Testing a new drug for hard-to-treat protein disease affecting organs

Plain-English translation of NCT06292780 on ClinicalTrials.gov ↗ · Source last updated June 2026 · Translation generated July 2026 · How we translate trials

Phase 1/2 — A combined trial that checks safety and dosing while also starting to look at whether the treatment works.

What is this trial?

This trial is testing an experimental medication called in people with AL amyloidosis (a rare disease where harmful proteins build up in the heart, kidneys, and other organs) that has either returned or stopped responding to previous treatments. The study has two phases: first, researchers will give the medication to a small group to see how safe it is and find the best dose, then to a larger group to confirm safety and measure how well it works.

Many people with AL amyloidosis run out of treatment options when their disease comes back or stops responding to standard therapies. This trial exists to see whether this new medication could offer hope for patients with limited alternatives.

Do you qualify?

You likely qualify if…

you have been diagnosed with AL amyloidosis that has either come back or stopped responding to at least one previous treatment
you have measurable signs of the disease in your blood (abnormal free light chains) that doctors can track
you have adequate organ function (heart, kidney, liver, and blood counts are healthy enough to participate)
you are in reasonably good overall health and able to care for yourself with minimal assistance (ECOG score 2 or better)
you are not having severe heart stress during screening (NT-proBNP level 8500 ng/L or lower)

You likely don't qualify if…

you have had a type of amyloidosis other than AL amyloidosis
you have had a heart attack in the past 6 months
you have an active infection requiring hospitalization or IV antibiotics within the past month
you have bone damage (lytic lesions) or tumors outside the bone marrow visible on imaging
you have more than 60% abnormal plasma cells on a recent bone marrow test

What participation looks like

In Phase 1, you would receive the medication at carefully controlled doses while doctors closely monitor you for side effects and determine the best dose to use. In Phase 2, you would be randomly assigned to receive either a lower or higher dose, and you would continue receiving the medication while doctors track whether your abnormal proteins decrease and whether your heart and kidney function improve. Throughout the study, you'll have regular blood tests, heart and kidney function checks, and visits to measure how you're responding to treatment.

Testing a new drug for hard-to-treat protein disease affecting organs

What is this trial?

Do you qualify?

What participation looks like

Trial locations(20 sites)