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 at 919-681-2723.

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