Our Trials Acceleration Programme

Our clinical trials test new drugs or combinations of drugs in people living with blood cancer. Trials are really important, because they’re the only way we can prove whether the new treatments developed through research will improve care for people with blood cancer.

A large part of our research investment is devoted to funding clinical trials, including trials funded through our dedicated Trials Acceleration Programme (TAP) network.

What is the Trials Acceleration Programme (TAP)?

Bloodwise launched TAP in 2011, to speed up blood cancer clinical trials. The quicker it can be established whether a new drug is safe, and if it is more effective than standard treatments, the quicker the best new treatments can be routinely made available on the NHS.

TAP is organised from a central hub at the University of Birmingham run by experts skilled at navigating the logistical and administrative steps needed to set up and conduct clinical trials within strict regulatory guidelines. The hub provides support for the efficient running of trials in hospitals reducing the time it takes to open clinical trials in locations around the country.

We currently have a network of 13 TAP centres where we support a research nurse, or other staff that help with clinical trials. Seven further locations are affiliated to TAP. These centres benefit from full access to the network, and can contribute to recruitment to individual TAP trials. Because of the geographical spread of our centres, people with blood cancer around the whole of the UK are able to join a TAP trial, speeding up recruitment.

Our current TAP portfolio

TAP provides support for early phase clinical trials. Phase I trials test the safety of new drugs or treatments and establish the appropriate dosage, and phase II trials confirm the optimum dosage of new drugs and identify any side effects. Both types of trial can also tell doctors whether there is evidence that a new treatment is working or not, but larger confirmatory studies are usually needed before a treatment can be approved for routine care. By investing in this early stage of the clinical trials process we are able to accelerate the testing of new treatments, either ruling out options that will not work, or moving promising approaches on quickly so that they can be tested in late phase trials that will confirm whether they should be used in routine care.

To date, we have run 19 trials through TAP that cover all types of blood cancer. In this section you can read more about each of the trials that we fund.

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LYMPHOMA

Hodgkin lymphoma

BREVITY trial: A phase II study of brentuximab vedotin (SGN-35) using a response adapted design in patients with Hodgkin lymphoma unsuitable for chemotherapy due to age, frailty or co-morbidity

Blood cancer type: Lymphoma/Hodgkin lymphoma

Chief Investigator: Professor John Radford, University of Manchester and the Christie NHS Foundation Trust.

Award start date: 01 April 2012

Recruitment start: 01 February 2014

Award duration: 5 years (60 months)

SUMMARY: People with Hodgkin lymphoma are usually treated with chemotherapy, but some are too frail to have this type of treatment, or have another medical condition which means they cannot have chemotherapy.

Brentuximab is a type of biological therapy called a monoclonal antibody. It can seek out cancer cells by looking for a molecule called CD30 that is found on the surface of Hodgkin lymphoma cells. Brentuximab vedotin has an additional link to a chemotherapy drug called monomethyl auristatin E. This means that the brentuximab monoclonal antibody can deliver the chemotherapy accurately to the Hodgkin lymphoma cell, causing the cell to die.

Brentuximab vedotin has been shown to be effective for people whose Hodgkin lymphoma has come back after chemotherapy treatment, and researchers want to see if this drug may also help people who can’t have standard chemotherapy. Because brentuximab vedotin is a targeted therapy, it may also have less side effects than chemotherapy.

The aims of this trial were to:

  • Find out if brentuximab vedotin could work as a first treatment for Hodgkin lymphoma
  • Find out more about the side effects

This trial is closed for recruitment, and we hope to update you with the results soon.

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Diffuse large B cell lymphoma (DLBCL)

TIER trial: A phase I/II study of thiotepa, ifosphamide, etoposide and rituximab for the treatment of relapsed and refractory primary central nervous system lymphoma

Blood cancer type: Lymphoma/non-Hodgkin lymphoma/primary central nervous system lymphoma (PCNSL)

Chief Investigator: Dr Christopher Fox, Nottingham University Hospitals NHS Trust

Award start date: 01 December 2013

Recruitment start: 03 February 2015

Award duration: 5 years (60 months)

SUMMARY: Primary central nervous system lymphoma (PCNSL) is a rare type of high-grade non-Hodgkin lymphoma that only affects the central nervous system (CNS), which means the brain, eye and spinal cord.

