P3001:pevonedistat+azacitidine for HR MDS/CMML/low blast AML treatment

• Research type

  Research Study

• Full title

  A Phase 3, Randomized, Controlled, Open-Label, Clinical Study of Pevonedistat Plus Azacitidine Versus Single-Agent Azacitidine as First-Line Treatment for Patients With Higher-Risk Myelodysplastic Syndromes, Chronic Myelomonocytic Leukemia, or Low-Blast Acute Myelogenous Leukemia

• IRAS ID

  236930

• Contact name

  Sally Killick

• Contact email

  sally.killick@rbch.nhs.uk

• Sponsor organisation

  Millennium Pharmaceuticals, Inc. (Takeda)

• Eudract number

  2017-000318-40

• Duration of Study in the UK

  5 years, 3 months, 2 days

• Research summary

  Summary of Research
  Leukemia is a cancer of the early blood-forming cells. There are many different types of leukemia. Myelodysplastic syndromes are closely related diseases of blood-forming cells that can precede leukemia. This study will include patients with higher-risk Myelodysplastic Syndromes (MDS), Chronic Myelomonocytic Leukemia (CMML), or Low-Blast Acute Myelogenous Leukemia (AML).
  The purpose of this study is to assess whether taking the study drug pevonedistat in combination with azacitidine increases the time before disease worsening when compared to azacitidine alone.
  At enrolment, patients will be randomised into two treatment groups in a 1:1 ratio to receive either:
  • single-agent azacitidine
  • combination of pevonedistat and azacitidine

  Patients will be treated in 28 day cycles. Patients in the combination group will receive pevonedistat on Days 1, 3 and 5. All patients will be administered azacitidine on Days 1-5, 8 and 9. During the first cycle, 7 visits to the hospital are required. During subsequent cycles, patients attend hospital for 4 visits per cycle.

  Patients may receive study treatment until they experience unacceptable toxicity, relapse, or worsening of disease. Patients will attend an end of treatment visit 30 days after the last dose of study drug or before the start of subsequent anti-cancer treatment. Depending on the reason for study drug discontinuation, patients may enter event-free survival or response follow-up with monthly visits. All patients ultimately enter the overall survival follow-up with telephone contacts every 3 months.

  This study is sponsored by Millennium Pharmaceuticals, Inc. (Takeda) and has a global target population of 450 participants. Approximately 22 of these patients will be based in the UK across 6 hospitals.

  Summary of Results
  The PLS is see link below Pevonedistat-3001 | Clinical Trial Summary https://eur03.safelinks.protection.outlook.com/?url=https%3A%2F%2Ftrack.pstmrk.it%2F3ts%2Fclinicaltrials.takeda.com%252Fstudy-detail%252F5f6b60294db2bf003ab499ea%253F%253Fpage%253D1%2526idFilter%253DPevonedistat-3001%2FNBTI%2FpZW_AQ%2FAQ%2Fa1fbbd4a-2310-4773-b2ec-480aade5ff1d%2F2%2F8eXm9-0cHb&data=05%7C02%7Cwales.rec2%40wales.nhs.uk%7C6dc96918065e492e49d308ddcdb76961%7Cbb5628b8e3284082a856433c9edc8fae%7C0%7C0%7C638892909979659644%7CUnknown%7CTWFpbGZsb3d8eyJFbXB0eU1hcGkiOnRydWUsIlYiOiIwLjAuMDAwMCIsIlAiOiJXaW4zMiIsIkFOIjoiTWFpbCIsIldUIjoyfQ%3D%3D%7C0%7C%7C%7C&sdata=wbeLmrB0knvB1AbBXXh9c8xyVLZ6MW0MU5PG12Caik4%3D&reserved=0

• REC name

  Wales REC 2

• REC reference

  18/WA/0104

• Date of REC Opinion

  25 Apr 2018

• REC opinion

  Further Information Favourable Opinion