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New study/early-phase trial for High-Risk MDS and Acute Myeloid Leukaemia (AML)

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MDS Patient Support

To be conducted by Dr Mili Shah and Dr Victoria Potter of King's College Hospital, London, and Professor Robert Wynn of Royal Manchester Children’s Hospital.

 

This upcoming study will focus on the use of expanded cord blood stem cells, alongside granulocytes (a type of white blood cell) in both adults and children with high-risk blood cancers, in particular very high-risk Myelodysplastic Syndromes and Acute Myeloid Leukaemia.  These cancers are incredibly difficult to cure, and even with a stem cell transplant long-term survival rates can be low, therefore this is a priority area for researchers.

The aim of this particular study is to increase the successful outcome of stem cell transplants in high risk patients.  Early studies using expanded cord blood suggest it may improve outcomes, and with the addition of granulocytes (which have shown encouraging results so far in an ongoing clinical trial with children with very high-risk disease) it is possible that a combination of the two methods will provide the best chance of preventing the disease from recurring post-transplant.

Introductory online meeting about the study June 27th 6pm

If you'd like to learn more about this study the clinicians are hosting an introductory online meeting on Thursday 27th June at 6pm.  Please email milinaresh.shah@nhs.net or robert.wynn@nhs.uk and they will send you a short questionnaire to complete.

 

 

REPAIR-MDS – a ground breaking clinical trial for low/intermediate risk MDS

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October 2024 - Update

Repair-MDS is now closed to recruitment.  Once there are results and data available, which is expected to be in early-mid 2025, we will post this here.

On behalf of the trial managers, the clinical team, and ourselves on the PPIE (Patient and Public Involvement and Education) front, can we take this opportunity to say a big THANK YOU to those members who signed up and actively participated.

A new diagnosis of low/intermediate risk MDS can be shocking – hearing you have cancer, that it's life limiting and that there are very few treatments available, may well be distressing. Often, the only real option for patients is to return home and 'watch and wait' until the next appointment, usually three months away. The REPAIR-MDS trial offered a chance to get involved in one of two new experimental treatment options.
MDS Patient Support

What is the REPAIR-MDS trial?

REPAIR-MDS is a ground-breaking clinical trial that aims to improve the symptoms and overall quality of life for patients with low/intermediate risk Myelodysplastic Syndromes.  Patients were put randomly into one of two treatment groups below. Both groups used drugs which are already used safely for other diseases (Repurposed drugs). 

Treatment Group 1 VBaP - Sodium Valproate (V), Bezafibrate (Ba), Medroxyprogesterone (P) 

Treatment Group 2 : Danazol.  

The study is trying to find out if these ‘repurposed’ drugs can be used to treat MDS patients and to improve their blood counts, reduce their need for transfusions, improve their quality of life, and prolong their survival.

Where are the trial sites?

The trial is taking place throughout the UK, is coordinated by the University of Warwick’s Clinical Trials Unit and was open in around 30 hospitals.

Follow the link to the REPAIR-MDS website for a regularly updated list of REPAIR-MDS trial sites

What's the trial duration?

September 2020 - June 2025

 

To find out more

Get more information from the trial website below.

REPAIR-MDS Trial Website

The REPAIR-MDS trial website which has regularly updated information about who to contact, trial eligibility criteria, trial sites and more.

REPAIR-MDS Central Management Team

MDS Patient Support

Dr Stephen Jenkins is Lead (Clinical) Chief Investigator on the REPAIR-MDS trial and consultant haematologist at Russells Hall Hospital, Dudley.

MDS Patient Support

Dr Manoj Raghavan is Deputy (Clinical) Chief Investigator on the REPAIR-MDS trial. He is a consultant haematologist based at the Centre for Clinical Haematology.

MDS Patient Support

Professor Janet Dunn is Co-Chief Investigator & Professor of clinical trials & head of cancer trials at - Warwick Clinical Trials Unit.

MDS Patient Support

Sophie Gasson is a PPI Research Fellow at Warwick Clinical Trials Unit.

A brief introduction to REPAIR-MDS from Co-Chief Investigator Dr Manoj Raghavan

Watch the recorded webinar on the REPAIR-MDS Clinical Trial from February 13th 2023

The AMMO Trial

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What is the AMMO trial?

MDS/MPN Overlap Syndromes are rare bone marrow cancers. They have features of both MDS (Myelodysplastic Syndromes - where too few healthy blood cells are produced) and MPNs (Myeloproliferative Neoplasms - where too many blood cells are produced).  One type of MDS/MPN Overlap Syndrome is CMML (Chronic Myelomonocytic Leukaemia). For most patients there are no effective treatments available. Patients experience bone marrow failure, leading to fatigue, infections and bleeding, with a damaging overgrowth of white blood cells. 