People with PCNSL are usually treated with chemotherapy. But sometimes the lymphoma does not respond to chemotherapy, or it comes back after treatment. In this situation there is no standard treatment, so we need to find new ways to treat this group of people.

Researchers want to try a new combination of cancer drugs comprising of chemotherapies and a biologic drug. The following drugs will be tested: thiotepa, ifosfamide, etoposide and rituximab.

Thiotepa, ifofamide and etoposide are chemotherapies. Thiotepa and ifosfamide work by interfering with the cancer cell’s DNA, preventing the cells from growing and dividing. Etoposide works by blocking an enzyme called topoisomerase 2, which is necessary for cancer cells to divide.

Rituximab is a type of biological therapy called a monoclonal antibody. It works by targeting a protein called CD20, which is found on the surface of healthy white blood cells and abnormal white blood cells that occur in leukaemia and lymphoma. The monoclonal antibody binds to all the cells that have CD20 on their surface, which triggers the immune system to attack the marked cells and kill them. Rituximab causes both healthy and cancerous white blood cells to be destroyed, but the body can replenish the healthy white blood cells once the treatment is over.

The aims of the study are to:

  • Discover the best dose of thiotepa
  • See how well the combination of thiotepa, ifosfamide, etoposide and rituximab works for PCNSL

You may be eligible to join this trial if:

  • You have DLBCL that started in your brain and spinal cord, which has come back after previous treatment or has not responded to treatment
  • You are at least 16 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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Diffuse large B cell lymphoma (DLBCL)

TORCH trial: A phase II study to determine the safety and efficacy of the dual mTORC inhibitor AZD2014 and to investigate additional toxicities in combination with rituximab in relapsed refractory DLBCL

Blood cancer type: Lymphoma/non-Hodgkin lymphoma/diffuse large B cell lymphoma (DLBCL)

Chief Investigator: Dr Graham Collins, Oxford University Hospitals NHS Trust

Award start date: 01 August 2015

Recruitment start: 19 October 2015

Award duration: 5 years (60 months)

SUMMARY: People with diffuse B cell lymphoma (DLBCL) – a type of non-Hodgkin lymphoma – are usually treated with chemotherapy and a biological therapy called rituximab. Although this approach works for many people, sometimes the treatment isn’t effective, or the lymphoma comes back.

This trial is looking at a new drug called vistusertib for people with DLBCL that has continued to grow during standard treatment, or has come back afterwards. Vistusertib is a type of biological therapy and prevents cancer cells from growing by targeting the mTOR pathway, blocking the signals that cancer cells use to divide and grow.

Rituximab is a type of biological therapy called a monoclonal antibody. It works by targeting a protein called CD20, which is found on the surface of healthy white blood cells and abnormal white blood cells that occur in leukaemia and lymphoma. The monoclonal antibody binds to all the cells that have CD20 on their surface, which triggers the immune system to attack the marked cells and kill them. Rituximab causes both healthy and cancerous white blood cells to be destroyed, but the body can replenish the healthy white blood cells once the treatment is over.

Researchers want to know if vistersertib is effective and safe for people with relapsed DLBCL. They also want to know if giving rituximab alongside vistusertib provides any additional benefit, so will be testing this drug combination in a small number of people.

The aims of this trial are to:

  • Find out If vistusertib helps people with DLBCL
  • Find out if combining vistusertib with rituximab is a useful and safe treatment
  • Learn more about the side effects of the combination therapy

This trial is closed for recruitment, and we hope to update you with the results soon.

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Peripheral T-cell lymphoma (PTCL)

RomiCar trial: A phase I/II study to determine the maximum tolerated dose and overall response rate of the combination of romidepsin and carfilzomib in relapsed or refractory peripheral T-cell lymphoma

Blood cancer type: Lymphoma/non-Hodgkin lymphoma/peripheral T-cell lymphoma (PTCL)

Chief Investigator: Dr Graham Collins, Oxford University Hospitals NHS Trust

Award start date: 01 August 2015

Recruitment start: 31 July 2015

Award duration: 4.5 years (54 months)

SUMMARY: Peripheral T-cell lymphoma (PTCL) is a particularly aggressive and difficult to treat non-Hodgkin lymphoma, which has relatively poor outcomes.

