CardioTTRansform (Cardiac Amyloidosis) - Clinical Trial

What is the Purpose of this Study?

We are doing this study to find out if an experimental drug called ION-682884 is safe and helpful for people with cardiac amyloidosis.

What is the Condition Being Studied?

Cardiac Amyloidosis

Who Can Participate in the Study?

Adults ages 18-90 who:
- Have cardiac amyloid confirmed by biopsy or positive scintigraphy scan
- Can walk more than 150 meters during a 6-minute walk test
- Are willing to be genetically tested for hereditary or wild-type amyloid if not previously done
- Have not received a heart or liver transplant and do not plan to do so within the next year

For more information about who can join the study, please contact the study team at919-681-2723.

Age Group
Adults

What is Involved?

The study is divided into three phases: screening, double-blind treatment, and follow-up.

If you choose to join the study, during the screening phase you will:
- Make 1 or 2 visits to our clinic
- Have a physical exam
- Have a 6-minute walk test
- Have blood draws
- Be genetically tested for hereditary or wild type cardiac amyloid (if not done previously)
- Have a cardiac biopsy or scintigraphy scan done (if not done previously)
- Give a urine sample
- Have a scan of your heart (ECG)

If you are eligible to continue after screening, you will begin the double-blind treatment phase. During this phase, you will:
- Be randomly assigned (like a coin flip) to 1 of 2 groups
-- One group will get the study drug (ION-682884) once a month as an injection under the skin
-- The other group will get a placebo (inactive substance) once a month
- Visit our clinic every 3 months for check-ups
- Answer questionnaires, give urine samples, and have blood draws, walking tests, and ECG's done during these visits
- Be visited by the study's home health nurse once a month for blood draws

You will take the study drug or placebo for up to 30 months. The follow-up phase will consist of 2 visits to our clinic after the treatment period ends. These visits will be 10 weeks apart from each other.

Study Details

Full Title
A Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients with Transthyretin-Mediated Amyloid Cardiomyopathy
Principal Investigator
Protocol Number
IRB: PRO00105193
NCT: NCT04136171
Phase
Phase III
ClinicalTrials.gov
Enrollment Status
Open for Enrollment
Contact the Duke Recruitment Innovation Center
MyResearchPartners@duke.edu
or
919-681-5698