Experimental drug shows promise for rare amyloidosis

NCT ID NCT04617925

Completed Disease control Sponsor: Stichting European Myeloma Network Source: ClinicalTrials.gov ↗

First seen Jun 25, 2026 · Last updated Jul 31, 2026 · Updated 2 times

Summary

This phase 2 study tested a drug called belantamab mafodotin in 35 people with AL amyloidosis, a rare disease where abnormal proteins build up in organs. Participants had already tried other treatments without success. The goal was to see if the drug could reduce or eliminate those harmful proteins. Researchers also monitored side effects closely.

What this could mean

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

Active substance: belantamab mafodotin
What this could lead to: If successful, this could provide a new treatment option for people with AL amyloidosis whose disease has returned or not responded to prior therapy.
What could go wrong: This is a small, early-phase trial with only 35 participants, so results may not apply broadly. The drug can cause eye problems and other side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

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Conditions

The condition(s) this trial relates to.

AL amyloidosis Immunoglobulin Light-chain Amyloidosis

As listed by the trial registrant

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

AL AMYLOIDOSIS

Contacts and locations

Locations

Centre hospitalier Universitaire de Limoges -

Limoges, 87042, France
Fondazione I.R.C.C.S Policlinico "San Matteo"

Pavia, 27100, Italy
General Hospital of Athens "Alexandra"

Athens, 115 28, Greece
Royal Free Hospital - London,

London, United Kingdom
UMC Utrecht

Utrecht, 3584 CX, Netherlands
University Hospital Heidelberg

Heidelberg, 69120, Germany

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