A study of carfilzomib with thalidomide and dexamethasone for relapsed amyloidosis (Catalyst)

Cancer type:

Acute leukaemia
Blood cancers
Leukaemia

Status:

Results

Phase:

Phase 1

This study was done to find the best dose of carfilzomib to have with thalidomide and dexamethasone for amyloidosis.  It was for people with amyloid light chain (AL) amyloidosis that had come back after treatment.

The study was open for people to join between 2017 and 2019. The team analysed the results in 2019.

More about this trial

Amyloidosis is not cancer. But it is sometimes linked to a type of cancer called myeloma. And doctors often use the same treatment as they do for myeloma.

There are cells called plasma cells in our bone morrow. These cells make proteins. When someone has amyloidosis, the plasma cells make abnormal proteins. These proteins build up in the body and can cause symptoms.

The aim of treatment is to remove the plasma cells that make the abnormal protein. 

When this study was done, the standard treatment was thalidomide and dexamethasone. Researchers wanted to see if it was useful to give carfilzomib as well. 

Carfilzomib is a type of targeted cancer drug called a proteasome inhibitor.

The main aims of the study were to find out:

  • the best dose of carfilzomib to have with thalidomide and dexamethasone
  • if carfilzomib is a useful treatment for AL amyloidosis that has come back after treatment 

Summary of results

The research team found the best dose of carfilzomib to have with thalidomide and dexamethasone for amyloidosis.

Study design
This study was for people with amyloid light chain (AL) amyloidosis. The build up of abnormal protein was affecting at least one of their major body organs, such as the heart or kidneys.

They had all had treatment already, but the amyloidosis had come back or got worse.

Results
A total of 10 people joined this study. They had all had thalidomide and dexamethasone, and:

  • 3 people had a lower dose of carfilzomib
  • 7 people had a higher dose of carfilzomib


The research team looked at how many people had a lower number of plasma cells at the end of treatment. They found it was:

  • 2 out of 3 people who had the lower dose of carfilzomib
  • 3 out of 7 people who had the higher dose of carfilzomib

They also looked at whether symptoms had improved in people who had a build up of protein in specific organs of the body. They found that this didn’t happen in anyone in either group.

Side effects
The research team looked at the side effects people had.  People in both groups had some side effects, but most were mild or didn’t last long. Some had side effects that were a bit worse:

  • 2 out of 3 people in the lower dose group
  • 3 out of 7 people in the higher dose group

No one in the lower dose group had a side effect bad enough to suggest the dose of carfilzomib was too high. One person in the higher dose group had a more serious side effect.

Conclusion
The research team concluded that the lower dose of carfilzomib was the best dose to use for amyloidosis. But it’s hard to say how well treatment worked because of the small number of people in the study. 

They suggest that more trials are done to find out whether carfilzomib is a useful treatment for amyloidosis.

Where this information comes from    
We have based this summary on information from the research team. As far as we are aware, the information they sent us has not been reviewed independently (peer reviewed Open a glossary item) or published in a medical journal yet. The figures we quote above were provided by the research team. We have not analysed the data ourselves.
 

Recruitment start:

Recruitment end:

How to join a clinical trial

Please note: In order to join a trial you will need to discuss it with your doctor, unless otherwise specified.

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

Chief Investigator

Dr Ashutosh Wechalekar

Supported by

University College London
University of Leeds Clinical Trials Research Unit
Amgen 

Freephone 0808 800 4040

Last review date

CRUK internal database number:

14491

Please note - unless we state otherwise in the summary, you need to talk to your doctor about joining a trial.

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