People with PTCL are usually given chemotherapy, but sometimes it continues to grow, or comes back after the treatment has finished.

This trial is looking at two drugs called romidepsin and carfilzomib.

Romidepsin inhibits proteins (enzymes) called histone deacetylases - cells need these enzymes to grow and divide. Drugs that block these enzymes are called histone deacetylase inhibitors, or HDAC inhibitors. Carfilzomib is a type of biological therapy called a proteasome inhibitor that also blocks cancer growth by stoppings signals that cancer cells use to divide and grow.

Researchers want to know if combining romidepsin and carfilzomib could help people with PTCL that has come back, or standard treatment has stopped working for them.

The aims of the trial are to:

  • Find the best dose of romidepsin and carfilzomib
  • Find out  if the combination of romidepsin and carfilzomib can help people with PTCL
  • Learn more about the side effects

You may be eligible to join this trial if:

  • You have PTCL that has come back or treatment has stopped working
  • You are at least 16 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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Peripheral T-cell lymphoma (PTCL)

AVAIL-T trial: A phase IIa trial of avelumab, an anti-PDL1 antibody, in relapsed and refractory peripheral T-cell lymphoma

Blood cancer type: Peripheral T-cell lymphoma (PTCL)/lymphoma/non-Hodgkin lymphoma

Chief Investigator: Professor Simon Wagner, University of Leicester

Award start date: 01 January 2017

Recruitment start: TBC

Award duration: 3 years (36 months)

SUMMARY: Peripheral T-cell lymphoma (PTCL) is an aggressive and difficult to treat non-Hodgkin’s lymphoma that has relatively poor outcomes. A proportion of people with PTCL fail to response to first line treatment, or relapse after receiving treatment. Currently there are no licensed drugs in Europe for this group of people.

PTCL cells exist in an environment containing supporting cells of various subtypes. It is believed that targeting both the lymphoma cells directly and the supporting cells will be effective routes to treatment. Avelumab is an antibody directed against a cell surface molecule called PD-L1. Avelumab removes cells which express the cell surface protein, PD-L1, which can be found either on lymphoma cells, or on the supporting cells. It is also likely to have other mechanisms of action. Avelumab has been used to treat other cancer types with some success, so researchers want to find out if this drug will be an effective addition to treatments for PTCL.

The aims of this trial are to:

  •          Determine the overall response to avelumab
  •          Determine whether patient response is related to levels and pattern of PD-L1 expression

Eligibility criteria to be confirmed.

This trial is in set up, and we hope to bring you more information soon.

 

LEUKAEMIA

Chronic myeloid leukaemia (CML)

MATCHPOINT trial: A phase I study of ponatinib in combination with conventional chemotherapy (FLAG-IDA) followed by stem cell transplantation for the treatment of chronic myeloid leukaemia in blastic transformation

Blood cancer type: Leukaemia/chronic myeloid leukaemia (CML) in the blast phase

Chief Investigator: Professor Mhairi Copland, University of Glasgow

Award start date: 01 January 2013

Recruitment start: 02 December 2014

Award duration: 3 years (122 months)

SUMMARY: Chronic myeloid leukaemia (CML) cells produce a protein called BCR-ABL that isn't found in healthy cells. BCR-ABL is a tyrosine kinase, which causes CML cells to grow and reproduce out of control. People with CML are often treated with tyrosine kinase inhibitors (TKIs), such as imatinib or nilotinib. TKIs block the action of the BCR-ABL tyrosine kinase, so signals that tell the CML cells to grow and divide are disrupted.

TKIs have improved the outcome for people with CML dramatically. However this improvement remains limited to people who receive TKI treatment whilst in the chronic phase of their disease. Unfortunately, the majority of people whose disease has progressed to blast phase have already received all the available drugs in succession during the chronic phase, so there is no TKI available when they progress. If this happens, people are usually given intensive treatment comprising high dose of chemotherapy alongside a TKI, and then a stem cell transplant.

This trial is looking at a conventional chemotherapy regimen called FLAG-IDA alongside a new TKI drug called ponatinib for people who are in the blast phase of their CML. Ponatinib works in a similar way to imatinib and nilotinib, in that it specifically targets BCR-ABL that is only found in CML cells. However, ponatinib was also designed to target mutated forms of the BCR-ABL that arise in people who become resistant to their TKI treatment, as well as other kinases implicated in blood cancer and solid tumours.

