PANTHER: Clinical Trial Open to Recruitment

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.


  1. SUB-TYPE OF MDS:Higher risk MDS, CMML, low blast count AML
  2. SEVERITY OF MDS: Intermediate, high risk or very high risk MDS as defined by the World Health Organization (WHO) criteria or Revised International Prognostic Scoring System (IPSS-R)
  3. NAME OF DRUG: Azacitidine, Pevonedistat
  4. Aims and benefits: Pevonedistat prevents the activity of a specific enzyme (Nedd8 activating enzyme) and thus may result in the inhibition of tumour cell growth and survival. This is a phase 3 study to determine if combining Pevonedistat with Azacitidine improves survival when compared with single agent Azacitidine.

  1. Primary outcome measures: 
    To determine event-free survival, an event being progression to AML or death.
  2. Secondary outcome measures:
    These include the assessment of complete remission rates, overall survival as well as survival rates at different time points.
  3. Basic inclusion criteria:
    • Higher risk MDS, CMML, low blast count AML.
    • Participants with AML (20%-30% blasts) must have a treatment-related mortality (TRM) score >=4 for intensive, induction chemotherapy as calculated using the simplified model described by Walter and coworkers.
    • - ECOG performance status 0-2.
  4. Basic exclusion criteria:
    • Previous treatment for higher risk MDS, CMML or low blast count AML. Treatment with hydroxycarbamide or Lenalidomide is permitted.
    • Eligible for intensive chemotherapy and/or allogeneic stem cell transplant.
    • CNS involvement by AML.
    • Active uncontrolled infection
    • Hepatic impairment
    • Cardiopulmonary disease
  5. Trial sites/locations and name of physician in charge of trial:
    • Royal Bournemouth Hospital, Bournemouth
    • Maidstone Hospital, Maidstone, Kent
    • St. Bartholomew’s Hospital, London
    • Singleton Hospital, Swansea

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

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