"I was delighted to take part in a clinical trial as it has the potential to really help others in the future.”
A study of carfilzomib with thalidomide and dexamethasone for relapsed amyloidosis (Catalyst)
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.
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.
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.
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.
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.
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 (
How to join a clinical trial
Dr Ashutosh Wechalekar
University College London
University of Leeds Clinical Trials Research Unit