If found to be safe, researchers hope that this combination of drugs can be used in future trials to see whether we can improve the outlook for people with CML who are in blast phase.

The aims of the trial are to:

  • Find the best dose of ponatinib  for people having intensive treatment for CML that is in blast phase
  • See how well ponatinib works for this group of people
  • Learn more about the side effects

You may be eligible to join this trial if:

  • You have chronic myeloid leukaemia (CML) that is in the blast phase
  • You are at least 16 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

Chronic lymphocytic leukaemia (CLL)

CyCLLe trial: An investigation of the effect of cyclosporine-A in chronic lymphocytic leukaemia: Development of a novel in-vivo strategy for dissecting the mechanism of drug action

Blood cancer type: Leukaemia/chronic lymphocytic leukaemia (CLL)

Chief Investigator: Professor Stephen Devereux, Kings College London

Award start date: 01 April 2013

Recruitment start: 29 April 2013

Award duration: 2 years (24 months)

SUMMARY: At the moment bone marrow transplantation is the only cure for CLL, but some people may not be able to undergo this treatment because they are too frail. Chemotherapy can be effective, but can have serious side effects especially in older people. Researchers are looking for treatments that will slow down the disease in its early stages and prevent progression and the need for such strong drugs.

The CyCLLe trial is looking at a drug called cyclosporin A. Cyclosporin A has been used for many years to reduce the activity of the immune system in people who have certain autoimmune diseases or who have received organ transplants. Researchers think that healthy cells in our immune system may help CLL to grow and so cyclosporin A could slow the rate at which leukaemia cells grow. If this is true, some people might be able to avoid harsher chemotherapy treatments.

The aims of the trial were to:

  • See if cyclosporin A changes the rate at which leukaemia cells grow
  • Find out if cyclosporin A helps people with early CLL
  • Learn more about the side effects

This trial is closed for recruitment, and we hope to update you with the results soon.

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IciCLLe trial: A phase II assessment of the mechanism of action of ibrutinib (PCI-32765) in B-cell receptor pathway inhibition in CLL

Blood cancer type: Leukaemia/chronic lymphocytic leukaemia (CLL)

Chief Investigator: Professor Peter Hillmen, St James's University Hospital

Award start date: 01 May 2014

Recruitment start: 12 June 2014

Award duration: 5 years (60 months) – funding includes the IciCLLe extension study)

SUMMARY: At the moment bone marrow transplantation is the only cure for CLL, but some people may not be able to undergo this treatment because they are too frail. Chemotherapy can be effective, but can have serious side effects especially in older people.

Research has shown that ibrutinib (also known as PCI-32765) can help people with CLL. Ibrutinib is a type of biological therapy called a Bruton’s Tyrosine Kinase (BTK) inhibitor that stops signals that cancer cells use to divide and grow. But researchers still need to know more about how it works, so doctors can understand how to combine ibrutinib with other drugs to make more effective treatments.

The main aims of this study were to:

  • Find out more about how ibrutinib works
  • See how well ibrutinib works for people with CLL who haven’t had treatment yet
  • Assess how well ibrutinib works for people whose CLL has continued to get worse during treatment, or came back after treatment

This trial is closed for recruitment, and we hope to update you with the results soon.

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IciCLLe extension: A phase II assessment of the mechanism of action of PCI-32765 in B-cell receptor pathway inhibition in CLL

Blood cancer type: Leukaemia/chronic lymphocytic leukaemia (CLL)

Chief Investigator: Professor Peter Hillmen, St James's University Hospital

Award start date: 01 May 2014

Recruitment start: 04 December 2015

Award duration: 5 years (60 months) – funding includes the IciCLLe study)

SUMMARY: At the moment bone marrow transplantation is the only cure for CLL, but some people may not be able to undergo this treatment because they are too frail. Chemotherapy can be effective, but can have serious side effects especially in older people.

We know from research that ibrutinib (also known as PCI-32765) can help people with CLL. Ibrutinib is a type of biological therapy called a Bruton’s Tyrosine Kinase (BTK) inhibitor that stops signals that cancer cells use to divide and grow. But we still need to know more about how it works, so doctors can understand how to combine ibrutinib with other drugs to make more effective treatments. One drug that could be combined with ibrutinib is obinutuzumab. Obinutuzumab is a monoclonal antibody and works in a similar way to rituximab, in that they both target a protein called CD20 on leukaemia cells. When obinutuzumab binds to cells that have CD20 on their surface, it triggers the immune system to attack the marked cells and kill them.

