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

Cancer type:

Acute leukaemia
Blood cancers
Leukaemia

Status:

Open

Phase:

Phase 1

This trial is comparing different doses of carfilzomib to have with thalidomide and dexamethasone for amyloidosis that has come back after treatment.

The trial is for people who have a type of amyloidosis called amyloid light chain (AL) amyloidosis.

More about this trial

Amyloid light chain (AL) amyloidosis is when an abnormal protein is made in the bone marrow by plasma cells. These abnormal proteins build up in the tissues and change the way they work. This can cause symptoms.

Treatment aims to remove the plasma cells that make the abnormal protein. Thalidomide and dexamethasone is standard treatment for AL amyloidosis.

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

This trial is combining carfilzomib with thalidomide and dexamethasone.

This trial aims to find out:

  • if carfilzomib is safe in people with AL amyloidosis that has come back after treatment (relapsed or refractory AL amyloidosis)
  • the best dose of carfilzomib to have with thalidomide and dexamethasone
  • about the side effects and how these drugs affect quality of life 

Who can enter

The following bullet points list the entry conditions for this trial. Talk to your doctor or the trial team if you are unsure about any of these. They will be able to advise you.

Who can take part
You may be able to join this trial if all of the following apply. You:

  • have been diagnosed with amyloidosis without any genetic changes (mutations)
  • amyloidosis is affecting a major organ
  • have disease that did not respond to or came back after chemotherapy or a stem cell transplant
  • have satisfactory blood test results
  • are well enough to be up and about for at least half of the day (performance status of 0,1 or 2)
  • are aged 18 or over
  • are willing to use contraception for 30 days before the trial, during the trial and for up to 90 days afterwards

Who can’t take part
You cannot join this trial if any of these apply.

Cancer related exclusions
You:

  • have multiple myeloma
  • have a type of amyloidosis other than amyloid light chain (AL) amyloidosis
  • amyloidosis is only affecting one organ
  • have disease that has not responded to treatment with another proteasome inhibitor
  • have disease that has come back after treatment with another proteasome inhibitor
  • had a stem cell transplant using the stem cells of another person (allogeneic transplant)
  • have another type of cancer apart from basal cell carcinoma of the skin or carcinoma in situ (such as carcinoma in situ of the cervix) that was successfully treated

Medical condition exclusions
You:

  • have had an organ transplant
  • have severe tingling and numbness in fingers or toes (peripheral neuropathy)
  • have severe heart problems such as heart failure, pulmonary hypertension, unstable angina or history of heart attack within the last 6 months, or if amyloidosis is affecting your heart.
  • have a wide-spread infection needing treatment
  • have or are thought to have HIV (Human Immunodeficiancy Virus) infection
  • have Hepatitis B

Other exclusions

  • are pregnant or breastfeeding
  • have any physical or mental health problem that the trial doctors feel might affect you taking part in the trial
  • have taken part in a trial using an experimental drug in the last 3 months
  • have a known allergy to carfilzomib, thalidomide or dexamethasone

Trial design

This is a phase 1 study. The trial team need about 18 people to take part.

There are 2 parts to this study. This summary is about the first part of the study. This is called the dose escalation part of the study.

Part 1
The first few people to take part will have a low dose of carfilzomib and the standard treatment dose of thalidomide and dexamethasone. If they don’t have any bad side effects the next few people will have a higher dose. And so on until the highest safe dose is found. This is a dose escalation study.

Part 2
When researchers find the highest safe dose they plan to start part 2 of the trial. They will need another 20 people to take part. Part 2 will study the side effects of the highest dose of carfilzomib. And will look at how well this combination of treatment works. This is a dose expansion study.

Carfilzomib is a drip into your blood stream. Thalidomide and dexamethasone are tablets. The drugs are given as cycles of treatment. Each cycle is 28 days. You might have 1 – 6 cycles of treatment.

You have carfilzomib and dexamethasone on day 1, 8, and 15 of each cycle. You have thalidomide every day. 

Hospital visits

You see the doctors and have some tests before taking part.

These tests include:

  • blood tests (including for Hepatitis B)
  • bone marrow tests
  • heart trace (ECG Open a glossary item)
  • heart scan  (Echo Open a glossary item)
  • pregnancy test (if applicable)
  • physical examination
  • assessment of your medical history

You will go to your nearest regional haematology centre for treatment on day 1, 8 and 15 of each cycle.

Before each cycle of treatment you have: 

  • a physical examination
  • a pregnancy test (if applicable)
  • blood tests to check you are well enough for the next dose
  • a blood sample for researchers to use to learn about the cancer and how carfilzomib works

And before you have treatment on days 8 and 15 you have blood a blood test to check you are well enough to have it.

After 3 cycles of treatment you will visit the National Amyloidosis centre for blood tests and an echocardiogram. This looks at how the treatment is working.

You will continue with treatment for up to 3 more cycles if your treatment is working. You will have the same tests as you have had in the previous 3 cycles of treatment.

If the treatment isn’t working you will stop and your doctor will speak to you about other treatments.

Follow up
You will have one follow up appointment at the national amyloidosis centre. This will be:

  • 6 months after you join the trial if you are having less than 6 cycles
  • 1 month after your final cycle if you have 6 cycles

Side effects

Common side effects can include:

  • fatigue
  • nausea and diarrhoea
  • increased risk of infection
  • shortness of breath or a cough
  • fever
  • headache and dizziness
  • chills
  • constipation
  • vomiting
  • swelling of arms and legs
  • pain or irritation at the injection site
  • allergic reaction
  • muscle or joint pain
  • difficulty sleeping
  • increase in blood pressure

Find more information about the side effects of thalidomide.

Get information about the side effects of dexamethasone.

Location

Birmingham
Leicester
London
Norwich
Plymouth
Southampton

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
Amgen 

Questions about cancer? Contact our information nurses

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.

Charlie took part in a trial to try new treatments

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“I think it’s really important that people keep signing up to these type of trials to push research forward.”

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