The AMMO trial will test a new drug called ASTX727 to see how effective it is at treating and extending/improving the lives of people with these diseases. The trial is a comparison of response to treatment of patients with MDS/MPN Overlap Syndromes, such as CMML, taking ASTX727 versus best supportive care.

 

 

MDS Patient Support
Chief Investigator - Dr Daniel Wiseman

 

Who can participate?

For MDS UK members, this trial is for people aged over 18 with a diagnosis of CMML-2, who have not received prior treatment with hypomethylating agents, such as Azacitidine. A more detailed list of inclusions and exclusions can be found on the AMMO trial summary.

Sites of the trial
  1. The Christie, Manchester-Open
  2. Churchill hospital, Oxford-Open
  3. Belfast City Hospital, Belfast-Open
  4. St James’s, Leeds-Open
  5. QE, Birmingham-Open
  6. Aberdeen Royal Infirmary, Aberdeen-Open
  7. King’s College Hospital, London-Open
  8. City Hospital, Nottingham-Open
  9. Southampton General Hospital, Southampton-Open
  10. University College London Hospitals (UCLH), London-Open
  11. University Hospital of Wales, Cardiff-Site in set up
  12. Beatson, Glasgow- Site in set up
  13. Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield- Site in set up

For more information about the trial

Follow the links to the resources below that will be regularly updated by the trial coordinators with information, details of who to contact and more.

AMMO Trial Website

The AMMO trial website has a very clear, plain English explanation of the background & aims of the trial, eligibility criteria, contact details and more.

Read more at the AMMO trial website

AMMO Trial Summary pdf

This guide has detailed medical information about the trial design, objectives, contact details and the main inclusion and exclusion criteria.

Download 'AMMO Trial Summary'

REPAIR-MDS : Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes

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MDS Patient Support

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed on our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

REPAIR-MDS Repurposed Drugs to Improve Haematological Responses in Myelodysplastic Syndromes

  1. SUB-TYPE OF MDS: All subtypes of MDS
  2. SEVERITY OF MDS:  Lower-risk MDS
  3. NAME OF DRUG: 
  4. Aims and benefits: REPAIR-MDS seeks to repurpose existing drugs in order to dramatically improve the outlook, health and quality of life of people with MDS. The trial treatments aim to improve the production of healthy functioning blood and immune cells that will fight against infections and boost the immune system's action against the MDS clone.
    REPAIR-MDS design is a is a multicentre open label phase 2 randomised controlled trial which will compare VBaP (sodium valproate, bezafibrate, medroxyprogesterone) with danazol in patients who have received either Erythropoiesis Stimulating Agents (ESAs) and lost response, not responded to ESAs or are deemed unlikely to respond to ESAs.
  5. Primary outcome: Haematological improvement (HI) in each arm and in the trial overall, with 25% or more of the participants having HI in each arm and overall. [ Time Frame: 12 months ]
  6. Secondary outcome:  Reduced burden of red cell and/or platelet transfusion in each arm and in the trial overall, as per the IWG 2018 response criteria. [ Time Frame: 12 months ] Changes in transfusion requirements will be assessed in each participant by comparison with their individual 16-week lead-in baseline as determined by the IWG 2018 haematology response criteria in patients with MDS.
    • Duration of haematological response [ Time Frame: 12 months ]
    • Clinically meaningful haematological responses that persist for 16 weeks or longer.
    • Reported improved Health Related Quality of Life scores in each arm and in the trial overall. [ Time Frame: 12 months ]
    • The four Health Related Quality of Life questions measure
      1. 1 - self-perceived health (excellent, very good, good, fair, or poor)
      2. 2 - number of days out of the past 30 that physical health was not good
      3. 3 - number of days out of the past 30 that mental health was not good,
      4. 4 - number of days out of the past 30 that usual activities were limited by poor physical or mental health of life scores will be evaluated using established protocols.
    • Overall survival

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CCS-1477

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All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

CCS-1477

  1. SUB-TYPE OF MDS: All subtypes of MDS
  2. SEVERITY OF MDS: High Risk MDS and Acute myeloid leukaemia.
  3. NAME OF DRUG: CCS-1477 . CCS-1477 is a drug in oral form that blocks certain enzymes that are needed to encourage the expression of genes that drive cancer. It also causes the destruction of some proteins that are important in cancer cell development.
  4. Aims and benefits: This Phase I/IIa study investigates the safety, tolerability and activity of CCS-1477 in the body.
  5. Primary outcome: To identify adverse events related to the treatment.
  6. Secondary outcome: To determine the rate of response and duration of response to the treatment.