People who are already taking ibrutinib as part of the IciCLLe trial were asked if they wanted to join the IciCLLe extension trial. This trial is also open to people who have CLL that has continued to get worse during treatment, or have relapsed after treatment.

Everyone who takes part will have ibrutinib and obinutuzumab.

The aims of this study are to:

  • Find out how well ibrutinib and obinutuzumab works for people with CLL
  • See what the side effects are

You may be eligible to join this trial if:

  • You have CLL that needs treatment
  • You have CLL that has relapsed, or continued to get worse during treatment
  • You are at least 18 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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CALiBRe trial: An assessment of the mechanism of action of CAL-101 in B-cell receptor pathway inhibition in CLL

Blood cancer type: Leukaemia/chronic lymphocytic leukaemia (CLL)

Chief Investigator: Professor Peter Hillmen, St James's University Hospital

Award start date: 01 February 2015

Recruitment start: 13 July 2015

Award duration: 5 years (60 months)

SUMMARY: At the moment bone marrow transplantation is the only cure for CLL, but some people may not be able to undergo this treatment because they are too frail. Chemotherapy can be effective, but can have serious side effects especially in older people.

Researchers are looking at how a biological drug called idelalisib (also known as CAL-101) works for people with chronic lymphocytic leukaemia (CLL). Idelalisib is type of drug called a PI3K inhibitor. By blocking the PI3K protein, idelalisib can stop cancer cells growing. There have been other trials of idelalisib for people with CLL but this trial aims to find out exactly how it works so doctors can develop rational therapy combinations.

The aims of this trial are to:

  • Find out how idelalisib works in the body to reduce the number of CLL cells
  • Find out how people with CLL respond to idelalisib
  • Identify people who would respond well to idelalisib

You may be eligible to join this trial if:

  • You have CLL and need treatment
  • You have CLL that has come back after treatment (relapsed) or continued to get worse during treatment (refractory)
  • You are at least 18 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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CLARITY trial: A phase II assessment of the mechanism of action of venetoclax (ABT-199) in combination with ibrutinib (PCI-32765) in relapsed/refractory chronic lymphocytic leukaemia

Blood cancer type: Leukaemia/chronic lymphocytic leukaemia (CLL)

Chief Investigator: Professor Peter Hillmen, St James's University Hospital

Award start date: 01 January 2016

Recruitment start: 20 May 2016

Award duration: 5 years (60 months)

SUMMARY: This study is for people with chronic lymphocytic leukaemia (CLL) that has come back or treatment has stopped working.

We know from research that ibrutinib can help people with CLL. Ibrutinib is a type of biological therapy called a Bruton’s Tyrosine Kinase (BTK) inhibitor that stops signals that cancer cells use to divide and grow. In this trial, researchers want to find out how well ibrutinib works alongside another drug called venetoclax. Venetoclax is a type of drug called a Bcl-2 inhibitor. Bcl-2 is a protein that controls a pathway in cells that causes cells to die, and high levels of this protein helps cancer cells survive indefinitely. Venetoclax works by blocking the effects of Bcl-2 causing cancer cells to die.

The aims of the trial are to:

  • Find out how well ibrutinib and venetoclax work together
  • Find out how safe the combination treatment is
  • Learn more about the side effects

You may be eligible to join this trial if:

  • Your CLL didn’t go away completely or only went away a little bit (you didn’t have a complete or partial response), or your CLL came back within 6 months after having a drug such as fludarabine alone or with chemotherapy
  • You are at least 18 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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Acute myeloid leukaemia (AML)

ROMAZA trial: Phase I trial of romidepsin plus azacitidine in patients with newly diagnosed, relapsed or refractory acute myeloid leukaemia ineligible for conventional chemotherapy

Blood cancer type: Leukaemia/acute myeloid leukaemia (AML)

Chief Investigator: Professor Charles Craddock, University Hospital Birmingham

Award start date: 01 January 2013

Recruitment start: 30 September 2013

Award duration: 5 years (60 months)

SUMMARY: People with acute myeloid leukaemia (AML) are usually given chemotherapy, but not everyone can have this type of treatment. Researchers think that combining azacitidine with romidepsin may help people with AML who can’t have standard chemotherapy treatment.

Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

Romidepsin is a new HDAC inhibitor – a drug that blocks enzymes called histone deacetylases, which cells need to grow and divide. It has provided clinical benefit in people with lymphoma, but to date has not been studied in combination with azacitidine in people with AML.

The aims of this trial were:

  • To find out what is the highest dose of romidepsin to give with azacitidine
  • See how safe it is to give romidepsin with azacitidine
  • Assess how well this combination of drugs work for people with AML

This trial is closed for recruitment, and we hope to update you with the results soon.

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VIOLA trial: A phase I trial of combined azacitidine and lenalidomide salvage therapy in patients with acute myeloid leukaemia who relapse after allogeneic stem cell transplantation

Blood cancer type: Leukaemia/acute myeloid leukaemia (AML) and myelodysplastic syndrome (MDS) 

Chief Investigator: Professor Charles Craddock, University Hospital Birmingham

Award start date: 01 September 2013

Recruitment start: 05 February 2014

Award duration: 4 years (52 months)

SUMMARY: People with acute myeloid leukaemia (AML) or myelodysplastic syndrome (MDS) are usually treated with high doses of chemotherapy, followed by a stem cell transplant. But AML and MDS can come back after a transplant, and when it does it becomes much more difficult to treat.

Two new drugs called azacitidine and lenalidomide may help people with AML and MDS who have relapsed after a stem cell transplant.

Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

Lenalidomide is a complicated drug with several mechanisms of action affecting lots of cell processes, including modifying the way the immune system works.

A number of small studies have demonstrated that both azacitidine and lenalidomide when administered alone can be effective in treating people with AML who relapse after a stem cell transplant. But combined treatment with azacitidine and lenalidomide has never been tested in this group of people.

The aims of the trial are to:

  • Find the highest dose of lenalidomide that people can safely have with azacitidine
  • Learn more about the side effects of this drug combination
  • See if the treatment helps people with AML or MDS that has come back after a stem cell transplant

This trial is closed for recruitment, and we hope to update you with the results soon.

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RAvVA trial: A randomised phase II trial of 5-azacitidine versus 5-azacitidine in combination with vorinostat in patients with relapsed acute myeloid leukaemia ineligible for salvage chemotherapy

Blood cancer type: Leukaemia/acute myeloid leukaemia (AML) and myelodysplasia (MDS) 

Chief Investigator: Professor Charles Craddock, University Hospital Birmingham

Award start date: 01 August 2011

Recruitment start: 25 September 2012

Award duration: 5.5 years (68 months)

SUMMARY: Doctors usually treat AML or high risk MDS with chemotherapy. But this treatment can be intensive, and some people cannot tolerate it because they are too frail. This group of people may have less intensive chemotherapy, but there is no standard treatment in this situation.

In this trial, researchers are looking at two drugs called azacitidine and vorinostat.

Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

Vorinostat inhibits proteins (enzymes) called histone deacetylases - cells need these enzymes to grow and divide. Drugs that block these enzymes are called histone deacetylase inhibitors, or HDAC inhibitors.

The aim of the trial is to:

  • See if combining azacitidine and vorinostat is better than azacitidine alone

This trial is closed for recruitment, and we hope to update you with the results soon.

MPNs

Myeloproliferative neoplasms (MPNs)

TAMARIN trial: A phase II trial to assess the effects of tamoxifen on the mutant allele burden and disease course in patients with myeloproliferative neoplasms

Blood cancer type: Myeloproliferative neoplasms (MPNs)

Chief Investigator: Professor Claire Harrison, Guys and St Thomas’ NHS Foundation Trust

Award start date: 01 December 2015

Recruitment start: 26 August 2016

Award duration: 3 years (36 months)

SUMMARY: This study is for people with a type of blood disorder called myeloproliferative neoplasms (MPN) that causes the bone marrow to produce too many blood cells. This includes: polycythaemia vera (PV), essential thrombocythaemia (ET) and myelofibrosis (MF).

