Patient Guide: Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM)

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Trial ID: NCTNCT04601051

Status: ACTIVE_NOT_RECRUITING

Condition: Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis

Phase: PHASE1

Find a Study Location Near You

This study is available at 4 research sites. We'll help you connect with the location that's right for you.

Participating sites include:

• Paris,
• Auckland,
• Umea,
• And 1 more locations - let us help you find the closest one

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Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2001 in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy (ATTRv-PN) and Patients With Transthyretin Amyloidosis-Related Cardiomyopathy (ATTR-CM) - Join Clinical Trial NCTNCT04601051

How to Join This Clinical Trial - NCTNCT04601051

Learn how to participate in this PHASE1 trial studying an investigational therapy for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis. Current status: ACTIVE_NOT_RECRUITING.

Am I Eligible for This Clinical Trial?

This clinical research study is looking for participants with Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis. Eligibility requirements include specific age ranges, health status, and medical history. Contact the study team to learn if you qualify.

Condition Being Studied: Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis
Treatment Being Tested: Investigational treatment
Study Phase: PHASE1 - Early safety study
Enrollment Status: ACTIVE_NOT_RECRUITING
Study Identifier: NCTNCT04601051 - ClinicalTrials.gov Identifier
Sponsored By: Study sponsor

What to Expect as a Participant

Participating in this clinical trial involves:

Initial screening to determine eligibility
Regular study visits and health assessments
Receiving the study treatment or placebo
Medical monitoring and follow-up care
Contributing to medical research that may help others
Potential access to new treatments before they're widely available

Clinical Trial Benefits and Compensation

Participants in this clinical research study may receive:

Close medical monitoring by healthcare professionals
Access to potential new treatments
Compensation for time and travel (varies by study)
No-cost study-related medical care
The opportunity to help advance medical knowledge

Where Is This Clinical Trial Located?

This study is enrolling participants at 4 locations. Find a study site near you:

Clinical Research Site

Paris, - France

Status: Contact for availability

Clinical Research Site

Auckland, - New Zealand

Status: Contact for availability

Clinical Research Site

Umea, - Sweden

Status: Contact for availability

Clinical Research Site

London, - United Kingdom

Status: Contact for availability

How to Enroll in This Study

To learn more about participating in this PHASE1 clinical trial for Transthyretin-Related (ATTR) Familial Amyloid Polyneuropathy, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Wild-Type Transthyretin Cardiac Amyloidosis:

Review the eligibility criteria with your healthcare provider
Contact the study team for a pre-screening interview
Schedule an in-person screening visit if eligible
Review and sign the informed consent form
Begin participation in the clinical trial

Why Choose Quri.ai as Your Clinical Trial Matching Service

Quri.ai is a free clinical trial matching service that helps patients connect with research sites. We make it easier to:

Find clinical trials that match your condition
Get matched to the right study location near you
Connect directly with study coordinators
Understand if you qualify with simplified eligibility screening
Navigate the enrollment process with personalized support
Access new treatments through clinical research participation