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PDR001 and/or MBG453 in Combination with Decitabine or Azacitidine

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MDS Patient Support

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

PDR001 and/or MBG453 in Combination with Decitabine or Azacitidine

  1. SUB-TYPE OF MDS: All patients with MDS (but see basic exclusion criteria below)
  2. SEVERITY OF MDS: Intermediate or High Risk MDS and Acute myeloid leukaemia.
  3. NAME OF DRUG: PDR001, MBG453. PDR001 is an antibody that makes cancer cells recognizable to the immune system thus resulting in reduction of tumour growth and size. MBG453 is an antibody that blocks a marker on leukaemia cells (TIM-3) thus making them more vulnerable to the effect of drugs used in leukaemia treatment.
  4. Aims and benefits: This Phase I study investigates different doses of the drugs PDR001 and/or MBG453 to determine the dose which is safe, well tolerated and effective. It investigates the drugs when used on their own as well as in combination with each other and in combination with Decitabine or Azacitidine.
  5. Primary outcome: To identify toxicities that occur due to dosage of the study drugs. To determine the safety of the study drugs when used on their own and in combination.

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Verona

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MDS Patient Support

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

Venetoclax

  1. SUB-TYPE OF MDS: All patients with MDS (but see basic exclusion criteria below)
  2. SEVERITY OF MDS: Revised International Prognostic Scoring System (IPSS-R) score > 3 (intermediate, high or very high).
  3. NAME OF DRUG: Venetoclax. Venetoclax is an inhibitor of BCL-2, thus promoting programmed death of tumour cells.
  4. Aims and benefits: This is a randomised double-blind, Phase 3 study aimed at determining the safety and effectiveness of Venetoclax when used in combination with Azacitidine in newly diagnosed patients with Higher risk Myelodysplastic syndrome.
  5. Primary outcome: To determine complete response and overall survival.
  6. Secondary outcome: To determine the rate of achieving transfusion independence.

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KRT-232

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MDS Patient Support

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

KRT-232

  1. SUB-TYPE OF MDS: Patients with MDS or CMML who have relapsed from or are refractory to previous therapy
  2. SEVERITY OF MDS: Intermediate, High or Very High Risk
  3. NAME OF DRUG: KRT-232
  4. Aims and benefits: This Phase Ib/2 study that investigates the safety and effectiveness of KRT-232 when taken alone and in combination with Low-dose Cytarabine or Decitabine in patients with acute myeloid leukaemia (AML) including AML secondary to myeloproliferative disorders.
  5. Primary outcome: To determine the recommended dose of KRT-232 as well as its safety and tolerability. KRT-232 targets MDM2 thus preventing it from inhibiting p53. P53 has a role in arresting the tumour cell cycle and in promoting programmed death of tumour cells.
  6. Secondary outcome: To determine response rates.

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EP0042 – New Clinical Study for MDS Patients

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All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

EP0042

  1. SUB-TYPE OF MDS: Patients with MDS or CMML who have relapsed from or are refractory to previous therapy
  2. SEVERITY OF MDS: Intermediate, High or Very High Risk
  3. NAME OF DRUG: EP0042
  4. Aims and benefits: This Phase I/IIa study investigates different doses of the drug EP0042 to determine the dose which is safe, well tolerated and effective.
  5. Primary outcome: To identify toxicities that occur due to dosage of the study drug.

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Sabatolimab (MBG453) – New Clinical Study for MDS Patients

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MDS Patient Support

Research FOR Patients
-For an informed and empowered opinion-

All the trials listed in our site have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust - for the continuous work in updating the listing.

Sabatolimab (MBG453)

  1. SUB-TYPE OF MDS: MDS patients and Chronic Myelomonocytic leukaemia -2 patients who require first-line treatment
  2. SEVERITY OF MDS: Intermediate, High or Very High Risk
  3. NAME OF DRUG: Sabatolimab (MBG453)
  4. Aims and benefits: MBG453 is an antibody that binds to a specific receptor on T-cells. In so doing, it abolishes the inhibitory effect that tumour cells have on T-cells and enhances the ability of the T-cells to destroy tumour cells. This Phase 3 trial seeks to determine the effectiveness of using MBG453 in combination with Azacitidine. It is a double-blind placebo controlled study. The main aim is to compare the overall survival with MBG453 plus Azacitidine versus placebo plus Azacitidine.
  5. Primary outcome: To determine overall survival.
  6. Secondary outcome: Assessment of the effects on quality of life, red cell transfusion-free intervals and time to disease progression.

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