People with PV produce too many red blood cells, causing the blood to become thicker than normal. ET occurs when there are too many platelets in the blood, which can cause the blood to clot. People with MF have an overactive bone marrow, which develops scar tissue (known as fibrosis). The scar tissue builds up inside the bone marrow preventing the normal development of blood cells and can cause complications including anaemia.

Many people with MPN have changes to the JAK2 and CALR genes found in blood cells. There are already some treatments that target cells that have these gene changes, including ruxolitinib. Ruxolitinib inhibits the abnormal JAK signalling associated with changes to the JAK2 gene, and stops cancer cells from growing and dividing. Ruxolitinib and other treatments, such as interferon and hydroxycarbamide, have helped people with MPN. But some are unable to tolerate the side effects of these treatments, so researchers are looking for new ways to treat MPN.

Tamoxifen is a drug currently used to treat breast cancer and laboratory research indicates it could be beneficial for people with MPN who have the JAK2 and the CALR gene changes. 

In this study, people will have tamoxifen alongside the usual treatment for their MPN.

The aim of this trial is to:

  • See if adding tamoxifen helps to reduce the number of cells carrying the JAK2 and CALR genetic changes

You may be eligible to join this trial if:

  • You have had PV, ET, or MF for six months or longer
  • You have the JAK2 or CALR gene change
  • You are aged 60 or older
    • Men with an MPN aged between 50 to 59 may be able to take part - the study team would confirm this

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

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MAJIC trial: A phase II study of ruxolitinib (INCB424) in patients with high risk polycythaemia vera or essential thrombocythaemia resistant to or intolerant of standard therapy

Blood cancer type: Myeloproliferative neoplasms (MPNs)

Chief Investigator: Professor Claire Harrison, Guys and St Thomas’ NHS Foundation Trust

Award start date: 01 August 2011

Recruitment start: 01 August 2012

Award duration: 7 years (84 months)

SUMMARY: This study is for people with a type of blood disorder called myeloproliferative neoplasms (MPN) that causes the bone marrow to produce too many blood cells. This includes: polycythaemia vera (PV), essential thrombocythaemia (ET) and myelofibrosis (MF).

People with PV produce too many red blood cells, causing the blood to become thicker than normal. ET occurs when there are too many platelets in the blood, which can cause the blood to clot. People with MF have an overactive bone marrow, which develops scar tissue (known as fibrosis). The scar tissue builds up inside the bone marrow and blood cells can’t develop properly causing other complications including anaemia.

Doctors usually treat MPNs with a chemotherapy called hydroxycarbamide, which works by stopping the cancer cells making and repairing DNA. But some people can’t take hydroxycarbamide and sometimes the MPN stops responding to it.

Researchers think that a drug called ruxolitinib may help people with high risk PV and high risk ET. Ruxolitinib is a JAK inhibitor, and works by blocking the abnormal JAK signalling associated with changes to the JAK2 gene found in blood cells in people with MPN. By inhibiting this pathway, ruxolitinib can stop cancer cells from growing and dividing.

The aims of this trial were to:

  • See how well ruxolitinib works for people with PV and ET who can’t have hydroxycarbamide
  • See how safe it is in people with PV and ET

This trial is closed for recruitment, and we hope to update you with the results soon.

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PHAZAR TRIAL: A phase Ib study to assess the safety and tolerability of oral ruxolitinib in combination with 5 azacitidine in patients with accelerated phase MPNs, MDS or AML after MPNs

Blood cancer type: MPNs, MDS or AML after MPNs

Chief Investigator: Dr Mark Drummond, University of Glasgow

Award start date: 01 April 2014

Recruitment start: 05 January 2016

Award duration: 4 years (48 months)

SUMMARY: Myeloproliferative neoplasms (MPN) are a rare group of disorders of the bone marrow that cause an increase in the number of blood cells. They are also called myeloproliferative disorders. MPNs includes: polycythaemia vera (PV), essential thrombocythaemia (ET) and myelofibrosis (MF).

People with PV produce too many red blood cells, causing the blood to become thicker than normal. ET occurs when there are too many platelets in the blood, which can cause the blood to clot. People with MF have an overactive bone marrow, which develops scar tissue (known as fibrosis). The scar tissue builds up inside the bone marrow and blood cells can’t develop properly causing other complications including anaemia.

