Helios-B (Cardiac Amyloidosis) - Clinical Trial

What is the Purpose of this Study?

We are doing this study to find out if an experimental drug called Vutrisiran 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 18-85 who:
- Have hereditary or wild-type cardiac amyloid with cardiomyopathy
- Can walk more than 150 meters during a 6-minute walk test
- Have not been treated with patisiran or inotersen
- Are not participating in any other clinical drug trial

For more information about who can join the study, please contact the study team at 919-681-7835.

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
- Give urine samples
- 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 (Vutrisiran) every 3 months as an injection under the skin
-- The other group will get a placebo (inactive substance) every 3 months
- 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
- Take a daily dose of Vitamin A

You will take the study drug or placebo for up to 36 months. The follow-up phase will consist of study visits every 12 weeks for a year after the last dose of the study drug. At these visits you will:
- Have blood draws
- Give urine samples
- Have vital signs like heart rate, blood pressure, and breathing rate taken

Study Details

Full Title
HELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)
Principal Investigator
Protocol Number
IRB: PRO00104273
NCT: NCT04153149
Phase
Phase III
ClinicalTrials.gov
Enrollment Status
Open for Enrollment
Contact the Duke Recruitment Innovation Center
MyResearchPartners@duke.edu
or
919-681-5698