MDS Current Trials UK

We aim to list an easily understandable description of any MDS clinical trials currently opened to recruitment in the UK.

Patients will be able to read up on the details of these trials and use this information to discuss with their consultants or nurses.

Not all patients can be enrolled in drug trials - as not every patient will fit the necessary inclusion criteria, which need to be followed very strictly. However, if a particular clinical trial drug is considered an option by the haematologist, it is sometimes possible to obtain the drug outside the trial, on the basis of compassionate use.

All the trials listed in this page have been properly vetted for scientific accuracy. Many thanks to Dr Simone Green – Hull and East Yorkshire Hospitals NHS Trust 

FG-4592-082 (Roxadustat)

  1. SUB-TYPE OF MDS:Lower Risk MDS With Low Red Blood Cell Transfusion Burden
  2. SEVERITY OF MDS: IPSS-R classification very low, low or intermediate risk with <5% blasts
  3. NAME OF DRUG: Roxadustat
  4. Aims and benefits: To determine whether Roxadustat is safe and effective in treating anaemia in patients with Primary Lower Risk Myelodysplastic Syndrome and Low Red Blood Cell Transfusion Burden.
    Roxadustat is an oral preparation that stimulates erythropoiesis (production of red cells) by increasing the body’s production of the hormone erythropoietin and it regulates the way in which the body uses iron. This study is a Phase 3 Randomized Double-Blind Placebo-Controlled Study in which there is a treatment period of 52 weeks and a 4 week end of treatment assessment.

  1. Primary outcome measures: 
    To determine how effective Roxadustat is at correcting and maintaining the haemoglobin and reducing the number of red cell transfusions needed.
  2. Secondary outcome measures:
    To establish the effect of Roxadustat on clinical observations (including blood pressure, heart rate and ECG), how it affects red blood cell transfusion requirements and its effect on quality of life.
  3. Basic inclusion criteria:
    • Primary MDS classified as very low, low or intermediate risk with <5% blasts
    • Low red blood cell transfusion requirement (either 2-4 units over the 8 weeks prior to randomization or 1 unit in two consecutive periods of 8 weeks within the 16 weeks prior to randomization)
    • ECOG performance of 0 – 2 at screening
  4. Basic exclusion criteria:
    • Diagnosis of secondary MDS
    • Significant myelofibrosis
    • MDS associated with 5q(del) abnormality
    • Serum erythropoietin level > 400 mIU/mL
    • Clinically significant anaemia due to other causes (unrelated to MDS)
  5. Trial sites/locations and name of physician in charge of trial:
    • King’s College Hospital, London
    • Pilgrim Hospital Boston, Lincolnshire

Please read information and always discuss trial information with your own physician.

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This study is a Phase 3 Randomized Double-Blind Placebo-Controlled Study in which there is a treatment period of 52 weeks and a 4 week end of treatment assessment.<�/li><�/ol><�p style="box-sizing: inherit; margin: 0px 0px 1rem; padding: 0px; font-size: inherit; line-height: 1.6; text-rendering: optimizelegibility;"><�/p><�ol style="box-sizing: inherit; margin: 0px 0px 1rem 1.25rem; padding: 0px; line-height: 1.6; list-style-position: outside;"><�li style="box-sizing: inherit; margin: 0px; padding: 0px; font-size: inherit;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Primary outcome measures:�<�/strong><�br style="box-sizing: inherit;">To determine how effective Roxadustat is at correcting and maintaining the haemoglobin and reducing the number of red cell transfusions needed.<�/li><�li style="box-sizing: inherit; margin: 0px; padding: 0px; font-size: inherit;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Secondary outcome measures:<�/strong><�br style="box-sizing: inherit;">To establish the effect of Roxadustat on clinical observations (including blood pressure, heart rate and ECG), how it affects red blood cell transfusion requirements and its effect on quality of life.<�/li><�li style="box-sizing: inherit; margin: 0px; padding: 0px; font-size: inherit;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Basic inclusion criteria:<�/strong><�ul style="box-sizing: inherit; margin: 0px 0px 0px 1.25rem; padding: 0px; line-height: 1.6; list-style-position: outside; list-style-type: disc;"><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Primary MDS classified as very low, low or intermediate risk with &lt;5% blasts<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Low red blood cell transfusion requirement (either 2-4 units over the 8 weeks prior to randomization or 1 unit in two consecutive periods of 8 weeks within the 16 weeks prior to randomization)<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">ECOG performance of 0  2 at screening<�/li><�/ul><�/li><�li style="box-sizing: inherit; margin: 0px; padding: 0px; font-size: inherit;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Basic exclusion criteria:<�/strong><�ul style="box-sizing: inherit; margin: 0px 0px 0px 1.25rem; padding: 0px; line-height: 1.6; list-style-position: outside; list-style-type: disc;"><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Diagnosis of secondary MDS<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Significant myelofibrosis<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">MDS associated with 5q(del) abnormality<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Serum erythropoietin level &gt; 400 mIU/mL<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">Clinically significant anaemia due to other causes (unrelated to MDS)<�/li><�/ul><�/li><�li style="box-sizing: inherit; margin: 0px; padding: 0px; font-size: inherit;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Trial sites/locations and name of physician in charge of trial:<�/strong><�ul style="box-sizing: inherit; margin: 0px 0px 0px 1.25rem; padding: 0px; line-height: 1.6; list-style-position: outside; list-style-type: disc;"><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url(&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;) 0px 5px no-repeat; line-height: 22px; list-style: none;">King s College Hospital, London<�/li><�li style="box-sizing: inherit; margin: 0px 0px 10px; padding: 0px 0px 0px 20px; font-size: 1rem; background: url('/&quot;/wp-content/themes/oe-mds/assets/stylesheets/../images/icons/li.png&quot;') 0px 5px no-repeat; line-height: 22px; list-style: none;">Pilgrim Hospital Boston, Lincolnshire<�/li><�/ul><�/li><�/ol><�p style="box-sizing: inherit; margin: 0px 0px 1rem; padding: 0px; font-size: inherit; line-height: 1.6; text-rendering: optimizelegibility;"><�strong style="box-sizing: inherit; font-weight: bold; line-height: inherit;">Please read information and always discuss trial information with your own physician.<�/strong><�/p><�/div>

Paper of the Month

Venetoclax and hypomethylating agents (HMAs) induce high response rates in MDS, including patients after HMA therapy failure

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IPSS-R MDS Risk Assessment Calculator

Revised International Prognostic Scoring System (IPSS-R) for Myelodysplastic Syndromes Risk Assessment Calculator

calculator

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MDS Fact Sheet

MDS Factsheet for GPs General Practicioners

MDS Fact Sheet

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Varicella Vaccine in MDS

Urgent Clarification on varicella vaccine policy

Current advice from the UK MDS Forum is not to give the Live Varicella Vaccine to patients with MDS.

Please read below correspondence between Professor Salisbury and Dr George Follows, the latter on behalf of the CLL Forum, on the varicella vaccine policy.
This is an external link to the British Society for Haematology  Click here

NICE Guideline

Lenalidomide for treating myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality.

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BSH Guidelines

This is an external link to the home of the British Society for Haematology (BSH). 

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Links

External links to another websites

British Society of Haematology

UK MDS Patient Support Group

MDS Foundation

European Leukemia Net