Researchers want to see if two drugs called azacitidine and ruxolitinib can help people with MPN. Both drugs are already used on their own to treat this group of people, but the trial wants to test how well they work when given together.

Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

Ruxolitinib is a JAK inhibitor, and works by blocking the abnormal JAK signalling associated with changes to the JAK2 gene found in blood cells in people with MPN. By inhibiting this pathway, ruxolitinib can stop cancer cells from growing and dividing.

The aims of the trial are to:

  • Find a safe dose of azactidine and ruxolitinib to have together
  • See how well treatment works
  • Find out more about the side effects 

You may be eligible to join this trial if:

  • You have a myeloproliferative neoplasm that includes essential thrombocythaemia, polycythaemia vera or myelofibrosis and you have a lot of immature cells called blasts in your blood and bone marrow
  • You need treatment for your blood disorder
  • You are at least 16 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

Myelodysplastic syndrome

ELASTIC trial: A phase Ib study of eltrombopag and azacitidine in patients with high risk myelodysplastic syndromes and related disorders

Blood cancer type: High risk myelodysplastic syndromes (MDS) 

Chief Investigator: Dr Alexander Sternberg, University of Oxford

Award start date: 01 October 2012

Recruitment start: 15 October 2014

Award duration: 5 years (59 months)

SUMMARY:

MDS is a serious disorder of blood cell production that causes fatigue, infection and bleeding. Certain types of MDS ('high risk' MDS) are prone to change their character and become cancerous - resulting in leukaemia.

Azacitidine is a type of chemotherapy drug that doctors already use to treat people with AML and MDS. Its exact mechanism of action is unknown, and it has more than one effect on cells. In AML and MDS, important genes that regulate cell growth and division are turned off by methyl groups becoming attached to the cells’ DNA, allowing cells to grow out of control. Azacitidine can act as a demethylating agent, removing the methyl groups and restoring normal gene function.

A recent trial has shown that a chemotherapy called azacitidine improves survival in people with high risk MDS. But many people in this trial had to have their azacitidine reduced or stopped because of low platelet counts and bleeding.

Researchers want to see a drug called eltrombopag helps to keep the platelet count up during azacitidine treatment. Although eltrombopag has been used for other disorders, doctors do not know what the correct dose eltrombopag should be for people with high risk MDS patients being treated with azacitidine.

The aims of the trial are to:

  • Find out the highest, safest dose of eltrombopag to give with azacitidine
  • Find out what the side effects are of having these two drugs together
  • See if the treatment helps people with MDS that has come back after a stem cell transplant

You may be eligible to join this trial if:

  • You have a low number of platelets
  • You are at least 16 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>

MYELOMA

BUBBLE trial: A phase Ib study of BKM120 with bortezomib in defined genetic subgroups of patients with relapsed or refractory multiple myeloma

Blood cancer type: Myeloma

Chief Investigator: Professor Kwee Yong, University College London

Award start date: 17 March 2014

Recruitment start: 23 June 2016

Award duration: 6 years (72 months)

SUMMARY: This study is looking at treatment for people with certain gene changes (mutations) in their myeloma cells. It is for people whose myeloma has come back or treatment has stopped working. 

People with gene mutations in their myeloma cells may cause them to not respond to usual chemotherapy as well as other treatments.

Bortezomib is one of the usual therapies for myeloma, and researchers think that adding another drug called buparlisib (also known as BKM120) could help people with gene changes.

Bortezomib is a proteasome inhibitor. It works by blocking the action of proteasomes which help the cell break down proteins that are not needed. This causes proteins to build up inside the cell causing the cell to stop growing and die.

Buparlisib is a PI3K inhibitor, and inhibits the PI3K cell signalling pathway which is often overactivated in cancer allowing cancer cells to grow rapidly.

The aims of the study are to:

  • Find the highest safe dose of buparlisib combined with bortezomib
  • Test the best combination dose in people with the gene changes
  • Learn more about the side effects

You may be eligible to join this trial if:

  • You have myeloma that has come back or stopped responding to treatment, and you have had at least one but no more than four treatments
  • You are at least 18 years old

Other eligibility criteria apply, so visit xxxx for more information <TBC where to sign post to>

If you are interested in taking part in this trial, and want to know more information click here <include link to trial on clinicaltrials.gov pages>

<Include link to Bloodwise patient information once link is available>