Caelum: Ph3 of CAEL-101 (Cardiac Amyloidosis)

What is the Purpose of this Study?

We are doing this study to see if an experimental drug called CAEL-101 can help people live longer with AL amyloidosis that has affected their hearts.

What is the Condition Being Studied?

AL Amyloidosis Mayo Stage IIIb

Who Can Participate in the Study?

Adults diagnosed with AL amyloidosis that has affected their heart function.

For more information about who can join this study, please contact the study team at 919-684-9220.

Age Group

What is Involved?

This study has three periods: screening, dosing, and follow-up.

If you choose to join this study, you will go through a screening period to find out if you are eligible. During this screening period, you will:
- Have a physical exam
- Have heart scans (ECG and echocardiogram)
- Have blood draws and give urine samples

If you are eligible to continue, you will:
- Be randomly assigned (like a coin flip) to 1 of 2 groups
-- One group will get infusions (IV) of the study drug
-- The other group will get a placebo (inactive substance)
- Get the study drug once a week for 4 weeks and then every other week for about a year

In addition to taking the study drug or placebo, your study doctor will treat you with anti-AL amyloidosis medications called CyBorD. Your CyBorD therapy will be tailored to your needs.

Once you stop taking the study drug, your study team will ask you to return approximately 14 days after your last IV infusion. You will then enter the follow-up phase and the study staff will contact you approximately every 12 weeks to see how you are doing.

Study Details

Full Title
A Phase 3, Double-Blind, Multicenter Study to Evaluate the Efficacy and Safety of CAEL-101 and Plasma Cell Dyscrasia Treatment Versus Placebo and Plasma Cell Dyscrasia Treatment in Plasma Cell Dyscrasia Treatment-Naïve Patients with Mayo Stage IIIb AL Amyloidosis
Principal Investigator
Assistant Professor of Medicine
Protocol Number
IRB: PRO00106175
NCT: NCT04504825
Phase III
Enrollment Status