the safety of NTLA-2001 as a 3. This rating indicates that there is existing data supporting both its efficacy and multiple rounds of safety information."

Answered by AI

Who else is applying?

What site did they apply to?

University of Kentucky Gill Heart Institute

What portion of applicants met pre-screening criteria?

Met criteria

Why did patients apply to this trial?

I recently noticed that you are a site for the MAGNITUDE study for NTLA-2001. This is the trial I am interested in. I’ve been searching for any clinical trials that might benefit my condition, ATTR-CM. Your MAGNITUDE trial seems like the perfect solution to stop the formation of the mal-folded proteins, since it eliminates the protein. I am desperately looking for the solution your trial can provide. I hope to hear from you soon. Thank you for your help. Eugene Mikelonis 513-674-2701 genemike@yahoo.com.

PatientReceived 2+ prior treatments

Recent research and studies

clinicaltrials.govStudy

MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)

2023. "MAGNITUDE: A Phase 3 Study of NTLA-2001 in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR-CM)". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT06128629.

All > Cardiomyopathy > Amyloidosis