Promising drug combo targets Hard-to-Treat amyloidosis

NCT ID NCT05451771

First seen Jun 27, 2026 · Last updated Jul 10, 2026 · Updated 1 time

Summary

This study tests a combination of two drugs, venetoclax and dexamethasone, in people with a rare blood disorder called AL amyloidosis that has come back or not improved after treatment. The trial includes 53 adults with a specific genetic marker (t(11;14)). The goal is to find the safest dose and see how well the drugs work at reducing abnormal proteins and improving organ function.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
Venetoclax (Venclexta) and dexamethasone
What this could lead to
If successful, this could offer a new treatment option for people with a specific genetic type of AL amyloidosis that has not responded to prior therapy.
What could go wrong
This is an early-phase trial with only 53 participants, so results may not apply broadly. Side effects like low blood counts and tumor lysis syndrome are possible.

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Conditions

The condition(s) this trial relates to.

AL amyloidosis Immunoglobulin Light-chain Amyloidosis proteostasis deficiencies

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Contacts and locations

Locations

  • Boston Medical Center

    Boston, Massachusetts, 02118, United States

  • Froedtert Hospital & the Medical College of Wisconsin

    Milwaukee, Wisconsin, 53226, United States

  • Mayo Clinic Rochester

    Rochester, Minnesota, 55905, United States

  • New York Presbyterian Hospital/Columbia University Irving Medical Center

    New York, New York, 10032, United States

  • Washington University School of Medicine

    St Louis, Missouri, 63110, United States

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