NTLA-2001 for ATTR-CM

Recruiting at 94 trial locations

Overseen By Trial Manager at Intellia

Age: 18+

Sex: Any

Travel: May Be Covered

Time Reimbursement: Varies

Trial Phase: Phase 3

Recruiting

Sponsor: Intellia Therapeutics

Must not be taking: RNA silencers, Tafamidis

Disqualifiers: NYHA Class IV HF, Hepatitis, HIV, others

Pivotal Trial (Near Approval)

Prior Safety Data

Trial Summary

What is the purpose of this trial?

To evaluate the efficacy and safety of a single dose of NTLA-2001 compared to placebo in participants with ATTR-CM.

Will I have to stop taking my current medications?

The trial protocol does not specify if you need to stop taking your current medications, but you cannot have used RNA silencer therapy like patisiran, inotersen, or eplontersen within the last 12 months, and you cannot start tafamidis or acoramidis within 56 days before the study.

Eligibility Criteria

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.

Inclusion Criteria

My heart failure symptoms are stable and well-managed for the last 28 days.

I have a history of heart failure.

I have been diagnosed with ATTR amyloidosis affecting my heart.

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive a single intravenous infusion of NTLA-2001 or placebo

1 day

1 visit (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

12 months

Treatment Details

Interventions

• NTLA-2001 (Gene Editing Therapy)

Trial Overview 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.

Participant Groups

Group I: NTLA-2001 Experimental Treatment 1 Intervention

Single intravenous (IV) infusion of NTLA-2001

Group II: Placebo Placebo Group 1 Intervention

Single IV infusion of normal saline

Who Is Running the Clinical Trial?

Intellia Therapeutics

Lead Sponsor

Trials

10

Recruited

1,300+

Regeneron Pharmaceuticals

Industry Sponsor

Trials

690

Recruited

948,000+

Founded

1988

Headquarters

Tarrytown, USA

Known For

Precision medicine

Top Products

Dupixent, EYLEA, Libtayo, Praluent

Leonard Schleifer

Regeneron Pharmaceuticals

Chief Executive Officer since 1988

MD and PhD in Medicine

George Yancopoulos

Regeneron Pharmaceuticals

Chief Medical Officer since 1997

MD from Harvard Medical School